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Today – the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, S. 1052 – led by Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) passed in the Senate!

 

This means that – thanks to the leadership of our Senate champions and our Duchenne community – the agency’s signature tool for evaluating risk-benefit may soon 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.

 

Please take a moment to thank our Duchenne champions in the Senate for ensuring that patient engagement continues to be prioritized thanks to a continued interest and focus by Congress.

 

Our focus now shifts to our champions in the House of Representatives so that we can ensure that the critical momentum of the BENEFIT Act will continue and will be signed into law. Only then will we fill a sizeable gap by ensuring that patient experience data is fully considered as part of the FDA’s risk-benefit assessment for any new products.

 

Overview

 

Congress has made considerable progress over the past five years 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 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 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

 

To address this gap, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) introduced the Better Empowerment Now to Enhance Framework and Improve Treatments or the BENEFIT Act. This 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.

 

BENEFIT Passes the Senate

 

Just as Congress is working hard to finish up the current Congressional session before adjourning for the August Recess, our community’s champions were determined to ensure that the bi-partisan effort that had been introduced during the winter received due attention. We are so grateful to Senator’s Wicker and Klobuchar – and to all those on the HELP Committee and in the Senate who care so deeply about Duchenne and rare disease.

 

The passage of the BENEFIT Act signals that the nascent field of patient engagement continues to flourish thanks to a continued interest and focus by Congress.

 

What’s Next?

 

The BENEFIT Act will now move to the House of Representatives for consideration. At this time, a companion bill has not yet been introduced in the House and discussions with House champions are currently underway.

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