Applicability of a Crowdfunding Application in Obtaining Modulator Treatment for Children with Cystic Fibrosis

Applicability of a Crowdfunding Application in Obtaining Modulator Treatment for Children with Cystic Fibrosis | Research Square window.SnipcartSettings = { analytics: { enabled: false } }; (function() { var accessVector = localStorage.getItem('access_vector') || ''; window.dataLayer = window.dataLayer || []; if (accessVector) { window.dataLayer.push({ user: { profile: { profileInfo: { snid: accessVector } } } }); } })(); (function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start':new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0],j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src='https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f);})(window,document,'script','dataLayer','GTM-K279D39R'); Browse Preprints In Review Journals COVID-19 Preprints AJE Video Bytes Research Tools Research Promotion AJE Professional Editing AJE Rubriq About Preprint Platform In Review Editorial Policies Our Team Advisory Board Help Center Sign In Submit a Preprint Cite Share Download PDF Research Article Applicability of a Crowdfunding Application in Obtaining Modulator Treatment for Children with Cystic Fibrosis Carolina Fagundes Dias Fonseca¹, Leonardo Araujo Pinto² This is a preprint; it has not been peer reviewed by a journal. https://doi.org/ 10.21203/rs.3.rs-6123824/v1 This work is licensed under a CC BY 4.0 License Status: Posted Version 1 posted You are reading this latest preprint version Abstract Purpose: Digital crowdfunding tools appear to be a promising answer to address in a cost-effective manner areas of need in the Brazilian Unified Health System in which the provision of medicines and other services are either absent or insufficient. Methods: Construct validation study in technology innovation research to evaluate the applicability of an original digital solution in online healthcare crowdfunding called “Appadrinha”. The pilot project focused on the judicialization of Trikafta® for pediatric patients at a Cystic Fibrosis (CF) outpatient clinic in Porto Alegre. Our main objective was the success rate of fundraising campaigns. Inclusion criteria were patients aged 6 to 18 years with a positive genetic test with at least one allele of the mutation F508del. Exclusion criteria were family access to Trikafta® via private funds or insurance plans. Results: The inclusion rate was 57% (4/7). All 4 campaigns achieved full sponsorship of their individual budget of R $ 4,000.00 within 24 hours, with a success rate of 100% (4/4). Conclusion: Despite requiring refinement in the development of its functionality, Appadrinha appears to be a promising disruptive and innovative option in the crowdfunding market, working in a complementary way to the Unified Health System, through focusing on solving problems at the level of local communities, not only in the financial sphere, but also as a facilitator between families, health professionals and service providers, based on strategic and financially viable partnerships, thus promoting increased reliability in the healthcare crowdfunding scenario for socioeconomically vulnerable populations. application crowdfunding health applicability Brazil Figures Figure 1 1 Introduction In Brazil, despite the constitutional guarantees of free universal and comprehensive access to healthcare [ 1 – 3 ], it is currently possible to observe in the Unified Health System (UHS) situations in which the provision of medicines and other services is either absent or insufficient [ 4 – 6 ]. One such gap can be observed in the latest treatment options of Cystic Fibrosis (CF), an autosomal recessive genetic disease caused by mutations in the CTFR gene that leads to the formation of thick mucus in the lungs and other organs, with high morbidity and mortality. Since 2019, therapies with modulators have proven promising in the treatment of CF as they act directly on the cause of the disease. Trikafta, which belongs to this drug group, is effective in individuals with at least one allele of the F508del mutation and acts to restore the function of the CTFR protein, resulting in a reduction in hospital admissions due to pulmonary infections, gain in lung function, increased survival and improvement of quality of life. Although Trikafta was incorporated into the Unified Health System [ 7 , 8 ], until July 2024 state pharmacies in Rio Grande do Sul did not have the medication available in stock for collection [ 9 ]. Therefore, as can be noted, several treatments and tests with effectiveness largely supported by current scientific literature, but which are not routinely obtainable through the UHS, can have a highly complex and difficult acquisition process for many families. In socioeconomically vulnerable populations, obtaining them requires a long wait through the judicial courts. Interventions that are more readily available for this population, and specially in Pediatrics, can have a direct effect on quality of life and school attendance, impacting the child's emotional, psychological, and cognitive development and, thus, future income in adulthood, ultimately having considerable repercussions on the intergenerational transmission of social vulnerability [ 10 – 12 ]. The internet and the widespread personal interconnection nowadays through smartphones appear as a promising cost-effective way to overcome deficiencies in the healthcare sector. Insofar as can be observed in scientific databases, currently there has been no published studies, in Brazil or in other countries, on the creation by healthcare professionals of an online crowdfunding tool specifically tailored for health needs within standardization and scientific supervision by a technical medical institution [ 13 ]. This new approach could result in increased reliability regarding the software and the correct use of the funds provided by the population, optimizing the allocation of funds to more urgent and scientifically based demands, and guaranteeing continuation of care after meeting fundraising goals, thus combining health technology with the principles of evidence-based Medicine. 2 Materials and Methods We hypothesized the development and implementation of Appadrinha, a digital fundraising crowdfunding platform specifically designed for the health sector, could lead to better timely access to treatments with clinical indication whose provision is either absent or insufficient in the current Brazilian healthcare system, hence personalizing the resolution of health problems through focusing on the individual and their community, resulting in improved clinical outcomes in a pediatric population with CF in conditions of socioeconomic vulnerability. Our main objective was to evaluate the applicability of the Appadrinha digital application in a clinical scenario via the success rate in effective fundraising capacity for registered cases of patients with both CF and recommendation for initiating treatment with Trikafta by a pediatric pneumologist. Secondary objectives included firstly describing the development process of creating a crowdfunding digital technology focused on the pediatric population, and secondly quantitatively analyzing the rate of patients included in the study among those who fulfilled the inclusion criteria. The chosen design for this technology innovation research was a descriptive study of construct validation of an online crowdfunding application. Through a partnership between the project belonging to the Postgraduate Program of the Pontifícia Universidade Católica do Rio Grande do Sul (PUCRS) School of Medicine and the PUCRS Polytechnic School, a startup was created to develop an online application, called Appadrinha, which allows the raising of funds through collective financing to address the healthcare needs of socioeconomically vulnerable population not met by the state. After development was completed, the platform was implemented in a pilot project to obtain legal assistance for registered pediatric patients linked to PUCRS CF outpatient care clinic. The platform overall test period ran from February 1, 2024 until July 31, 2024. Inclusion criteria therefore comprehended children and adolescents aged 6 to 18 years undergoing follow-up at the CF outpatient clinic of Hospital São Lucas at PUCRS with indications for treatment by their attending pediatric pneumologist with the drug Trikafta® (elexacaftor, tezacaftor and ivacaftor) and with a positive genetic test with at