UPDATE – Putting Patients First: Recommendations to speed responsible access to new therapies for Duchenne

Two weeks ago, PPMD proudly published Putting Patients First, a white paper outlining recommendations to .... In that short amount of time, the reaction to this white paper has been phenomenal. From interest by the media to accolades from industry and agencies, we have been truly humbled by the response we have received.

And while the Duchenne community has been particularly responsive, I realized that many of you may not understand the significance of this white paper and how it will help PPMD achieve our ambitious advocacy agenda.

So if you’ll indulge me, I wanted to give a little background of where we’ve been, where we are, and where we are striving to go. 

A look back- How we got here

The single most significant event to happen in Washington, D.C. for the Duchenne community was the passing of the MD-CARE Act in 2001. Many of you know and understand the significance of this legislation, which we are currently in the process of reauthorizing. But if you need a refresher or if you are new to our community, take a minute to learn more about the MD-CARE Act and see how you can help to pass this important legislation. The MD-CARE Act has played a key role in moving the field forward impacting a number of programs and bringing many of the therapies we are seeing today in early and late stage trials into fruition.  

Looking at Today- What regulatory law says

Public Law 112-144

On July 9, 2012, the President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012. This new law includes the reauthorization of the Prescription Drug User Fee Act (PDUFA) that provides FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products. 

This is the fifth authorization of PDUFA that includes Title I of FDASIA and the performance goals and procedures for PDUFA V.  PPMD, biopharma representatives, healthcare professionals, as well as, many other advocacy groups participated in meetings and discussions around proposed enhancements which are referred to in Title I of FDASIA. PPMD advocated with other rare disease organization for the strongest possible provisions in the law for rare diseases. The final bill included those provisions giving the FDA unprecedented tools for speeding the development of potential therapies. 

The new law ensures that FDA will continue to receive a source of stable and consistent funding during fiscal years 2013-2017 that will allow the agency to fulfill its mission to protect and promote public health by helping to bring to market critical new medicines for patients.

The legislative process may well be a memory of some long-forgotten government class.  But the history of PDUFA – and specifically PDUFA V – should be on your ‘to do’ list for many very important reasons.  Renewed every five years, PDUFA is one of the single most important and successful pieces of legislation affecting the FDA, the pharmaceutical and biopharmaceutical industries, and patients.

Congress passes laws and within those laws, Congress outlines the road, draw the white lines on the road which ensures support for and outlines a framework for the FDA to review drug applications.   This ‘road to approval’ provides FDA opportunities to apply discretion and flexibility to the review process in an effort to deliver promising therapies to the patients who need it.

The ‘road’ has been paved by Congress.  It is now up to the regulatory agencies to utilize the tools that have been developed, to apply flexibility and discretion where possible and to accelerate the approval process.  The patients – our children – are waiting.

Preparing for Tomorrow- How we can affect what is coming through the pipeline


We have been working with the FDA and other regulatory agencies for the last decade to educate them on Duchenne and the catastrophic affect this disorder has on both patients and families. As our relationship continues to grow and build, and with the implementation of FDASIA, we are hopeful that the FDA will work with PPMD and the rare disease community to take advantage of the new opportunities created by this landmark legislation.

To ensure we make the most of these new opportunities, PPMD brought together an expert Advisory Committee, including leading voices in academia, industry, and patient advocacy, to issue recommendations about how to effectively evaluate new therapies for Duchene and other, rare, serious, and life-threatening disorders.   We are pleased to share these recommendations with you and have already begun to disseminate them to all stakeholders.   Read the white paper recommendations. For a brief synopsis of the white paper recommendations, see the executive summary.


After years of investments in research, there is now a very promising pipeline of therapies under investigation for the treatment of Duchenne.  As you know, at least seven compounds are in Phase 2 or 3 clinical trials and are approaching consideration for approval.  This rich pipeline is giving new hope to our community and we must work together to ensure that potentially life-saving therapies are evaluated in a manner that will allow us to speed responsible access.


Last week we hosted a webinar exploring the white paper more and outlining more of our strategy. If you missed that presentation, click here to download and watch.


I think as time goes on, these recommendations will prove to have been a critical tool in the approval of Duchenne therapies. We are so grateful to your ongoing support and involvement in our advocacy efforts and hope you will continue to participate.


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