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Proposed Programs Could Speed Up Drug Approval for Rare Diseases

New pilot programs to expedite the approval process for drugs that treat rare and currently untreatable diseases could be on the horizon.  

The proposed agreement between the U.S. Food and Drug Administration and pharmaceutical manufacturers would establish three different programs meant to advance the discovery of new treatments for rare diseases.  

Many patients with rare conditions have no FDA approved treatments available to them, so the proposed programs have the potential to address a massive unmet need. 

Real-Time Review 

One program – the Split Real Time Application Review Pilot Program – would allow drug companies to submit clinical trial results on certain rare disease treatments in two phases – as opposed to waiting for all the data before submitting a complete “final” application. This would give the FDA a jumpstart on reviewing certain data.  

The concept is based on a similar program, called the Real-Time Oncology Review Pilot Program, the federal agency already uses for its review of certain cancer drugs. 

Improved Testing Goals and Benchmarks 

A second pilot program, called the Rare Disease Endpoint Advancement Pilot Program, would allow drug companies and the FDA to collaborate on defining research endpoints for new drugs. 

Patients suffering from incurable conditions don’t have unlimited time to wait for promising treatments to make their way through the approval process. The second program allows drug companies the flexibility to tailor clinical trials’ goals and benchmarks to the urgent needs of those patients. 

Using Real-World Evidence 

The third new program would allow the FDA to consider additional real-world evidence in the drug approval process. This would allow regulators – and the American people – to benefit from test results produced outside, and often before, traditional drug trials. These include clinical trials with experimental designs or observational studies of patients’ progress with a given medication.  

Real-world evidence would not replace traditional drug trials but could provide the FDA enough data to safely expedite certain approvals. 

Collaboration, Not Tug-of-War 

Drug companies and federal regulators have shown how collaboration in the public interest can do better than the political tug-of-war that so often defines policymaking in Washington. 

Now, it’s up to Congress to ratify the proposal so the programs can take effect.  

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