least one allele of the mutation F508del. The exclusion criteria consisted of participants whose family had access to the Trikafta medication privately or had obtained authorization to start treatment via insurance plans. CF was defined according to the WHO International Classification of Diseases (ICD). Study variables were the following – age, family income and ICD. The main outcome of this project was the success rate in obtaining assistance for the judicialization of the drug Trikafta® through personalized legal professional support with funds from a fundraising campaign through the Appadrinha crowdfunding application. The secondary outcome was the inclusion rate of approached patients. As for data analysis, the results are expressed in absolute and relative data, in the format of rates and percentages. This study was reviewed by our Research Ethics Committee which has confirmed that no ethical approval is required, seeing as it only used data from public crowdfunding campaigns. A model of declaration of participation in the Appadrinha application was prepared and signed by each user, the patient’s legal guardian, included in the platform. The application was run in compliance with the personal data protection legislation, included, but not limited to the Brazilian law General Law of Personal Data Protection (LGPD). 3 Results 3.1 Application Development The Appadrinha application began its conceptualization in January 2020. Since its inception, it has undergone multiple phases of evolution, beginning with its visual identity and graphic design at the end of the same year. Its design was inspired by the classic children's illustrations from The Tale of Peter Rabbit [ 14 ] and Winnie-the-Pooh [ 15 ], aiming to create a charity application interface with a playful and welcoming approach. This design seeks to evoke feelings of affection and empathy in users by recalling the joyful simplicity of childhood, thereby fostering a connection between donors and campaign beneficiaries through universal sensations of wonder and admiration for life and nature. This contributes to the construction of a welcoming and accessible digital environment, regardless of social and economic strata. In this way, the application establishes a direct emotional connection with its users while avoiding the ethical pitfalls often associated with charity appeals that rely on tragic personal narratives or images of patients in situations of pain and vulnerability. Particular attention was given to ensuring the representation of diverse physiognomies, as can be seen in Fig. 1 , so that children of varying appearances could feel visually represented when included in the application. This artistic development ensures that the user experience is less emotionally manipulative, more humane, and simultaneously provides psychological protection to the focal point of the donation—the individual in need of assistance. Subsequently, the coding phase began, carried out free of charge during the first semesters of 2021 and 2022 through the Integrative II course of the Information Systems program, in partnership with the Polytechnic School of PUCRS. The planning focused on key aspects for developing a minimum viable product (MVP). The development process was divided into four sprints, which are predefined time periods allocated for completing grouped tasks within a project to be executed together, as defined by Espinha [ 16 ]. This approach ensured that an incremental and usable version of the product could be obtained within the stipulated timeline by meeting each goal. These concepts are part of the Scrum framework, an agile project development methodology widely used in Information Technology. At Appadrinha, each sprint lasted one month, which aligns with the typical range of 1 to 4 weeks. All development phases were documented using the Miro platform [ 17 ]. Regarding the technical aspects of the digital application, GitHub was chosen as the version control tool to manage different versions of the source code and documentation. PostgreSQL was selected as the database management system for handling database information and user interaction. The software stack coordinated the set of software subsystems required to build a complete platform, divided into back-end and front-end. The back end encompasses all processes behind the site, including the server, database, and application, while the front-end is responsible for the architecture, design, content, interaction, performance, and responsiveness of the site. At Appadrinha, Node.js and React.js were utilized for back-end and front-end development, respectively. Amazon Web Services was designated as the data hosting server. User Experience was designed using Figma, which detailed the usage flows for various users, including administrators, physicians, registered donors, and unregistered donors [ 18 ]. The project underwent further refinement between late 2022 and 2024 through its inclusion in the master’s program in the Graduate Program in Child and Adolescent Health at PUCRS. This phase incorporated scientific methods grounded in academic literature to deepen the technical understanding of themes directly influencing the applicability of health technologies, particularly crowdfunding, within the context of startup entrepreneurship. This research served as the foundation for the pilot project. It involved selecting a specific clinical scenario for controlled testing before the project’s introduction to the market (entering the minimum marketable product, or MMP, phase). As part of the application’s refinement and its readiness for testing, a promotional video was produced in 2022 to communicate the project. The video utilized simple and concise messages to facilitate public understanding of the app, considering the fast-paced communication dynamics of social media [ 19 ]. Academic articles also support the effectiveness of such an approach, demonstrating evidence that the use of videos and images increases the likelihood of achieving the total donation target in fundraising campaigns, as they are often preferred by potential donors [ 20 ]. To enhance accessibility, simultaneous translation into Brazilian Sign Language (Libras) was included. Concurrently, a dedicated website for the project was developed, which the “About Us” page in the app redirects to when selected [ 21 ]. 3.2 Applicability Initially, a partnership was established with the CF outpatient clinic at the PUCRS Clinical Center. Patients who met the medical criteria for initiating treatment with the medication Trikafta but had no records in their medical files indicating access to this treatment were selected by the attending physician. This selection was based on the fact that, although Trikafta—a groundbreaking and revolutionary drug for the treatment of a chronic condition like CF—was incorporated into the UHS in September 2023, by the time of this pilot project (conducted between February and July 2024), most state pharmacies in Rio Grande do Sul did not have the medication available in stock for patient distribution. Among the 95 patients under follow-up at the outpatient clinic, 7 met the inclusion criteria for the pilot project and were approached by the medical team on May 20, 2024, to assess their willingness to participate. Of these, 4 patients expressed interest in being included in the project—3 belonging to the first category and 1 to the second. Further user characteristics are described in Table 1 . Thus, the success rate for inclusion in the project among the patients approached was 57%. Table 1 User characteristics Users Age (years) City/State of origin UHS Type of mutation 1 6 Vera Cruz/RS Yes F508del heterozygote 2 11 Pelotas/RS Yes F508del heterozygote 3 12 Erechim/RS Yes F508del homozygote 4 18 Porto Alegre/RS No (Insurance plan) F508del homozygote Secondly, a partnership was established by Appadrinha with the law firm Corrêa da Silva, Martins - Advogados, based in Porto Alegre and specialized in medical law, on May 20, 2024. An agreement was reached for a below-market rate of R $ 4,000.00 per case for the judicialization of Trikafta, with no additional costs for potential appeals. The average market price for such actions varies depending on the medication, as it entails different workloads for the law firm. However, the initial cost typically ranges from six to eight minimum wages (one minimum wage in 2024 corresponds to R $ 1,412 [ 22 ]), with appeals averaging R $ 1,800.00 each. Following