Yesterday, the FDA issued draft guidance on common issues related to rare disease drug development. It is another importance event in the shift of thinking about how drugs are developed for rare and devastating diseases. You can find the complete document here and the docket on the Federal Register will be here (as of 8/17/15). We will also post a link to where you can submit comments on Regulations.gov when it is posted. [pullquote]It’s altogether too easy for sponsors to gloss over the references to patient perspective and decide that actually asking for patient input on those issues is unnecessary, too biased, or insufficiently scientific.[/pullquote]
But I pose the question: Does it go far enough? It is a compendium, in many ways, of the issues in drug development generally and how they might apply in rare disease, but there remains a lot of wiggle room that will leave rare advocates wanting more, such as the discount of historical controls and reiteration of the import of control groups. And while there are allusions to patient inclusion in the process (“An understanding of which aspects of the disease are meaningful to the patient and might also be affected by the drug’s activity.”), it’s altogether too easy for sponsors to gloss over the references to patient perspective and decide that actually asking for patient input on those issues is unnecessary, too biased, or insufficiently scientific.
Given the large influx of companies into the rare disease space in recent years, both small biotechs and big pharma, this may serve as guidance for the newly initiated. But for the seasoned, it seems unlikely to give them enough assurance to significantly alter their current course of drug development. Rather, let’s hope there are some golden nuggets that still cause them to think and take new steps toward better programs, such as the discussion about endpoints and assessments.
Some salient points from the guidance:
I encourage you to submit comments to the draft guidance, offering suggestions for additions, clarifications, or improvements [link to be posted 8/17/15]. We have submitted numerous and extensive comments on issues related to rare disease drug development. If you’re not sure what might be appropriate thoughts or suggestions for such comments, feel free to read our past docket submissions below. We will also be posting our comments to this draft guidance once submitted.