Rare Diseases: BIO Comments on Complex Issues for Rare Diseases

BIO appreciates FDA’s efforts in convening its two day public workshop on January 6-7th entitled “Complex Issues in Developing Drug and Biological Products for Rare Diseases” as required by the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) and opening a docket for public comment. BIO was pleased to be invited to participate in Day One “Complex Issues in Rare Disease Drug Development” to provide an industry perspective of rare disease development.
 
Rare diseases represent an area of substantial unmet medical need: many are serious or life-threatening, and for the vast majority there are no approved therapies available for their treatment. Product development programs face unique challenges in terms of small patient populations and often a high degree of variability and heterogeneity within these small populations. As such, there is no one-size-fits-all approach to rare disease drug development and, likewise, no one-size-fits-all regulatory approach. Thus, thinking creatively and acting flexibly are crucial for both developers and regulators to ensure patients get safe and effective treatments as quickly as possible. Furthermore, patients can play a critical role in the development of products for rare diseases and should be engaged throughout the drug discovery process. It is particularly critical for rare disease therapies that the FDA drug review process be grounded in a careful evaluation and balance of both benefits and risks made in the broader context of disease rarity and severity, patient perspectives, and the body of available scientific evidence.
 
 
 
BIO believes FDA has taken some important first steps regarding its regulation of drugs for rare diseases and in implementing its FDASIA requirements. Below, we provide several questions and requests for FDA as the agency continues to move forward in this area and look forward to continuing to engage with FDA and other stakeholders.