the partnership agreement and the consent of families to participate and be contacted by Appadrinha, the attending physician provided the legal guardians' phone numbers to the project. On the same day, contact was made to collect the necessary documents, including proof of family income, identification of the legal guardian and the patient, a medical report confirming the diagnosis of CF and clinical indication for Trikafta, a stamped medical prescription specifying the dosage, and proof of address. Subsequently, families digitally signed a participation agreement for the Appadrinha project, which was drafted free of charge by the partner law firm. One of the key differentiators of the Appadrinha project, particularly in crowdfunding, is that it requires only the operational costs to be covered, as there is no remuneration for the invested capital. The funds come from donations and are directly applied to finance the project's objectives, with no profit motive. On April 12, 2024, the launch date of the campaigns for four pilot project users, all campaigns achieved full sponsorship of their individual budgets of R $ 4,000.00 within 24 hours, resulting in a 100% success rate. The campaigns were promoted through a friends-and-family approach, sharing the app's link via WhatsApp. This process mirrored the early lifecycle of many startups, as highlighted in the literature, where initial funding typically comes from the founders themselves, friends and family, and, if possible, angel investors [ 23 ]. Donations were made to the founder's account, with all collected funds entirely allocated to the campaigns' purposes without any fees retained by the application. The next day, the individual campaign funds were transferred to the partner law firm, which then contacted the families to finalize the contracts for the judicialization of Trikafta. The Appadrinha project, through its founder, was cited as the funding source in each contract. During the judicialization process, Appadrinha maintained contact with families, the medical team at the CF Clinic, and the law firm, mediating the need for documentation such as medical reports and prescriptions. A significant challenge during this phase was obtaining the genetic test results that identified each user's CFTR gene mutation profile. Without this, families could not register their medication request with the state pharmacy, and the denial was required to be attached to the legal process. This is due to the medication's demonstrated efficacy only in patients with at least one F508del gene mutation allele. However, this test is usually performed in infancy near the time of diagnosis, and its misplacement is not uncommon. Two families lacked physical copies of the report and could not recall the laboratory where the test had been conducted. Appadrinha had to mediate the retrieval of this document. The families faced difficulties not only in understanding the nature of the genetic test but also in practical matters like making phone calls to the laboratories. Initially, the project contacted the physicians responsible at the time of diagnosis to determine the laboratories they used for genetic testing during that period. Subsequently, the identified laboratories were contacted to verify if users had undergone testing there. One child’s report was located at a laboratory and forwarded to the patient's mother. In the second case, the laboratory requested direct contact from the legal guardian. The project provided the mother with guidance on submitting an email request. Due to difficulties in drafting and sending the email, the mother sought Appadrinha’s help, which assisted her in composing the email. After two weeks of searching hospital records, the test could not be located. Consequently, the project recommended the family obtain the patient’s discharge summary from the hospital, hoping the genetic mutation would be documented and could be used as evidence for the state pharmacy. The other two families assisted were able to obtain the genetic test results through their attending physicians. By August 2024, these two patients already had access to the medication. Thus, contrary to initial expectations that the greatest challenge would be securing funds for crowdfunding campaigns, the main difficulty in ensuring users' access to medication lay in assisting families with the bureaucratic processes. This included obtaining documents, prescriptions, and test results and maintaining timely communication with the law firm at every stage. The project often struggled to contact families, sometimes for days or weeks, whether via WhatsApp or phone calls. Additionally, due to mobility challenges and unfamiliarity with technical terms like test names, the project had to play an active role in ensuring court deadlines were met, repeatedly reminding families of the necessary documentation and its intended recipient. Without this intervention, even with successful fundraising, the ultimate goal of promptly initiating treatment for each patient might not have been achieved, given the critical importance of adhering to legal deadlines. This highlights another unique aspect of Appadrinha: it is not merely a crowdfunding platform but an innovative and disruptive process addressing medical needs in Brazil’s context, particularly for socioeconomically vulnerable families. The project's personalized support and specialized medical knowledge allow for new solutions within a healthcare system whose bureaucratic hurdles often alienate those who need it most. Traditional complementary measures for securing the constitutional right to health, such as legal assistance for obtaining medication, prove insufficient because individuals often lack the tools necessary to navigate them, including literacy, understanding technical terms, and time off work for travel to state pharmacies, medical offices, and law firms. 4 Discussion This pilot project achieved a 100% success rate in its four fundraising campaigns to obtain legal assistance for the judicialization process of the drug Trikafta for patients with CF who were undergoing outpatient pediatric pulmonology treatment in the city of Porto Alegre, Brazil, thereby helping to provide access to a unique therapy in its potential to alter the natural history of the disease through its specific action on the etiological cause, the genetic mutation of the CFTR protein. This result goes beyond the mere functionality of the crowdfunding software, including raising awareness and mobilization to obtain donations, but also depends on the partnerships established with the PUCRS CF outpatient clinic and with the law firm Corrêa da Silva & Martins, on the research team’s proactive role of resolving documentation and communication problems with families even after the start of legal processes, and, lastly, on the development and subsequent assistance in maintenance of the software provided by the PUCRS Polytechnic School at no cost. Furthermore, discrepancy was clearly observed in comparison to current crowdfunding platforms in the startup market, which only provide access to a fundraising tool, with clear ethical issues for both the user and donors pertaining to health campaigns, as well as for the healthcare professionals involved with the cases, thereupon creating difficulties in terms of data reliability and of guarantees that the money raised will in fact be used for what was announced. By using digital technology in a disruptive way to traditional financing scenarios and functioning in a complementary way to the current UHS treatment funding system, through focusing on problem solving at the level of local communities, we aimed to help healthcare professionals to provide excellent care to their patients even in economically unfavorable scenarios in a country with evident socioeconomic inequalities in the social strata. To achieve that objective, we relied on strategic and financially viable partnerships. In view of this, the project's contribution to the United Nations Sustainable Development Goals is made evident, specifically for numbers three “Health and well-being” and ten “Reducing inequalities” [ 24 ]. In the future, it would be crucial, through additional investment, to increase the platform payment options with credit and debit cards. A dedicated team of computing engineers would be essential to deal with system problems as they occur, ensuring a quick and efficient response. Providing other login options through social media