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Proposed PDUFA Agreement: the Right Prescription for People with Lou Gehrig's Disease
The ALS Association applauds the proposed agreement for the reauthorization of the Prescription Drug User Fee
Act (PDUFA), which includes provisions that would help to encourage and expedite the approval of new treatments
for rare diseases like ALS, also known as Lou Gehrig’s Disease. The proposed agreement was released by the
Food and Drug Administration (FDA) on September 1.
PDUFA authorizes the FDA to collect fees from companies that develop drugs and biologic products. These fees
help the agency cover the costs of drug reviews and currently account for about half of the agency’s drug review
costs. The law originally was established in 1992 and has been reauthorized every five years since that time. The
current law expires next year unless it is reauthorized by September 30, 2012.
“The reauthorization of PDUFA is critical to discovering a treatment and cure for ALS,” said Jane Gilbert, President
and CEO of The ALS Association. “There is no effective treatment for ALS and it is fatal in an average of just two
to five years. By providing the agency with the resources it needs to review new treatments and ensure their efficacy
and safety, PDUFA helps to make sure that funding limitations do not block innovative treatments from reaching
the patients who so urgently need them.”
“The additional resources and the establishment of drug review timeframes are important elements of PDUFA,”
said Steve Gibson, The ALS Association’s Chief Public Policy Officer. “This proposal is particularly significant because
it was developed with the input of the patient community in partnership with the FDA and industry. And it is
significant because it includes key provisions that promote the development of new treatments and recognize that
the practice of medicine is not a one-size-fits-all proposition, especially when it comes to a disease like Lou Gehrig’s
Disease.”
Since July 2010, The ALS Association participated in monthly meetings with FDA officials to bring the perspective
of people with ALS to the discussion on the reauthorization of PDUFA. Three key provisions advocated by The
Association were included in the proposed agreement. They include:
Developing Treatments for Rare Diseases: Directs FDA to issue regulatory policies and guidance that encourage
the development of drugs to treat rare diseases and to include patients as active participants in this
process.
Assessing Risk-Benefit: Instructs the agency to develop an objective and qualitative framework for assessing
risk and benefit during the drug review process and to ensure patient involvement. The provision takes into
account the fact that people with ALS who have no treatment options and who will die in just two to five years
have a much different view of risk and benefit than does someone with a treatable or chronic condition.
Utilizing Biomarkers in Clinical Trials: The proposal encourages greater use of biomarkers and other markers,
or “sign-posts,” to help determine risk and benefit when reviewing new drugs. Biomarkers have the potential
to significantly accelerate access to new drugs by helping to determine much sooner whether a treatment
works and in which patients.
“Congress has authorized PDUFA four times beginning in 1992. But perhaps no other version of the law is as important
to people with ALS as PDUFA V,” said Gibson. “Right now people with ALS have no way to defend themselves
in the fight against the disease. It will take their lives. But PDUFA V can help them fight back. It promotes
new treatments. It speeds access. And it makes sense. We urge Congress to swiftly adopt the PDUFA V proposal
in 2012.”
The proposed agreement released by the FDA is available on the FDA website here:
http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf.
The ALS Association is the only non-profit organization fighting Lou Gehrig’s Disease on every front. By leading
the way in global research, providing assistance for people with ALS through a nationwide network of chapters,
coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships,
The Association builds hope and enhances quality of life while aggressively searching for new treatments and a
cure.