As FDA Takes Notice of Patients, Groups Trying to Influence Regulatory Policy do as Well

Regulatory NewsRegulatory News | 03 June 2014 |  By 

An advocacy group focused on accelerating the discovery and approval of new therapies—and in particular therapies for under-served and rare diseases—has launched a new effort aimed at helping to bring patient perspectives into the regulatory process.


In recent years, regulators and regulatory-minded groups alike have pressed to increase the involvement of patients in the regulatory process. The thinking is that patients are the best stakeholders to ask about certain topics, such as tolerance for risk and meaningful benefits.

Perhaps the most substantial of these efforts was created through the Food and Drug Administration Safety and Innovation Act (FDASIA), and is known as the Patient-Focused Drug Development Initiative. Formed under Section X of FDASIA, the initiative has focused on setting up meetings with patients suffering from debilitating conditions which lack adequate treatment options. To date, FDA has held eight meetings under the program.

The agency has also launched a new Patient Network, which it hopes will help to educate patients on the regulatory process, including how they can get involved with the regulation of new products.

But FDA isn't the only entity coordinating patient involvement.

In November 2013, industry trade group PhRMA launched its own patient initiative, partnering with several disease groups to crowd-source input from patients not able to attend FDA's patient-focused drug development meetings.

And in May 2014, legislators with the House Energy and Commerce Committee (E&C) announced that they, too, were reaching out to patients in the hopes of obtaining input on the regulatory process, with the intent of reforming it to be more patient-friendly. That process is still in its early stages.

FasterCures' Approach

Now, yet another group is throwing its hat into the ring. On 29 May 2014, the advocacy group FasterCures announced it would be forming a new "Benefit Risk Advisory Council" featuring members from a diverse group of stakeholders.

The group is set to "provide advice and recommendations aimed at expanding opportunities for patient perspectives to shape product development and influence regulatory decisions," FasterCures said in a statement.

“The tradeoffs between desired benefits and tolerable risks may look quite different whether you’re a patient, a physician, a regulator, or a drug/device developer. They also might change over time,” said Margaret Anderson, executive director of FasterCures.

Information obtained by the group will be used to provide feedback to drug developers and regulators, and FasterCures specifically mentioned FDA's new patient-centered initiatives under FDASIA as being its inspiration.

“For FDA to do its job properly, it must have a robust understanding of how patients perceive the burden of their own diseases and how they would weigh alleviation of their disease’s impact versus the potential risks of any new treatment," said Robert Meyer, director of the Virginia Center for Regulatory Sciences at the University of Virginia School of Medicine and member of the advisory council, in a statement.

The group includes members from industry, patient groups, trade groups (PhRMA, BIO), rare disease advocates (NORD), academia and even government (PCORI's Joseph Selby).


FasterCures Statement

Benefit Risk Advisory Council


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