MDA Urges Legislators to Speed Up Approval of Rare Disease Drugs

Update (July 10, 2012) —Reauthorization of the Prescription Drug User Fee Act (PDUFA) was signed by President Obama on July 9, 2012, complete with a provision that creates an accelerated approval pathway for drugs for rare diseases.

“MDA is gratified that Congress recognizes the importance of advancing therapies for rare diseases and developing new approaches to drug approval. The Association also is pleased that the FDA now will have the benefit of experts in rare diseases at the table, representing the unique characteristics and features of rare neuromuscular diseases,” said Annie Kennedy, MDA senior vice president of advocacy.

MDA Advocates were part of the push to pass the new provision. PDUFA reauthorization received strong bipartisan support in both the U.S. House and Senate.

The drug development process for rare diseases needs all the help it can get. That’s why MDA’s advocacy program is marshaling support for proposed new language to be included in the Prescription Drug User Fee Act (PDUFA) reauthorization that would “fast track” the approval of new drugs aimed at rare diseases.

“I implore all members of the MDA community to take a moment to contact their legislators and urge them to approve the inclusion of the critical accelerated approval pathway language in this important legislation,” said Annie Kennedy, MDA senior vice president of advocacy. “It’s one more way we can help advance the discovery of drug therapies for our diseases.”

First passed in 1992, PDUFA authorizes the U.S. Food and Drug Administration (FDA) to collect fees from companies that produce certain human drug and biological products. These user fees have helped speed up the FDA approval process. PDUFA was revised and renewed in 1997, 2002 and 2007, and is up for renewal again this year; the accelerated approval pathway for rare diseases is part of the proposed revision.

Accelerated approval —with conditions

The accelerated approval provision requires the development of innovative “surrogate and clinical endpoints” that enable rare disease drugs to more quickly demonstrate their effectiveness to the FDA.

Surrogateendpoints must be “reasonably likely to predict clinical benefit” and clinicalendpoints must be able to be measured “earlier than irreversible morbidity or mortality.”  Such endpoints may include “epidemiological, pathophysiological, therapeutic or other evidence developed using biomarkers … or other scientific methods or tools.”

The proposed new language requires that these endpoints be developed by experts in the specific disease area, in order to ensure that clinical trials are designed as meaningfully and as effectively as possible.

The proposed new language acknowledges that an accelerated approval process could sometimes result in an approved drug that doesn’t actually provide the benefits it was expected to provide. Therefore, the language stipulates that the FDA may require the drug developer to:

  • conduct post-approval studies to verify and describe the predicted effect on irreversible morbidity or mortality or other clinical benefit; and/or
  • submit copies of all promotional materials related to the product.

The language also states that accelerated approval can be withdrawn if the sponsor fails to conduct any required post-approval studies; or if the study shows that the drug either doesn’t work as expected or isn’t safe; or if the drug sponsor disseminates false or misleading promotional materials about the product.

For more on the timeline and requirements for implementing an accelerated approval pathway for rare diseases, see the FDA document PDUFA Reauthorization Performance Goals and Procedures and go to part E, “Advancing Development of Drugs for Rare Diseases.”

Take 5 to support new PDUFA language

MDA’s “Take 5” initiative encourages members of the neuromuscular community to “take five minutes” to advocate for this important legislation. The MDA advocacy page about the proposed PDUFA language makes it extremely easy to send letters and emails to elected officials spelling out exactly why this legislation is important to our community.

“It is vital that we reach out to our elected officials to provide them with the support that they need so they can fight on our behalf,” Kennedy added. “Contact your federal elected officials today and let them know that rare diseases need a fast-track process, and that enhancing regulatory science is vital to your family.”

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