PPMD Community

PPMD is thrilled to announce that on May 4 the BENEFIT Act was introduced in the Senate by longtime Duchenne community champions, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN).

 

Over the past five years, Congress has made considerable progress in driving forward policies and procedures to ensure the patient perspective is considered by Food and Drug Administration (FDA) reviewers evaluating candidate drugs and other medical products. As a result of key provisions that PPMD and our community partners have moved forward within the last Prescription Drug User Fee Act (PDUFA) updates from 2012 (FDASIA) and, more recently, several provisions in the 21st Century Cures Act, the FDA now has a number of programs and policies in place to evaluate the benefits and risks of potential therapies and to gather and assess the patient perspectives.

 

But while much progress has been made, some significant gaps remain.

 

One such gap is the lack of any requirement in law today that the FDA include, as part of its risk-benefit framework, any patient experience or patient-focused drug development (PFDD) data. This means that the agency’s signature tool for evaluating risk-benefit does not have to include data from the patient perspective that could be critical to informing the agency’s evaluation and, ultimately, decision on whether or not to approve a product.

 

The BENEFIT Act, S. 1052

 

To address this gap, PPMD worked with our Congressional champions and HELP Committee leadership over the last several months, and on May 4 Sen. Wicker and Sen. Klobuchar introduced the Better Empowerment Now to Enhance Framework and Improve Treatments or the BENEFIT Act, S. 1052.

 

This proposed legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD, and related data – including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution – be considered as part of the risk-benefit assessment. This action will send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data.

 

The BENEFIT Act will also enhance an important transparency and accountability provision included in the 21st Century Cures Act by requiring the FDA to say how such patient experience and PFDD data was considered within the risk-benefit assessment for any approved therapies. This will provide additional learnings to all stakeholders, particularly patients, and help further refine and develop such tools going forward.

 

PPMD has also worked closely with many of our sister organizations and national coalition leaders on this legislation. The BENEFIT Act will positively impact communities far beyond our Duchenne community and we are grateful for the support and collaboration of our partners.

 

Moving Forward

 

We will continue to keep you updated as the BENEFIT Act moves forward in the Senate and disseminate Action Alerts for outreach as critical inflection points. We are hopeful that the nascent field of patient engagement will continue to flourish thanks to a continued interest and focus by Congress – and the powerful leadership and outreach of the PPMD community.

 

Views: 48

Comment

You need to be a member of PPMD Community to add comments!

Join PPMD Community

© 2017   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service