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Rare Diseases: BIO Comments on Rare Disease Clinical Trial Network Request for Information

July 31, 2020

July 31, 2020

Re: Docket No. FDA–2020-N-0837: Raare Disease Clinical Trial Network; Request for Information.

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments on the Request for Information on the Global Rare Disease Clinical Trial Network.

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 diseases, and to prevent diseases in the first place.

BIO appreciates FDA’s efforts to address challenges associated with developing therapies for the thousands of rare diseases that do not yet have treatments. We further appreciate FDA efforts to seek feedback from the stakeholder community on the third component of the Rare Disease Accelerator focused on the development of a global rare disease clinical trial network. However, given the number of identified rare diseases, we have concerns and questions regarding the potential scope of a single rare disease clinical trial network. As discussed in greater detail below, it may be more appropriate to consider the development of more than one rare disease clinical trial network. It will also be important for FDA to clearly outline the scope and mission of the clinical trial network(s).

It is also important to note that rare disease drug developers still face key challenges unrelated to the availability of robust, high quality global trial networks. These challenges include limited, well-matched natural history data, lack of natural history data for less-prevalent phenotypes within a given disease,  limited established or relevant endpoints and surrogate biomarkers that can be used to support drug development and regulatory decision-making, and the inability to use innovative clinical trial designs to address the logistical challenges associated with limited patient populations. To this end, in addition to the efforts focused on the global rare disease clinical trial network(s), BIO encourages the Agency to continue to support rare disease drug development through the development of disease-specific guidance, finalization of draft rare disease guidance,[1] and public discourse regarding the key challenges mentioned above.

BIO encourages FDA to consider the following immediate and long-term objectives/milestones for the rare disease clinical trial network(s):

  1. Establishment of a steering committee for the clinical trial network(s), including participation from patients, caregivers, patient organizations, clinicians, drug developers, and other health authorities;
  2. Engagement of stakeholders through a public meeting and public comment process;
  3. Analysis of existing clinical trial network(s) and development of the scope and mission for the clinical trial network(s), including potential grouping or prioritization of rare diseases to be targeted through the new network(s);
  4. Development of standards for the clinical trial network(s), including common data elements, mechanisms and platforms for data/information sharing, structured institutional review board, data collection including collection of natural history data, identification of potential third parties to manage and implement the network(s); and
  5. Continued engagement of stakeholders on lessons learned and opportunities for improving the clinical trial network(s).

[1] Rare Diseases: Common Issues in Drug Development and Rare Diseases: Natural History Studies for Drug Development.

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BIO Comments on Rare Disease Clinical Trial Networks RFI
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