PPMD Heralds Final Approval of Cures Bill That Contains Patient Experience & Other Policies to Advance Therapy Development

The bill, which cleared the Senate today and now heads to President Obama's desk, contains several provisions supported and driven by PPMD, including Sections 3001 and 3002, which will require the U.S. Food and Drug Administration (FDA) to disclose how patient experience data factored into the review of all approved products. The legislation also requires the FDA to issuance additional guidance or direction to inform the further development and use of patient experience data by the agency.

The provisions are rooted in bipartisan legislation introduced in June 2015 by Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) known as the Patient-Focused Impact Assessment Act (PFIA). A modified version of the bill was approved by the Health, Education, Labor, and Pensions (HELP) Committee in March. PFIA (S. 1597) was also cosponsored by Senators Susan Collins (R-ME), Al Franken (D-MN), Johnny Isakson (R-GA), Michael Bennet (D-CO), Joe Donnelly (D-IN), Tom Cotton (R-AR), and Lisa Murkowski (R-AK).

"PPMD is so grateful for the tremendous leadership of Senators Wicker and Klobuchar and for the work of HELP Chairman Lamar Alexander (R-TN) and Ranking Member Patty Murray (D-WA), as well as House Energy & Commerce Committee Chairman Fred Upton (R-MI) and Ranking Member Frank Pallone (D-NJ) in developing and advancing this most important policy provision to advance the development and use of patient experience data," said PPMD Founding President and CEO Pat Furlong.

"For several years, PPMD and the Duchenne community have led the development of tools in this domain, including a patient-developed draft guidance and related projects. These provisions will bring about important reporting and transparency requirements so stakeholders can better understand how such tools are or are not being used, as well as clearer direction and guidance to those who want to know how to collect patient experience data in a way that will maximize their utility to the FDA," Furlong added.

"The reforms and investments in medical research included in the 21st Century Cures Act are a big win for patients," said Sen. Wicker. "I am pleased that language inspired by my Patient-Focused Impact Assessment Act was included in the package that passed the Senate today. Helping patients and advocates understand how the FDA uses patient experience data will be essential as the FDA considers new therapies. I am honored to have worked with Parent Project Muscular Dystrophy to enact this policy, helping us get one step closer to a cure for Duchenne. I remained committed to finding a cure for this terrible disease and ensuring that Congress provides NIH and FDA with the appropriate resources to do so."

"Patients deserve a strong voice in the drug development process," said Sen. Klobuchar. "Our bipartisan legislation will help advocates understand how the Food and Drug Administration engages patients, including those with rare diseases, as it reviews drugs and therapies. It will also increase transparency in how the FDA uses patient-focused drug development tools. Passing this bill into law moves us one step closer to improving the quality of life for those living with rare diseases."

In addition to the patient experience provisions, the Cures bill includes a number of other provisions strongly supported by PPMD. These include policies to help further accelerate the pace of reviews by the FDA, including by exploring uses of novel clinical trials designs and by permitting sponsors of rare disease therapies to incorporate data from previously approved product submissions that use the same or similar underlying technology as the candidate therapy.

The legislation also reauthorizes the Pediatric Rare Disease Priority Review Voucher to help further incent development of drugs for rare pediatric diseases and requires biopharmaceutical manufacturers to disclose their policies for expanded access or compassionate use.

"PPMD is very pleased that this legislation will soon become law, and we look forward to continuing to build on the patient experience provision in 2017 as Congress prepares to reauthorize the Prescription Drug User Fee Act (PDUFA)," Furlong said.

To learn more about PPMD's advocacy efforts, please visit the website.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.  

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SOURCE Parent Project Muscular Dystrophy

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