by: Parent Project Muscular Dystrophy
When people think of dynamic duos and classic pairings, they probably think of Batman and Robin, Fred and Ginger, or Oreos and milk. For those of us in the Duchenne and rare disease community, we probably think of Senators Roger Wicker and Amy Klobuchar.
Senators Wicker and Klobuchar have done so much for the Duchenne and muscular dystrophy community broadly over so many years. They have written or revised the MD-CARE Act. They have led or supported our annual efforts to ensure those laws and their programs achieve their intended results. They have led the congressional push to ensure the voice of the patient is listened to by the Food and Drug Administration (FDA). And today, they have reintroduced the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act ahead of PPMD’s 2021 Advocacy Conference on March 10th (this year, being held virtually — and there’s still time to register!).
Establishing the Foundation
A little more than four years ago, these two lawmakers wrote what was commonly referred to as the Patient-Focused Impact Assessment Act that became law via the 21st Century Cures Act. Its name may sound complicated, but its intent is simple as it requires the FDA to report on how they use patient experience data as part of the review of any drugs they approve. It also requires FDA to issue more guidance to developers of patient experience tools so organizations like PPMD know what the agency wants to see so we can make the best decisions about spending our time and resources.
Fast Forward to 2021 and this law is working. FDA includes a patient experience summary table as part of the data package it releases for all approved drugs. While there is some room for improvement to standardize and beef up the data being reported, the law is achieving the results we hoped for.
Reintroducing the BENEFIT ACT
Senators Wicker and Klobuchar have just reintroduced a bill, known as the Better Empowerment Now to Enhance Framework & Improve Treatments (BENEFIT) Act, that seeks to build upon this foundation by ensuring patient experience data is a core component of the FDA’s risk-benefit framework.
While FDA has done much right over the years, we need to ensure patient experience data – such as a patient preference study, patient reported outcomes or a voice of the patient report – is enshrined in our nation’s food and drug laws. The BENEFIT Act will make this small but very important goal a reality.
We Need Your Voice!
Now that this bill has been introduced in the new 117th Congress, it’s our time to do our job. This means you calling and writing your Senators – and soon your House Member – to ask them to cosponsor this bill. And it means PPMD working with key Congressional committees so the BENEFIT Act will become law this Congress.
The BENEFIT Act will be part of PPMD’s Advocacy Conference on March 10th – with this year going virtual due to the pandemic. That means all families can participate! And there is still time to register. For those unable to attend virtual meeting, be on the lookout for PPMD Action Alerts to make the request online to your members.
Register for PPMD’s Virtual Advocacy Conference >In 2017, we got halfway there when the bill passed the Senate. Now, we need to finish the job so the BENEFIT Act can become law and our dynamic duo of Wicker and Klobuchar can enact yet another law to benefit the Duchenne and countless other disease communities.