such as Google and Facebook would also be ideal, as would the option to share online cases and donations, thus encouraging new individuals to participate in the platform. Migrating it to application stores for Android, IOS and Windows Phone would facilitate access, expanding the number of potential donors. Furthermore, a new set of accounts could be tailored for healthcare professionals providing services (at present only medical doctors have a separate login in order to be able to register their patients), such as speech therapists, physiotherapists, physical educators, nutritionists and specialist doctors, in which they could register availability for outpatient appointments, thus adding an application function as an intermediary between these services and socioeconomically vulnerable patients with such needs. Finally, after the experimental period of initial technology development and later usability and applicability analysis, the implementation of this application in the clinical scenario of patients with CF was successfully demonstrated. The focus henceforth will be on seeking investments to address the previously mentioned software issues and limitations in a continuous effort to expand the startup’s field of impact with new partnerships in different care settings, so that a broader number of people can potentially benefit from this project. In this manner, Appadrinha constitutes a problem-solving model that goes beyond its beginnings as a crowdfunding application, having a role as an active agent facilitating partnerships between institutions relevant to patient-centered solutions in the healthcare area. Declarations Funding The authors received no financial support for the research, authorship, and publication of this article. Conflict of Interest The authors certify that they have no affiliations with or involvement in any organization or entity with any financial or non-financial interest in the subject matter or materials discussed in this manuscript Competing Interests The authors have no relevant financial or non-financial interests to disclose. Consent to participate Verbal informed consent was obtained from legal guardians. Additional informed consent was obtained from all individual participants’ legal guardians for whom identifying information is included in this article. Consent to publish Legal guardians gave verbal informed consent regarding publishing data Author contributions All authors contributed to the study conception. L.A.P. was responsible for study design and methods. Material preparation, data collection and analysis were performed by C. F. The first draft of the manuscript was written by C.F. and L.A.P. reviewed and commented on previous versions of the manuscript. All authors read and approved the final manuscript. Data and code availability statement All data in this study, including the Appadrinha application code, is available on request. The application can be accessed through the following link http://appadrinha-server.s3-website-us-east-1.amazonaws.com/ Acknowledgements The authors thank the graphic designer, Júlia Schaan Fernandes, for the development of the application’s visual identity. They also give special thanks to the professors Jorge Horácio Audi and Cássio Trindade of the Information Systems course at the Polytechnic School of PUCRS, and to all their students, for dedicating their knowledge, time and classes to the free development of the initial version of the Appadrinha application, as well as to the Experimental Software Engineering Agency of the Polytechnic School, for providing its space for course activities. Clinical trial number: not applicable. References Brasil. 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BMJ Glob Health. 2022;7(7):e009110. doi: 10.1136/bmjgh-2022-009110 Appadrinha. Página principal [Internet]. Porto Alegre; 2022. Available from: https://appadrinha.com.br/ Salário mínimo de 2024 terá ganho real e crescerá três pontos percentuais além dos 3,85% da inflação [Internet]. Planalto. Available from: https://www.gov.br/planalto/pt-br/acompanhe-o-planalto/noticias/2023/12/salario-minimo-de-2024-tera-ganho-real-e-crescera-3pp-alem-dos-3-85-da-inflacao Moritz A, Block JH. Crowdfunding: a literature review and research directions. FGF Stud Small Bus Entrep. 2016;25–53. doi: 10.1007/978-3-319-18017-5_3 ONU. Objetivos de Desenvolvimento Sustentável | As Nações Unidas no Brasil [Internet]. Available from: https://brasil.un.org/pt-br/sdgs Additional Declarations No competing interests reported. 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Since 2019, therapies with modulators have proven promising in the treatment of CF as they act directly on the cause of the disease. Trikafta, which belongs to this drug group, is effective in individuals with at least one allele of the F508del mutation and acts to restore the function of the CTFR protein, resulting in a reduction in hospital admissions due to pulmonary infections, gain in lung function, increased survival and improvement of quality of life. Although Trikafta was incorporated into the Unified Health System [\u003cspan citationid=\"CR7\" class=\"CitationRef\"\u003e7\u003c/span\u003e, \u003cspan citationid=\"CR8\" class=\"CitationRef\"\u003e8\u003c/span\u003e], until July 2024 state pharmacies in Rio Grande do Sul did not have the medication available in stock for collection [\u003cspan citationid=\"CR9\" class=\"CitationRef\"\u003e9\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eTherefore, as can be noted, several treatments and tests with effectiveness largely supported by current scientific literature, but which are not routinely obtainable through the UHS, can have a highly complex and difficult acquisition process for many families. In socioeconomically vulnerable populations, obtaining them requires a long wait through the judicial courts. Interventions that are more readily available for this population, and specially in Pediatrics, can have a direct effect on quality of life and school attendance, impacting the child's emotional, psychological, and cognitive development and, thus, future income in adulthood, ultimately having considerable repercussions on the intergenerational transmission of social vulnerability [\u003cspan additionalcitationids=\"CR11\" citationid=\"CR10\" class=\"CitationRef\"\u003e10\u003c/span\u003e\u0026ndash;\u003cspan citationid=\"CR12\" class=\"CitationRef\"\u003e12\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eThe internet and the widespread personal interconnection nowadays through smartphones appear as a promising cost-effective way to overcome deficiencies in the healthcare sector. Insofar as can be observed in scientific databases, currently there has been no published studies, in Brazil or in other countries, on the creation by healthcare professionals of an online crowdfunding tool specifically tailored for health needs within standardization and scientific supervision by a technical medical institution [\u003cspan citationid=\"CR13\" class=\"CitationRef\"\u003e13\u003c/span\u003e]. This new approach could result in increased reliability regarding the software and the correct use of the funds provided by the population, optimizing the allocation of funds to more urgent and scientifically based demands, and guaranteeing continuation of care after meeting fundraising goals, thus combining health technology with the principles of evidence-based Medicine.\u003c/p\u003e"},{"header":"2 Materials and Methods","content":"\u003cp\u003eWe hypothesized the development and implementation of Appadrinha, a digital fundraising crowdfunding platform specifically designed for the health sector, could lead to better timely access to treatments with clinical indication whose provision is either absent or insufficient in the current Brazilian healthcare system, hence personalizing the resolution of health problems through focusing on the individual and their community, resulting in improved clinical outcomes in a pediatric population with CF in conditions of socioeconomic vulnerability.\u003c/p\u003e \u003cp\u003eOur main objective was to evaluate the applicability of the Appadrinha digital application in a clinical scenario via the success rate in effective fundraising capacity for registered cases of patients with both CF and recommendation for initiating treatment with Trikafta by a pediatric pneumologist. Secondary objectives included firstly describing the development process of creating a crowdfunding digital technology focused on the pediatric population, and secondly quantitatively analyzing the rate of patients included in the study among those who fulfilled the inclusion criteria.