Drugs

Support for Rare Disease Education Materials

David McFarland

— 1 min read
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The National Organization for Rare Disorders (NORD) says that as FDA considers what rare disease resources to share and in what format, it should tailor materials towards both developers and patients, so patients can be educated about productive ways to engage in the product development process. The NORD comment came in response to an agency opportunity for public comment on CDER rare disease LEADER 3D educational materials (Learning and Education to Advance and Empower Rare Disease Drug Developers).

NORD also says that it appreciates that the Accelerating Rare disease Cures (ARC) program Web site shows agency regulatory flexibility. It asks that, as biotechnology allows for more tailored therapies, gene therapies, and other emerging forms of treatment, appropriate technologies are represented in the materials.

In its comment, UCB says the LEADER 3D materials “make complex regulatory issues more accessible to drug developers such as UCB….” It recommends that the agency include these additional topics of interest to enhance the clarity, relevance, and usefulness of the educational materials:

  • case studies of recently approved products, including unique cases warranting additional regulatory flexibility;
  • programs demonstrating successful partnerships between academia or patient advocacy groups and industry;
  • the use of non-traditional controls;
  • expectations for data packages supporting nonclinical confirmatory evidence;
  • the collection and use of real-world evidence to support regulatory decision-making;
  • statistical approaches to overcoming potential bias with open-label or externally controlled clinical trial designs;
  • non-traditional approaches to dose selection;
  • conducting human factor trials for rare diseases where there may not be sufficient participants of patients/caregivers; and
  • the successful use of accelerated regulatory pathways.

The Lymphoma Research Foundation says it supports FDA efforts to develop materials on knowledge gaps and priority topics in rare disease drug development and related regulatory considerations. It recommends the development of patient-facing materials on these topics:

  • an explanation of the plausible mechanism framework and a comparison of that framework to other regulatory pathways;
  • an explanation of natural history controls;
  • explanation/discussion of clinical trial design issues in rare disease research and development; and
  • listening sessions with patients and patient advocates to inform development of materials on communication regarding risks and benefits of novel therapeutics, including cell and gene therapies.

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