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Let's Get PDUFA V Approved, Fast

January 12, 2012
By Marc Boutin, JD, Executive Vice President and Chief Operating Officer, National Health Council

At this time of the year, it can be helpful to reflect on the past to guide us in the future. I was recently reading an editorial about the National Health Council (NHC) printed in January 1957 – 37 years after the organization’s creation. It spoke about the need for action with “an unprecedented degree of cooperation among health agencies and the people they serve.” Well, that time is now and the issue is reauthorization of the Prescription Drug User Fee Act (PDFUA).

[caption id="attachment_5738" align="alignright" width="115" caption="Marc Boutin, JD, Executive Vice President and COO, National Health Council"]Marc Boutin[/caption]

About a year and a half ago, at a Food and Drug Administration (FDA) meeting with patient advocacy organizations, we talked about how it was patients, two decades earlier, who chained themselves to the gates outside the FDA and the National Institutes of Health demanding access to treatments that were still in clinical trials and under review. Patients were told that they couldn't have access to those treatments because researchers didn't know if the compounds were safe or effective. But the patient community pushed back; many were going to be dead in a year. Their actions resulted in the creation of the early access program and a new environment that led to the first PDUFA.

I want to thank the FDA and the pharmaceutical industry for again hearing the concerns of patients and for addressing our issues in a meaningful way. Today, we have a forward-thinking reauthorization agreement – PDUFA V.

Since the authorization of the first PDUFA, we have seen tremendous progress in the development of medicines for people with chronic diseases and disabilities. The time it took for the FDA to approve new drugs got shorter. However, there is now an emerging frustration among many people with chronic conditions who feel they still are not getting access to treatments as quickly as they would like. For many, such as patients with degenerative diseases like Alzheimer’s, clinical development moves slower than the progression of the disease.

For the past year and a half, the patient advocacy community has worked with the FDA and industry to get its three key priorities included in the proposed reauthorization agreement.

First, we need a qualitative, objective, framework for assessing the benefit-risk of new drugs. Benefit-risk is an important part of the FDA review process, but there is no consistently used framework or agreement among all stakeholders. How do we account for known and unknown risk or variation among subpopulations? Are benefits and risks appropriately placed in the therapeutic context?

No country in the world has articulated such a process, and there is increasing anxiety among patients who are denied access to new drugs based on a benefit/risk assessment they simply don’t understand.

The FDA and industry have agreed to include in PDUFA V the creation of a qualitative framework that would assess the level of certainty and variability of the benefits and risks, and that would provide for flexible scoring in the context of the therapeutic indication.

This benefit-risk framework will work for both patients and consumers. Both are stakeholders in this issue, but depending on where you fall on the health care needs spectrum, your perspective of benefit-risk can shift dramatically. For healthy consumers, the tolerance for risk, variability, and uncertainty for a new medicine to treat a condition such as hay fever is virtually zero. But if you are diagnosed with ALS (Lou Gehrig's disease) and you have only two years to live, your tolerance for a risky new drug is dramatically different.

Second, we need greater use of patient-reported outcomes and biomarkers. Patients want more of a say in the drug review process, and they want the approval process to go faster than the progression of their disease. The patient community saw the reauthorization of PDUFA as an opportunity to develop metrics and tools to make the review process more effective, more efficient, and allow for the delivery of safe and effective medicines to people who need them.

Under PDUFA V, resources for patient-reported outcomes (PROs) will be increased, resulting in the patient perspective being incorporated earlier in the review process, dramatically reshaping drug research. It was psoriasis patients who taught us that it is not necessarily the size of their lesions that matters, but where the lesions are located on their body, such as on their faces or joints. This realization greatly altered the focus of psoriasis drug research.

PDUFA V would also increase resources to help the FDA speed up the approval of biomarkers in clinical trials. The use of these surrogate endpoints or clinical markers is greatly needed for people with chronic conditions. Polycystic kidney disease (PKD) is a genetic disorder of the kidneys. With PKD, fluid-filled cysts develop in the kidneys, which then can increase in both size and weight, sometimes weighing many pounds each. Currently, the recognized end point for PKD is kidney failure, which can occur as late as 40 years after diagnosis. No company would undertake a 40-year clinical trial, so qualifying a biomarker for a PKD drug is imperative. It is conceivable that if a new compound prevents a PKD patient’s kidney from getting larger, then something positive is happening.

Third, for the millions of people with rare diseases, we need new resources and greater flexibility in the regulatory review process. As proposed by FDA and industry, PDUFA V will utilize new regulatory science to speed the development of and access to new medicines for people who desperately need them.

The opportunity to engage in the PDUFA reauthorization process has been tremendously beneficial for the patient advocacy community, and I hope for the FDA and industry.

I say to those reading this column and to members of Congress, let's get PDUFA V approved. Let's get it done fast.