\u003c/p\u003e \u003cp\u003eThe chosen design for this technology innovation research was a descriptive study of construct validation of an online crowdfunding application. Through a partnership between the project belonging to the Postgraduate Program of the Pontif\u0026iacute;cia Universidade Cat\u0026oacute;lica do Rio Grande do Sul (PUCRS) School of Medicine and the PUCRS Polytechnic School, a startup was created to develop an online application, called Appadrinha, which allows the raising of funds through collective financing to address the healthcare needs of socioeconomically vulnerable population not met by the state. After development was completed, the platform was implemented in a pilot project to obtain legal assistance for registered pediatric patients linked to PUCRS CF outpatient care clinic. The platform overall test period ran from February 1, 2024 until July 31, 2024.\u003c/p\u003e \u003cp\u003eInclusion criteria therefore comprehended children and adolescents aged 6 to 18 years undergoing follow-up at the CF outpatient clinic of Hospital S\u0026atilde;o Lucas at PUCRS with indications for treatment by their attending pediatric pneumologist with the drug Trikafta\u0026reg; (elexacaftor, tezacaftor and ivacaftor) and with a positive genetic test with at least one allele of the mutation F508del. The exclusion criteria consisted of participants whose family had access to the Trikafta medication privately or had obtained authorization to start treatment via insurance plans. CF was defined according to the WHO International Classification of Diseases (ICD).\u003c/p\u003e \u003cp\u003eStudy variables were the following \u0026ndash; age, family income and ICD.\u003c/p\u003e \u003cp\u003eThe main outcome of this project was the success rate in obtaining assistance for the judicialization of the drug Trikafta\u0026reg; through personalized legal professional support with funds from a fundraising campaign through the Appadrinha crowdfunding application. The secondary outcome was the inclusion rate of approached patients.\u003c/p\u003e \u003cp\u003eAs for data analysis, the results are expressed in absolute and relative data, in the format of rates and percentages.\u003c/p\u003e \u003cp\u003eThis study was reviewed by our Research Ethics Committee which has confirmed that no ethical approval is required, seeing as it only used data from public crowdfunding campaigns. A model of declaration of participation in the Appadrinha application was prepared and signed by each user, the patient\u0026rsquo;s legal guardian, included in the platform. The application was run in compliance with the personal data protection legislation, included, but not limited to the Brazilian law General Law of Personal Data Protection (LGPD).\u003c/p\u003e"},{"header":"3 Results","content":"\u003cdiv id=\"Sec4\" class=\"Section2\"\u003e \u003ch2\u003e3.1 Application Development\u003c/h2\u003e \u003cp\u003eThe Appadrinha application began its conceptualization in January 2020. Since its inception, it has undergone multiple phases of evolution, beginning with its visual identity and graphic design at the end of the same year. Its design was inspired by the classic children's illustrations from The Tale of Peter Rabbit [\u003cspan citationid=\"CR14\" class=\"CitationRef\"\u003e14\u003c/span\u003e] and Winnie-the-Pooh [\u003cspan citationid=\"CR15\" class=\"CitationRef\"\u003e15\u003c/span\u003e], aiming to create a charity application interface with a playful and welcoming approach. This design seeks to evoke feelings of affection and empathy in users by recalling the joyful simplicity of childhood, thereby fostering a connection between donors and campaign beneficiaries through universal sensations of wonder and admiration for life and nature. This contributes to the construction of a welcoming and accessible digital environment, regardless of social and economic strata. In this way, the application establishes a direct emotional connection with its users while avoiding the ethical pitfalls often associated with charity appeals that rely on tragic personal narratives or images of patients in situations of pain and vulnerability. Particular attention was given to ensuring the representation of diverse physiognomies, as can be seen in Fig.\u0026nbsp;\u003cspan refid=\"Fig1\" class=\"InternalRef\"\u003e1\u003c/span\u003e, so that children of varying appearances could feel visually represented when included in the application. This artistic development ensures that the user experience is less emotionally manipulative, more humane, and simultaneously provides psychological protection to the focal point of the donation\u0026mdash;the individual in need of assistance.\u003c/p\u003e \u003cp\u003e \u003c/p\u003e \u003cp\u003eSubsequently, the coding phase began, carried out free of charge during the first semesters of 2021 and 2022 through the Integrative II course of the Information Systems program, in partnership with the Polytechnic School of PUCRS. The planning focused on key aspects for developing a minimum viable product (MVP). The development process was divided into four sprints, which are predefined time periods allocated for completing grouped tasks within a project to be executed together, as defined by Espinha [\u003cspan citationid=\"CR16\" class=\"CitationRef\"\u003e16\u003c/span\u003e]. This approach ensured that an incremental and usable version of the product could be obtained within the stipulated timeline by meeting each goal. These concepts are part of the Scrum framework, an agile project development methodology widely used in Information Technology. At Appadrinha, each sprint lasted one month, which aligns with the typical range of 1 to 4 weeks. All development phases were documented using the Miro platform [\u003cspan citationid=\"CR17\" class=\"CitationRef\"\u003e17\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eRegarding the technical aspects of the digital application, GitHub was chosen as the version control tool to manage different versions of the source code and documentation. PostgreSQL was selected as the database management system for handling database information and user interaction. The software stack coordinated the set of software subsystems required to build a complete platform, divided into back-end and front-end. The back end encompasses all processes behind the site, including the server, database, and application, while the front-end is responsible for the architecture, design, content, interaction, performance, and responsiveness of the site. At Appadrinha, Node.js and React.js were utilized for back-end and front-end development, respectively. Amazon Web Services was designated as the data hosting server. User Experience was designed using Figma, which detailed the usage flows for various users, including administrators, physicians, registered donors, and unregistered donors [\u003cspan citationid=\"CR18\" class=\"CitationRef\"\u003e18\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eThe project underwent further refinement between late 2022 and 2024 through its inclusion in the master\u0026rsquo;s program in the Graduate Program in Child and Adolescent Health at PUCRS. This phase incorporated scientific methods grounded in academic literature to deepen the technical understanding of themes directly influencing the applicability of health technologies, particularly crowdfunding, within the context of startup entrepreneurship. This research served as the foundation for the pilot project. It involved selecting a specific clinical scenario for controlled testing before the project\u0026rsquo;s introduction to the market (entering the minimum marketable product, or MMP, phase).\u003c/p\u003e \u003cp\u003eAs part of the application\u0026rsquo;s refinement and its readiness for testing, a promotional video was produced in 2022 to communicate the project. The video utilized simple and concise messages to facilitate public understanding of the app, considering the fast-paced communication dynamics of social media [\u003cspan citationid=\"CR19\" class=\"CitationRef\"\u003e19\u003c/span\u003e]. Academic articles also support the effectiveness of such an approach, demonstrating evidence that the use of videos and images increases the likelihood of achieving the total donation target in fundraising campaigns, as they are often preferred by potential donors [\u003cspan citationid=\"CR20\" class=\"CitationRef\"\u003e20\u003c/span\u003e]. To enhance accessibility, simultaneous translation into Brazilian Sign Language (Libras) was included. Concurrently, a dedicated website for the project was developed, which the \u0026ldquo;About Us\u0026rdquo; page in the app redirects to when selected [\u003cspan citationid=\"CR21\" class=\"CitationRef\"\u003e21\u003c/span\u003e].