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  1. From Our Perspective

FDA Opens Doors for More Treatments for Rare Diseases through the New START Pilot Program

By: Patrizia Cavazzoni, M.D., Director, Center for Drug Evaluation and Research and Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research

Patrizia Cavazzoni, M.D.
CDER Director, Patrizia Cavazzoni, M.D.

We are at a turning point in understanding the science that is opening doors to rare disease treatments that were unthinkable only a few years ago, such as gene therapies and drugs that work on gene expression. Despite this progress, there are still many rare diseases that lack treatment options, and we need to do more to alleviate the suffering of patients and their families. For this reason, FDA is taking steps to help accelerate the development of novel drugs and biologics through the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.

FDA’s Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) initiated the START Pilot Program with hopes that the insight gained through this pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies for rare diseases and help sponsors generate high-quality, actionable data to support future new drug or biologics license applications. Selected participants will have access to frequent advice from FDA staff to address product-specific development issues, including clinical study design, choice of control group, and fine-tuning the choice of patient population. These increased interactions include addressing early development issues that may otherwise delay or prevent a promising novel product from progressing to the pivotal clinical trial stage and ensuring a clear understanding of the information and data needed to facilitate product development. 

CBER Director, Peter Marks, M.D., Ph.D.
CBER Director, Peter Marks, M.D., Ph.D.

Participants in the pilot program will also benefit from access to several existing programs and initiatives within CBER or CDER:

  • Advancing Real-World Evidence (RWE) Program- The program seeks to improve the quality and acceptability of RWE-based approaches in support of new labeling claims.
  • Complex Innovative Trial Design (CID)- The CID Program is designed to facilitate and advance the use of complex adaptive, Bayesian, and other novel clinical trial designs with emphasis on late-stage drug development.
  • CDER’s Translational Science Team (TST)- The TST is a multidisciplinary team with expertise in precision medicine, biomarkers, modeling, pharmacology/toxicology, and rare diseases.
  • Rare Disease Endpoint Advancement (RDEA) Pilot- The RDEA Pilot allows sponsors to collaborate with FDA throughout the efficacy endpoint development process, promote innovation and evolving science by sharing lessons learned on novel endpoint development.

Pilot Program Participants

Although there were a number of excellent and qualified applicants, there was only availability for a limited number of programs within the START Pilot Program. In making these selections, we considered the strength of the development programs, and the unmet medical need represented by the indicated conditions. To help aid our evaluation of the program, we also attempted to include a spectrum of entities in the pilot ranging from companies initially established to produce a single product to established biotechnology companies.

CDER Participants

CDER considered development programs for novel drugs intended to treat rare neurodegenerative conditions (including those of rare genetic metabolic etiology). CDER has selected the following sponsors to participate in the pilot:

Sponsor Product Disease of Interest
Larimar Therapeutics Nomlabofusp Friedreich's ataxia
Calico Life Sciences ABBV-CLS-7262 Vanishing White Matter disease
Denali Therapeutics DNL126 Mucopolysaccharidosis Type IIIA (Sanfilippo syndrome)

CBER Participants

CBER considered development programs for gene or cellular therapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first 10 years of life. CBER has selected the following sponsors to participate in the pilot:

Sponsor Product Disease of Interest
Grace Science, LLC GS-100 (AAV0-rhNGLY1) NGLY1 Deficiency
Moderna TX mRNA-3705 Isolated methylmalonic acidemia due to complete or partial methylmalonyl-coenzyme A mutase deficiency
Myrtelle, Inc. rAAV-Olig001-ASPA Canavan Disease
Neurogene, Inc. NGN-401 Rett Syndrome

CBER and CDER look forward to actively working together to fulfill our shared vision of bringing better treatment options to patients and their families. Through the START Pilot Program, the opportunity for increased engagement with the selected sponsors should facilitate more rapid achievement of regulatory milestones, if supported by the data and the science. For questions about the START Pilot Program, please contact CDER’s START Pilot Program mailbox (CDER.STARTProgram@fda.hhs.gov) or CBER’s Office of Communication, Outreach and Development (ocod@fda.hhs.gov). For additional information about individual sponsors and products, please contact the sponsors directly.

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