Guest Column | December 18, 2023

Understanding FDA's Rare Disease Therapeutics Pilot Program

By Salma Saiger, MD, physician, SMS Clinical Research

FDA Approved GettyImages-1215516118

The FDA's commitment to advancing medical research and therapeutic solutions for rare diseases is exemplified through its innovative Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. Launched with the intent to expedite the development of groundbreaking drugs and biological products targeting rare diseases, this initiative opens new avenues for selected sponsors to engage in enhanced communication with FDA staff, addressing specific programmatic needs in a collaborative manner.

Program Overview And Key Dates

The START Pilot Program is set to accept applications from Jan. 2, 2024, to March 1, 2024. Designed to be inclusive, the eligibility criteria encompass both CBER and CDER regulated products.

For CBER, the focus is on cellular or gene therapy products addressing an unmet medical need for a rare or serious condition, particularly those with the potential for significant disability or death within the first decade of life.

CDER-specific eligibility targets treatments for rare neurodegenerative conditions. General eligibility criteria include an active IND in eCTD format and alignment between chemistry, manufacturing, and controls (CMC) and clinical development.

Exploring The Benefits And Drawbacks


1. Enhanced Communication: One of the standout advantages of the START Program is the intensified communication sponsors receive from FDA staff. This heightened interaction allows sponsors to seek more frequent advice, facilitating a dynamic exchange of insights throughout the drug development journey.

2. Faster Development: Sponsors participating in the program may experience accelerated progress toward pivotal clinical trials or pre-BLA/pre-NDA meetings. The potential for a more streamlined development process translates to quicker advancements in the journey from research to patient access.

3. Improved Data Quality: The increased communication opportunities can contribute to the generation of high-quality data, a crucial factor in regulatory submissions. The iterative dialogue between sponsors and FDA staff enhances the robustness and reliability of the clinical data generated during the development process.

4. Increased Efficiency: The program aims to streamline the development process, offering a pathway to increased efficiency. This not only saves valuable time but also optimizes the allocation of resources, contributing to a more focused and effective development strategy.


1. Limited Availability: A potential drawback is the limited number of sponsors selected for the pilot program. While the program's exclusivity ensures a concentrated effort, the downside is that only a select few can benefit from this unique opportunity.

2. Increased Workload: Sponsors opting for the START program must be prepared to dedicate additional resources to communication with FDA staff. While the enhanced interaction is beneficial, it also imposes an added workload on sponsors.

3. Uncertainty About Long-Term Impact: As with any pilot program, there is uncertainty about the long-term impact on drug development. While the immediate benefits are apparent, the lasting influence on the rare disease drug development landscape remains to be seen.

Expert Insights And Program Comparison

In the realm of incentivized drug development, the START Pilot Program finds itself akin to existing initiatives such as the Breakthrough Therapy designation and the Orphan Drug designation.

These programs offer incentives to encourage the development of drugs for rare diseases. However, the START program distinguishes itself by providing ongoing communication and guidance, creating a dynamic partnership throughout the development process.

Areas For Improvement:

1. Expanding Program Scope: To maximize its impact, consideration could be given to expanding the program's scope, allowing more sponsors to participate. This adjustment could broaden the reach of the program, benefiting a more extensive range of rare disease patients.

2. Streamlining Communication: Clear guidelines and expectations for communication between sponsors and FDA staff could enhance the efficiency of interactions. A well-defined framework would ensure that the communication process is transparent, structured, and conducive to productive collaboration.

3. Providing Long-Term Support: While the pilot program offers valuable insights, there is room for exploring avenues of continued guidance beyond the initial stages. A more prolonged commitment to supporting sponsors could contribute to sustained success in rare disease product development.

The Potential Impact

The FDA's START Pilot Program emerges as a pivotal initiative with the potential to significantly accelerate the development of new treatments for rare diseases. By providing sponsors with unprecedented access to FDA expertise, this program stands as a beacon of collaboration, fostering an environment where innovation and regulatory guidance converge. The ultimate beneficiaries are the patients grappling with these devastating conditions, as the program endeavors to bring about positive transformation in the landscape of rare disease therapeutics.

About The Author:

Salma Saiger, MD, is a board-certified internal medicine physician at SMS Clinical Research, who specializes in preventing, diagnosing, and treating adult diseases without surgery. She graduated from Dow Medical College in Pakistan and completed her internship and residency at Overlook Hospital in New Jersey. After working at Yavapai County Community Health Center in Arizona, she relocated to Texas in 2008. Saiger has served as vice chief of staff and medical director at East Texas First Physicians Clinic before establishing her private practice in Mesquite, TX, in 2009.