\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec5\" class=\"Section2\"\u003e \u003ch2\u003e3.2 Applicability\u003c/h2\u003e \u003cp\u003eInitially, a partnership was established with the CF outpatient clinic at the PUCRS Clinical Center. Patients who met the medical criteria for initiating treatment with the medication Trikafta but had no records in their medical files indicating access to this treatment were selected by the attending physician. This selection was based on the fact that, although Trikafta\u0026mdash;a groundbreaking and revolutionary drug for the treatment of a chronic condition like CF\u0026mdash;was incorporated into the UHS in September 2023, by the time of this pilot project (conducted between February and July 2024), most state pharmacies in Rio Grande do Sul did not have the medication available in stock for patient distribution.\u003c/p\u003e \u003cp\u003eAmong the 95 patients under follow-up at the outpatient clinic, 7 met the inclusion criteria for the pilot project and were approached by the medical team on May 20, 2024, to assess their willingness to participate. Of these, 4 patients expressed interest in being included in the project\u0026mdash;3 belonging to the first category and 1 to the second. Further user characteristics are described in Table\u0026nbsp;\u003cspan refid=\"Tab1\" class=\"InternalRef\"\u003e1\u003c/span\u003e. Thus, the success rate for inclusion in the project among the patients approached was 57%.\u003c/p\u003e \u003cp\u003e \u003cdiv class=\"gridtable\"\u003e\u003ctable float=\"Yes\" id=\"Tab1\" border=\"1\"\u003e \u003ccaption language=\"En\"\u003e \u003cdiv class=\"CaptionNumber\"\u003eTable 1\u003c/div\u003e \u003cdiv class=\"CaptionContent\"\u003e \u003cp\u003eUser characteristics\u003c/p\u003e \u003c/div\u003e \u003c/caption\u003e \u003ccolgroup cols=\"5\"\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c1\" colnum=\"1\"\u003e\u003c/div\u003e \u003cdiv align=\"char\" char=\".\" class=\"colspec\" colname=\"c2\" colnum=\"2\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c3\" colnum=\"3\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c4\" colnum=\"4\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c5\" colnum=\"5\"\u003e\u003c/div\u003e \u003cthead\u003e \u003ctr\u003e \u003cth align=\"left\" colname=\"c1\"\u003e \u003cp\u003eUsers\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c2\"\u003e \u003cp\u003eAge (years)\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c3\"\u003e \u003cp\u003eCity/State of origin\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c4\"\u003e \u003cp\u003eUHS\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c5\"\u003e \u003cp\u003eType of mutation\u003c/p\u003e \u003c/th\u003e \u003c/tr\u003e \u003c/thead\u003e \u003ctbody\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003e1\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e6\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003eVera Cruz/RS\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003eYes\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c5\"\u003e \u003cp\u003eF508del heterozygote\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003e2\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e11\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003ePelotas/RS\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003eYes\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c5\"\u003e \u003cp\u003eF508del heterozygote\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003e3\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e12\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003eErechim/RS\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003eYes\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c5\"\u003e \u003cp\u003eF508del homozygote\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003e4\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e18\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003ePorto Alegre/RS\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003eNo (Insurance plan)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c5\"\u003e \u003cp\u003eF508del homozygote\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003c/tbody\u003e \u003c/colgroup\u003e \u003c/table\u003e\u003c/div\u003e \u003c/p\u003e \u003cp\u003e Secondly, a partnership was established by Appadrinha with the law firm Corr\u0026ecirc;a da Silva, Martins - Advogados, based in Porto Alegre and specialized in medical law, on May 20, 2024. An agreement was reached for a below-market rate of R\u003cspan\u003e$\u003c/span\u003e4,000.00 per case for the judicialization of Trikafta, with no additional costs for potential appeals. The average market price for such actions varies depending on the medication, as it entails different workloads for the law firm. However, the initial cost typically ranges from six to eight minimum wages (one minimum wage in 2024 corresponds to R\u003cspan\u003e$\u003c/span\u003e1,412 [\u003cspan citationid=\"CR22\" class=\"CitationRef\"\u003e22\u003c/span\u003e]), with appeals averaging R\u003cspan\u003e$\u003c/span\u003e1,800.00 each.\u003c/p\u003e \u003cp\u003eFollowing the partnership agreement and the consent of families to participate and be contacted by Appadrinha, the attending physician provided the legal guardians' phone numbers to the project. On the same day, contact was made to collect the necessary documents, including proof of family income, identification of the legal guardian and the patient, a medical report confirming the diagnosis of CF and clinical indication for Trikafta, a stamped medical prescription specifying the dosage, and proof of address. Subsequently, families digitally signed a participation agreement for the Appadrinha project, which was drafted free of charge by the partner law firm.\u003c/p\u003e \u003cp\u003eOne of the key differentiators of the Appadrinha project, particularly in crowdfunding, is that it requires only the operational costs to be covered, as there is no remuneration for the invested capital. The funds come from donations and are directly applied to finance the project's objectives, with no profit motive. On April 12, 2024, the launch date of the campaigns for four pilot project users, all campaigns achieved full sponsorship of their individual budgets of R\u003cspan\u003e$\u003c/span\u003e4,000.00 within 24 hours, resulting in a 100% success rate. The campaigns were promoted through a friends-and-family approach, sharing the app's link via WhatsApp. This process mirrored the early lifecycle of many startups, as highlighted in the literature, where initial funding typically comes from the founders themselves, friends and family, and, if possible, angel investors [\u003cspan citationid=\"CR23\" class=\"CitationRef\"\u003e23\u003c/span\u003e]. Donations were made to the founder's account, with all collected funds entirely allocated to the campaigns' purposes without any fees retained by the application. The next day, the individual campaign funds were transferred to the partner law firm, which then contacted the families to finalize the contracts for the judicialization of Trikafta. The Appadrinha project, through its founder, was cited as the funding source in each contract.\u003c/p\u003e \u003cp\u003eDuring the judicialization process, Appadrinha maintained contact with families, the medical team at the CF Clinic, and the law firm, mediating the need for documentation such as medical reports and prescriptions. A significant challenge during this phase was obtaining the genetic test results that identified each user's CFTR gene mutation profile. Without this, families could not register their medication request with the state pharmacy, and the denial was required to be attached to the legal process. This is due to the medication's demonstrated efficacy only in patients with at least one F508del gene mutation allele. However, this test is usually performed in infancy near the time of diagnosis, and its misplacement is not uncommon.