Three years removed from Operation Warp Speed, which delivered a bevy of safe, effective COVID-19 vaccines, the Food and Drug Administration is taking those processes to put a greater spotlight on rare diseases.

Through the recently launched pilot program, the agency is helping guide industry players in accelerating research and development efforts to address unmet needs in the space.

The FDA program is dubbed START — or Support for clinical Trials Advancing Rare disease Therapeutics — and targets some of the challenges associated with rare disease development.

Partially inspired by Operation Warp Speed, START is designed to leverage those pandemic-era learnings for a space that has received renewed attention from drugmakers in recent years.

In particular, START will allow participating companies, or sponsors, to get guidance and advice from the FDA on addressing clinical development issues, from clinical study design to patient population.

The ultimate goal, according to Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, is to move new therapies for rare diseases through the regulatory process quicker by working more closely with manufacturers.

“We hope the insight gained from this pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies with rare disease indications and help sponsors generate high-quality, compelling data to support a future marketing application,” Marks said in a statement.

Gaining regulatory approval in the rare disease space can be challenging for pharma companies, which often face difficulties in designing clinical trials and investigating therapies for diseases that little is known about.

In the last year, several rare disease products were hit with surprise rejections, including Ipsen’s palovarotene for fibrodysplasia ossificans progressiva (FOP) in December 2022. Palovarotene ended up being approved eight months later after Ipsen provided more data.

Recently, the FDA also rejected Alnylam’s drug patisiran for the expanded use label of cardiomyopathy of hereditary transthyretin-mediated amyloidosis.

Pharma-fed partnership

Marks noted that these are complex products and said the agency recognizes the importance of sponsor communication to facilitate development of products for patients with unmet medical needs.

Eligible companies must have products that are seeking to treat rare neurodegenerative diseases and are currently in clinical trials under an Investigational New Drug (IND) application.

Companies seeking to participate can submit applications to the START program starting on January 2, 2024 through March 1.

In order to be eligible, companies must also be developing products that are a gene or cellular therapy that addresses an unmet medical need in a rare disease which is likely to lead to significant disability or death within the first decade of life. 

In particular, the FDA is centering the program around rare neurodegenerative conditions, including genetic metabolic diseases.

Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said the FDA is committed to helping sponsors achieve regulatory milestones, while ensuring the safety, effectiveness and quality of these products.

This is the FDA’s latest push in seeking to accelerate developing rare disease products. It also published a Request for Information, asking stakeholders to send in feedback about some of the biggest challenges and knowledge gaps around cellular and gene therapies for rare diseases.