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Pediatrics Rare Disease: BIO Comment Letter on FDA Draft Guidance Pediatric Rare Diseases-A Collaborative Approach for Drug Development Using Gaucher Disease as a Model

Re: Docket No. FDA-2017-N-6476: Pediatric Rare Diseases-A Collaborative Approach for Drug Development Using Gaucher Disease as a Model; Draft Guidance for Industry; Availability

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) appreciates the opportunity to provide comments to the Food and Drug Administration’s (FDA or Agency) Draft Guidance for Industry “Pediatric Rare Diseases-A Collaborative Approach for Drug Development Using Gaucher Disease as a Model” (Draft Guidance).

BIO is the world’s largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place.

BIO appreciates the FDA’s intended goal of the Draft Guidance, which is to reduce the total number of children needed for enrollment in clinical studies. We also applaud the Agency’s willingness to accept innovative trial designs to limit the number of patients in the control arm and improve trial efficiency. However, BIO has several concerns regarding the Draft Guidance, particularly in reference to the FDA’s suggestion for the use of multi-arm, multi-company clinical studies, which have inherent limitations and challenges. BIO has identified the following concerns with respect to this Draft Guidance which are addressed in more detail below:

  1. The Draft Guidance lacks detail regarding applicability across a broad range of rare diseases.
  1. The Draft Guidance outlines the use of multi-arm, multi-company clinical studies, but does not provide procedural information regarding how challenges of this approach will be addressed.
  1. The Draft Guidance fails to address other possible methodologies that would help reduce the total number of children required for enrollment in clinical trials.

In addition to the concerns mentioned above, BIO has also provided additional comments and line edits beginning on page six of this letter.