\u003c/p\u003e \u003cp\u003eTwo families lacked physical copies of the report and could not recall the laboratory where the test had been conducted. Appadrinha had to mediate the retrieval of this document. The families faced difficulties not only in understanding the nature of the genetic test but also in practical matters like making phone calls to the laboratories. Initially, the project contacted the physicians responsible at the time of diagnosis to determine the laboratories they used for genetic testing during that period. Subsequently, the identified laboratories were contacted to verify if users had undergone testing there.\u003c/p\u003e \u003cp\u003eOne child\u0026rsquo;s report was located at a laboratory and forwarded to the patient's mother. In the second case, the laboratory requested direct contact from the legal guardian. The project provided the mother with guidance on submitting an email request. Due to difficulties in drafting and sending the email, the mother sought Appadrinha\u0026rsquo;s help, which assisted her in composing the email. After two weeks of searching hospital records, the test could not be located. Consequently, the project recommended the family obtain the patient\u0026rsquo;s discharge summary from the hospital, hoping the genetic mutation would be documented and could be used as evidence for the state pharmacy. The other two families assisted were able to obtain the genetic test results through their attending physicians. By August 2024, these two patients already had access to the medication.\u003c/p\u003e \u003cp\u003eThus, contrary to initial expectations that the greatest challenge would be securing funds for crowdfunding campaigns, the main difficulty in ensuring users' access to medication lay in assisting families with the bureaucratic processes. This included obtaining documents, prescriptions, and test results and maintaining timely communication with the law firm at every stage. The project often struggled to contact families, sometimes for days or weeks, whether via WhatsApp or phone calls. Additionally, due to mobility challenges and unfamiliarity with technical terms like test names, the project had to play an active role in ensuring court deadlines were met, repeatedly reminding families of the necessary documentation and its intended recipient. Without this intervention, even with successful fundraising, the ultimate goal of promptly initiating treatment for each patient might not have been achieved, given the critical importance of adhering to legal deadlines.\u003c/p\u003e \u003cp\u003eThis highlights another unique aspect of Appadrinha: it is not merely a crowdfunding platform but an innovative and disruptive process addressing medical needs in Brazil\u0026rsquo;s context, particularly for socioeconomically vulnerable families. The project's personalized support and specialized medical knowledge allow for new solutions within a healthcare system whose bureaucratic hurdles often alienate those who need it most. Traditional complementary measures for securing the constitutional right to health, such as legal assistance for obtaining medication, prove insufficient because individuals often lack the tools necessary to navigate them, including literacy, understanding technical terms, and time off work for travel to state pharmacies, medical offices, and law firms.\u003c/p\u003e \u003c/div\u003e"},{"header":"4 Discussion","content":"\u003cp\u003e This pilot project achieved a 100% success rate in its four fundraising campaigns to obtain legal assistance for the judicialization process of the drug Trikafta for patients with CF who were undergoing outpatient pediatric pulmonology treatment in the city of Porto Alegre, Brazil, thereby helping to provide access to a unique therapy in its potential to alter the natural history of the disease through its specific action on the etiological cause, the genetic mutation of the CFTR protein. This result goes beyond the mere functionality of the crowdfunding software, including raising awareness and mobilization to obtain donations, but also depends on the partnerships established with the PUCRS CF outpatient clinic and with the law firm Corr\u0026ecirc;a da Silva \u0026amp; Martins, on the research team\u0026rsquo;s proactive role of resolving documentation and communication problems with families even after the start of legal processes, and, lastly, on the development and subsequent assistance in maintenance of the software provided by the PUCRS Polytechnic School at no cost.\u003c/p\u003e \u003cp\u003eFurthermore, discrepancy was clearly observed in comparison to current crowdfunding platforms in the startup market, which only provide access to a fundraising tool, with clear ethical issues for both the user and donors pertaining to health campaigns, as well as for the healthcare professionals involved with the cases, thereupon creating difficulties in terms of data reliability and of guarantees that the money raised will in fact be used for what was announced. By using digital technology in a disruptive way to traditional financing scenarios and functioning in a complementary way to the current UHS treatment funding system, through focusing on problem solving at the level of local communities, we aimed to help healthcare professionals to provide excellent care to their patients even in economically unfavorable scenarios in a country with evident socioeconomic inequalities in the social strata. To achieve that objective, we relied on strategic and financially viable partnerships. In view of this, the project's contribution to the United Nations Sustainable Development Goals is made evident, specifically for numbers three \u0026ldquo;Health and well-being\u0026rdquo; and ten \u0026ldquo;Reducing inequalities\u0026rdquo; [\u003cspan citationid=\"CR24\" class=\"CitationRef\"\u003e24\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eIn the future, it would be crucial, through additional investment, to increase the platform payment options with credit and debit cards. A dedicated team of computing engineers would be essential to deal with system problems as they occur, ensuring a quick and efficient response. Providing other login options through social media such as Google and Facebook would also be ideal, as would the option to share online cases and donations, thus encouraging new individuals to participate in the platform. Migrating it to application stores for Android, IOS and Windows Phone would facilitate access, expanding the number of potential donors. Furthermore, a new set of accounts could be tailored for healthcare professionals providing services (at present only medical doctors have a separate login in order to be able to register their patients), such as speech therapists, physiotherapists, physical educators, nutritionists and specialist doctors, in which they could register availability for outpatient appointments, thus adding an application function as an intermediary between these services and socioeconomically vulnerable patients with such needs.\u003c/p\u003e \u003cp\u003eFinally, after the experimental period of initial technology development and later usability and applicability analysis, the implementation of this application in the clinical scenario of patients with CF was successfully demonstrated. The focus henceforth will be on seeking investments to address the previously mentioned software issues and limitations in a continuous effort to expand the startup\u0026rsquo;s field of impact with new partnerships in different care settings, so that a broader number of people can potentially benefit from this project. In this manner, Appadrinha constitutes a problem-solving model that goes beyond its beginnings as a crowdfunding application, having a role as an active agent facilitating partnerships between institutions relevant to patient-centered solutions in the healthcare area.\u003c/p\u003e"},{"header":"Declarations","content":"\u003cp\u003eFunding\u003c/p\u003e\n\u003cp\u003eThe authors received no financial support for the research, authorship, and publication of this article.\u003c/p\u003e\n\u003cp\u003eConflict of Interest\u003c/p\u003e\n\u003cp\u003eThe authors certify that they have no affiliations with or involvement in any organization or entity with any financial or non-financial interest in the subject matter or materials discussed in this manuscript\u003c/p\u003e\n\u003cp\u003eCompeting Interests\u003c/p\u003e\n\u003cp\u003eThe authors have no relevant financial or non-financial interests to disclose.