Below are some organizational perspectives.
National Health Council Announcement
The National Health Council applauds the FDA for agreeing to include in its recommendations three proposals championed by patients: the development of an objective, qualitative benefit-risk framework that includes robust patient input; the advancement of new therapies through the expanded use of biomarkers and patient-reported outcomes in clinical trials; and the creation of regulatory policy, procedures, and guidance to encourage the development of treatments for rare diseases.
All three of these components are necessary for advancing access to desperately needed treatments and cures. To exclude any one of these elements would be to offer a reauthorization package that would not meet the needs of patients – particularly those with few or no treatment options.
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:Comments From A Pharmaceutical Industry Representative•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.
PDUFA is only part of larger FDA reforms needed to improve public health.
It's not a done deal. There are aspects that are a matter of life and death for people with ALS who are currently without an effective treatment and without a biomarker. It's important that we all pay attention as this proposal moves forward. Mission has not been accomplished.
Also, for ALS, a disease without biomarkers, are we going to slow down trials that might be mixing biomarker theories with efficacy theories? Sure, it would be great to use proven biomarkers in efficacy trials, but ...