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

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.