\u003c/p\u003e\n\u003cp\u003eConsent to participate\u003c/p\u003e\n\u003cp\u003eVerbal informed consent was obtained from legal guardians.\u003c/p\u003e\n\u003cp\u003eAdditional informed consent was obtained from all individual participants\u0026rsquo; legal guardians for whom identifying information is included in this article.\u003c/p\u003e\n\u003cp\u003eConsent to publish\u003c/p\u003e\n\u003cp\u003eLegal guardians gave verbal informed consent regarding publishing data\u003c/p\u003e\n\u003cp\u003eAuthor contributions\u003c/p\u003e\n\u003cp\u003eAll authors contributed to the study conception. L.A.P. was responsible for study design and methods. Material preparation, data collection and analysis were performed by C. F. The first draft of the manuscript was written by C.F. and L.A.P. reviewed and commented on previous versions of the manuscript. All authors read and approved the final manuscript.\u003c/p\u003e\n\u003cp\u003eData and code availability statement\u003c/p\u003e\n\u003cp\u003eAll data in this study, including the Appadrinha application code, is available on request. The application can be accessed through the following link \u003cu\u003ehttp://appadrinha-server.s3-website-us-east-1.amazonaws.com/\u003c/u\u003e\u003c/p\u003e\n\u003cp\u003eAcknowledgements\u003c/p\u003e\n\u003cp\u003eThe authors thank the graphic designer, J\u0026uacute;lia Schaan Fernandes, for the development of the application\u0026rsquo;s visual identity. They also give special thanks to the professors Jorge Hor\u0026aacute;cio Audi and C\u0026aacute;ssio Trindade of the Information Systems course at the Polytechnic School of PUCRS, and to all their students, for dedicating their knowledge, time and \u0026nbsp;classes to the free development of the initial version of the Appadrinha application, as well as to the Experimental Software Engineering Agency of the Polytechnic School, for providing its space for course activities.\u003c/p\u003e\n\u003cp\u003eClinical trial number: not applicable.\u003c/p\u003e"},{"header":"References","content":"\u003col\u003e\u003cli\u003e\u003cspan\u003eBrasil. Constitui\u0026ccedil;\u0026atilde;o da Rep\u0026uacute;blica Federativa do Brasil de 1988 [Internet]. Bras\u0026iacute;lia: Presid\u0026ecirc;ncia da Rep\u0026uacute;blica; 1988. 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Available from: \u003cspan class=\"ExternalRef\"\u003e\u003cspan class=\"RefSource\"\u003ehttps://www.gov.br/planalto/pt-br/acompanhe-o-planalto/noticias/2023/12/salario-minimo-de-2024-tera-ganho-real-e-crescera-3pp-alem-dos-3-85-da-inflacao\u003c/span\u003e\u003cspan address=\"https://www.gov.br/planalto/pt-br/acompanhe-o-planalto/noticias/2023/12/salario-minimo-de-2024-tera-ganho-real-e-crescera-3pp-alem-dos-3-85-da-inflacao\" targettype=\"URL\" class=\"RefTarget\"\u003e\u003c/span\u003e\u003c/span\u003e\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eMoritz A, Block JH. Crowdfunding: a literature review and research directions. FGF Stud Small Bus Entrep. 2016;25\u0026ndash;53. doi: \u003cspan class=\"ExternalRef\"\u003e\u003cspan class=\"RefSource\"\u003e10.1007/978-3-319-18017-5_3\u003c/span\u003e\u003cspan address=\"10.1007/978-3-319-18017-5_3\" targettype=\"DOI\" class=\"RefTarget\"\u003e\u003c/span\u003e\u003c/span\u003e\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eONU. Objetivos de Desenvolvimento Sustent\u0026aacute;vel | As Na\u0026ccedil;\u0026otilde;es Unidas no Brasil [Internet]. Available from: \u003cspan class=\"ExternalRef\"\u003e\u003cspan class=\"RefSource\"\u003ehttps://brasil.un.org/pt-br/sdgs\u003c/span\u003e\u003cspan address=\"https://brasil.un.org/pt-br/sdgs\" targettype=\"URL\" class=\"RefTarget\"\u003e\u003c/span\u003e\u003c/span\u003e\u003c/span\u003e\u003c/li\u003e\u003c/ol\u003e"}],"fulltextSource":"","fullText":"","funders":[],"hasAdminPriorityOnWorkflow":false,"hasManuscriptDocX":true,"hasOptedInToPreprint":true,"hasPassedJournalQc":"","hasAnyPriority":false,"hideJournal":true,"highlight":"","institution":"","isAcceptedByJournal":false,"isAuthorSuppliedPdf":false,"isDeskRejected":"","isHiddenFromSearch":false,"isInQc":false,"isInWorkflow":false,"isPdf":false,"isPdfUpToDate":true,"isWithdrawnOrRetracted":false,"journal":{"display":true,"email":"[email protected]","identity":"researchsquare","isNatureJournal":false,"hasQc":true,"allowDirectSubmit":true,"externalIdentity":"","sideBox":"","snPcode":"","submissionUrl":"/submission","title":"Research Square","twitterHandle":"researchsquare","acdcEnabled":true,"dfaEnabled":false,"editorialSystem":"","reportingPortfolio":"","inReviewEnabled":false,"inReviewRevisionsEnabled":true},"keywords":"application, crowdfunding, health, applicability, Brazil","lastPublishedDoi":"10.21203/rs.3.rs-6123824/v1","lastPublishedDoiUrl":"https://doi.org/10.21203/rs.3.rs-6123824/v1","license":{"name":"CC BY 4.0","url":"https://creativecommons.org/licenses/by/4.0/"},"manuscriptAbstract":"\u003cp\u003e\u003cb\u003ePurpose:\u003c/b\u003e\u003c/p\u003e \u003cp\u003eDigital crowdfunding tools appear to be a promising answer to address in a cost-effective manner areas of need in the Brazilian Unified Health System in which the provision of medicines and other services are either absent or insufficient.\u003c/p\u003e\u003cp\u003e\u003cb\u003eMethods:\u003c/b\u003e\u003c/p\u003e \u003cp\u003eConstruct validation study in technology innovation research to evaluate the applicability of an original digital solution in online healthcare crowdfunding called \u0026ldquo;Appadrinha\u0026rdquo;. The pilot project focused on the judicialization of Trikafta\u0026reg; for pediatric patients at a Cystic Fibrosis (CF) outpatient clinic in Porto Alegre. Our main objective was the success rate of fundraising campaigns. Inclusion criteria were patients aged 6 to 18 years with a positive genetic test with at least one allele of the mutation F508del. Exclusion criteria were family access to Trikafta\u0026reg; via private funds or insurance plans.\u003c/p\u003e\u003cp\u003e\u003cb\u003eResults:\u003c/b\u003e\u003c/p\u003e \u003cp\u003eThe inclusion rate was 57% (4/7). All 4 campaigns achieved full sponsorship of their individual budget of R\u003cspan\u003e$\u003c/span\u003e4,000.00 within 24 hours, with a success rate of 100% (4/4).\u003c/p\u003e\u003cp\u003e\u003cb\u003eConclusion:\u003c/b\u003e\u003c/p\u003e \u003cp\u003e Despite requiring refinement in the development of its functionality, Appadrinha appears to be a promising disruptive and innovative option in the crowdfunding market, working in a complementary way to the Unified Health System, through focusing on solving problems at the level of local communities, not only in the financial sphere, but also as a facilitator between families, health professionals and service providers, based on strategic and financially viable partnerships, thus promoting increased reliability in the healthcare crowdfunding scenario for socioeconomically vulnerable populations.\u003c/p\u003e","manuscriptTitle":"Applicability of a Crowdfunding Application in Obtaining Modulator Treatment for Children with Cystic Fibrosis","msid":"","msnumber":"","nonDraftVersions":[{"code":1,"date":"2025-04-10 08:14:18","doi":"10.21203/rs.3.rs-6123824/v1","editorialEvents":[{"type":"communityComments","content":0}],"status":"published","journal":{"display":true,"email":"[email protected]","identity":"researchsquare","isNatureJournal":false,"hasQc":true,"allowDirectSubmit":true,"externalIdentity":"","sideBox":"","snPcode":"","submissionUrl":"/submission","title":"Research Square","twitterHandle":"researchsquare","acdcEnabled":true,"dfaEnabled":false,"editorialSystem":"","reportingPortfolio":"","inReviewEnabled":false,"inReviewRevisionsEnabled":true}}],"origin":"","ownerIdentity":"d04bda19-4f01-4cda-b0a1-d0269223eba4","owner":[],"postedDate":"April 10th, 2025","published":true,"recentEditorialEvents":[],"rejectedJournal":[],"revision":"","amendment":"","status":"posted","subjectAreas":[],"tags":[],"updatedAt":"2025-05-23T07:38:43+00:00","versionOfRecord":[],"versionCreatedAt":"2025-04-10 08:14:18","video":"","vorDoi":"","vorDoiUrl":"","workflowStages":[]},"version":"v1","identity":"rs-6123824","journalConfig":"researchsquare"},"__N_SSP":true},"page":"/article/[identity]/[[...version]]","query":{"redirect":"/article/rs-6123824","identity":"rs-6123824","version":["v1"]},"buildId":"8U1c8b4HqxoKbykW_rLl7","isFallback":false,"isExperimentalCompile":false,"dynamicIds":[84888],"gssp":true,"scriptLoader":[]}