As part of the launch of the US Food and Drug Administration (FDA)’s Rare Disease Cures Accelerator, the agency asked for stakeholder input on how FDA and other agencies can achieve a more cooperative approach in supporting the drug development pipeline for rare diseases. By the 30 July deadline, over 60 comments had been received from individuals and family members affected by rare diseases, from pharmaceutical companies and trade associations, and from other federal agencies and regulators. (RELATED: FDA seeks input on rare disease clinical trials network, Regulatory Focus 29 May 2020)
The Accelerator will build out technologic support such as common data platforms to support clinical trials in rare disease populations and will work to create standardized clinical outcome assessments and endpoints that are pertinent to rare diseases.
Among the government organizations providing commentary was the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) houses the Office of Rare Diseases Research (ORDR). In her comments, ORDR director Anne Pariser, MD, pointed to NCATS’ own Trial Innovation Network, which addresses clinical trial roadblocks through operational innovation. Examples, she said, are the network’s unified institutional review board system and master contracting agreements.
Pariser said that immediate needs are for better foundational science and more clinical trial readiness, among other hurdles generated by the rarity of diseases and the often-lengthy path to diagnosis for patients with these conditions. She recommended developing a toolkit with broad accessibility that would include a way to obtain “iterative regulatory device” for rare disease stakeholders, who could benefit from this early and accessible advice “to facilitate the development of good quality programs likely to support regulatory submissions.”
Danielle Friend, PhD, senior director of science and regulatory affairs for the Biotechnology Innovation Organization (BIO) concurred in her comments that the clinical trial network infrastructure that FDA develops should support “the collection of high-quality data that are endorsed by the Agency for regulatory decision-making.” BIO also seeks guidance from FDA to develop quality criteria for natural history registries, but also wishes that the agency be transparent and realistic about the costs associated with data acquisition and analysis.
Friend noted a number of existing challenges, including heterogeneity in rare diseases, lack of harmonization among global regulators in rare disease regulatory policy, and current inconsistencies in clinical trial network operations. A thoughtfully planned new rare diseases clinical trial network could address these challenges, she said. She also asked that the new network consider the difficulty in recruiting patients and give consideration to combining rare diseases into a single trial design when appropriate, and to single-arm trial designs in some instances.
The National Organization for Rare Diseases (NORD), a partner in FDA’s development of the Rare Diseases Cures Accelerator, noted that the program has “the potential to revolutionize rare disease drug development and review.” NORD’s vice president for policy and regulatory affairs, Rachel Sher, JD, also stressed the need for trial designs that overcome some of the rare disease-specific challenges called out by BIO. Increased collaboration and a focus on increasing the speed and success of clinical trials can have the effect of “breaking down the silos of activity currently taking place in rare disease research,” said Sher.
The NORD response lauded FDA for thinking globally, but advocated for taking small steps initially to build a robust US infrastructure: “We believe that achieving global collaboration on a clinical trials network would likely be more attainable once we take the first step to implement the network and infrastructure on a national scale.”
The biopharmaceutical trade association Pharmaceutical Research and Manufacturers of America (PhRMA) also submitted comments that echoed the concerns of other organizations about recruitment and data collection challenges, data management, and the need for guidance on the adequacy and quality of data needed to support regulatory decision-making.
PhRMA’s senior director for science and regulatory advocacy Maria Apostolaros, JD, PharmD, added that “Consistent and robust funding will be needed to attract representative experts to the team and maintain operational delivery.” She called for a core team with the “right mix” of public-private expertise.
Within NCATS’ ORDR, the Rare Diseases Clinical Research Network (RDCRN) supports multiple NIH Institutes and Centers in researching nearly 200 rare diseases. This office proposed a Clinical Trials Resource Center with five core functions, including a regulatory core staffed by individuals with global regulatory expertise in rare and ultra-rare diseases. The particular focus of the regulatory core would be to inform clinical trial strategies that minimize the number of trials and patients necessary in a drug development program for the rare disease population.
Several global bodies left commentary, including The European network of pediatric research at the European Medicines Agency, The European Confederation of Pharmaceutical Entrepreneurs, Rare Voices Australia and Canada’s Maternal Infant Child Youth Research Network.
Many patient advocacy associations and disease-specific organizations also weighed in; the Cystic Fibrosis Foundation, the Muscular Dystrophy Association, the American Epilepsy Society, and the Endocrine Society were among those who provided comments. Academic research centers such as the Cleveland Clinic and the University of Pennsylvania posted comments, as did pharmaceutical companies including Boehringer Ingelheim, Otsuka and Retrophin.
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