By Amy Dockser Marcus

A new paper offers a possible road map for getting more drugs approved to treat rare diseases. One key recommendation: improving access to the FDA’s accelerated approval pathway, originally developed to quickly get treatments to patients with life-threatening diseases.

The paper, written by Brigitta Miyamoto and Emil Kakkis of the Kakkis EveryLife Foundation and published today in the Orphanet Journal of Rare Diseases, says the FDA should offer specific guidelines on how companies seeking to develop drugs for rare diseases can better utilize accelerated approval.

Kakkis helped develop and guide the approval process for three treatments for rare diseases at BioMarin Pharmaceutical, where he served as chief medical officer. He’s now CEO and president of Ultragenyx Pharmaceutical, which recently announced $45 million in financing to support the development of rare-disease therapies. (So he’d also benefit from anything that helped those drugs get to market more quickly.)

The FDA created the accelerated approval process in 1992 in the wake of advocacy by HIV/AIDS patients angry at the slow pace of drug development. It allows drug companies to ask FDA to approve a new therapy using so-called surrogate endpoints — blood tests, urine tests, or other biomarkers that likely predict a patient will benefit from a drug but aren’t on their own a measure of improved health. If the drug is approved, the company has to agree to keep testing to make sure that the results hold up.

(And sometimes they don’t. Just last week an FDA appeals panel recommended the revocation of accelerated approval for Avastin for use in breast-cancer patients after studies showed the drug provided minimal benefits.)

In less-common diseases, drug development is difficult all around. There is often very little understanding of the disease, its natural history and how to treat it, and scant data on managing or treating patients. That makes it very hard for companies to know what information the FDA will need to decide that a surrogate endpoint meets its standard of being “reasonably likely” to predict a benefit in a patient.

Anne Pariser, associate director for rare diseases in the Office of New Drugs at the FDA, tells the Health Blog that it’s crucial to be sure surrogate endpoints are valid indicators of the drug’s efficacy; some have failed to predict outcomes. So “you need evidence to support” any surrogate endpoints. She also notes that drug development fails for many reasons, so easier access to accelerated approval is no panacea.

Kakkis says his foundation plans to hold a workshop in the fall to jump-start a discussion on getting FDA to write guidelines on just how much evidence a company needs to meet the “reasonably likely” standard, and whether the criteria should be different for at least some rare diseases.

In the paper, Kakkis and his co-author identified 15 rare diseases where a treatment reversed the disease in animal models but still stalled in drug development. The reasons why varied, he said. Some were looked at by companies but deemed too expensive to successfully develop, others sat in academia without attracting investment. A few languished for years but are being considered again. All of them, he adds, could benefit by clearing the path to accelerated approval.

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