21st Century Cures: A sign that the tide could be turning

If I were to tell you that it takes an average of 10 years and one billion dollars to yield a single new therapy – would you be surprised? Probably not, yet none of us has that luxury of time or money.

If I were to share that many of our nation’s leaders admit that our scientific and clinical institutions and the agencies that regulate them are set up in an antiquated and inefficient way – would you be surprised? Again, not likely. But we cannot afford to lose precious time and momentum on inefficiencies.

But there is a sign that the tide could be turning. And PPMD – our staff leadership, our community members, our clinicians and scientists, and our advocates – has been at the forefront of this turning tide.

On April 30th of this year, Congressional leaders launched the most unified effort of the 113th Congress aimed at bridging the gap between scientific innovation and the delivery of therapies to patients – the “21st Century Cures” initiative. A collaboration led by Representative Fred Upton (R–MI), the chair of the Energy and Commerce Committee, and Representative Diana DeGette (D–CO), one of the panel's senior Democrats, 21st Century Cures has brought together stakeholders from all facets of the scientific, clinical, biomedical, and regulatory space, as well as the patient community to evaluate where the opportunities to streamline processes and refine the system are.

Earlier this year, Ryan provided a terrific summary of some of the ways that our community members have been working to ensure that the issues of importance to our Duchenne community remained central within the 21st Century Cures discussions in Washington, D.C. and around the nation. In addition, our team has been working closely with the Energy and Commerce Committee leadership as they now work to translate all of the suggestions provided through hearings and Roundtables throughout the summer months, to create legislation. The 21st Century Cures bill will be a culmination of input from many stakeholder groups and will include both big and narrow ideas aimed at changing the two statistics I shared. We must ensure that we can streamline therapeutic development processes, incentivize all stakeholders, and get life-saving drugs through regulatory approval faster.

PPMD has submitted critical ideas and related legislative language to the 21st Century Cures bill that centers on 2 provisions that reflect the progress made by the Duchenne community, the leadership that we have taken, and roads we have yet to pave:


1.  Development of the FDA Patient Impact Assessment

The Food and Drug Administration Safety and Innovation Act (FDASIA) and related PDUFA and MDUFA agreements included a number of provisions under the patient-focused drug development and patient preference headings. While our community greatly appreciates these actions and has seen an enhanced engagement between individual stakeholders and the Agency, there exists no transparent process to evaluate how the FDA is actually using these tools and whether increased engagement is yielding an impact on outcomes. To address this concern, we recommend that the Cures legislation authorize a process to develop a patient impact assessment tool able to quantify how FDA reviewers use – or do not use – available patient-focused drug development tools and data in making review decisions. By focusing at the product review level, we will spotlight the most critical stage and better understand how the agency is utilizing its authorities and resources.


We recommend that the committee consider a tool that is both quantitative and qualitative and that the language ask for an assessment of how reviewers utilized the following inputs:


  • Patient-reported outcomes data
  • Patient preference data
  • Quantifiable benefit/risk data
  • Community-generated or FDA-produced guidance documents
  • Perspective of patients serving on advisory committees
  • Perspective of external medical and scientific professionals within relevant review panels
  • Other content as determined by the Secretary


If a reviewer did not take into account data from one or more of the fields, he or she should state why, including the absence of available data. We recommend that patient impact assessments be publicly accessible for all reviewed drugs, including those not approved, and that the FDA produce an annual report summarizing its patient engagement activities and utilization for the preceding year. PPMD believes that this transparency will make it much more likely that patient views are an integral part of the regulatory process.


2.  Issue Clear Guidance on Interactions of Industry and Patient Organizations

Thanks to the patient-focused drug development reforms of FDASIA, a new dawn is upon us in terms of the opportunity for industry sponsors and patient organizations to engage in collaborations aimed at informing the regulatory and review process. Such collaborations offer the possibility of far greater levels of understanding as to the patient perspective on important topics such as patient-reported outcomes and benefit/risk assessments that can inform reviewer perspectives on applications.  Despite this potential, we are concerned that a lack of clear and updated FDA guidance on such interactions may be deterring this critical collaboration.

To address this ambiguity, we recommend that the legislation direct the FDA to clarify in guidance that the Agency encourages collaboration between sponsors and patients to better capture patients’ views on benefits and risks and other important issues. Patients are the only stakeholders who can accurately define meaningful clinical benefit and detrimental risks of therapy. Industry should partner with patients to develop data and tools intended to present the patient perspective on therapies being developed to treat specific conditions and diseases.

In short, we have taken advantage of every opportunity to engage with the FDA and other regulatory bodies, led ground-breaking forums, supported a global registry, produced an historic benefit-risk study to provide quantifiable measures of patient preference, and published draft regulatory guidance for Industry,  yet we don’t know what outcomes all of these efforts will actually yield on product review. Furthermore, as we seek to develop mutually beneficial collaborations with industry partners, we must have the assurance that our efforts will not be seen as compromising our priority of accelerating therapy development.


Watch for updates as the bill is introduced early in the 114th Congress (likely early January) and we gear up for our February Advocacy Conference. This bill will be a fast moving train – and we are on board!


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