#patients are playing an increasingly central role in #drugdevelopment, and doubly so for #rarediseases. Yet, navigating FDA can be a challenge for #patients and #patientadvocacy groups, as well as #academic researchers, small #biotech #startups, and other organizations with limited #drugdevelopment experience. Scientific and regulatory differences, as well as organizational inconsistencies, between the #CDER and #CBER are exacerbating these challenges, in particular for innovative novel technologies like #cell and #genetherapies and #antisenseoligonucleotides #ASOs.
The new #FDA hub, announced today, promises to help address (some of) these issues, offering a single point-of-contact and 'no wrong doors approach' for anyone engaged in #raredisease drug development.
The renewed focus on novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods specific to #rarediseases is encouraging - after all, many of the common statistical tools reach their limits when faced with the small and heterogeneous patient populations common in #raredisease trials, #realworlddata and #realworldevidence holds unique challenges (and opportunities!) for #rarediseases given the dearth of disease-specific #ICD-10 codes, and #innovative #trialdesigns that minimize how many patients are needed for #control arms (and for how long they have to be on the #control rather than an open-label extension) make #raredisease #clinicaltrials more feasible and #patientcentric. In many cases, endpoint, biomarker, and assay development has to be pre-competitive almost by necessity given the limited patients available in many rare diseases and the associated economics and timelines.
The new #rarediseasehub at #FDA certainly reinforces the agency's commitment to our space, where more than 95% of the 10,000+ rare diseases have *NO* FDA-approved treatment options. Read the full announcement here:
https://lnkd.in/eyYcmiCV
Certainly a great place to work with both highly skilled colleagues, a great company culture and a very meaningful purpose 🙏 We are proud to have you in our community!