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Legislators Borrow From FDA's Approach in Seeking Patient Input About Regulatory Process

Posted 19 May 2014 | By Alexander Gaffney, RAC 

Legislators Borrow From FDA's Approach in Seeking Patient Input About Regulatory Process

House legislators are quickly moving ahead with a new plan that hopes to overhaul how the US regulatory system reviews new medical products, and are now soliciting input from a group that has not traditionally been at the center of the regulatory process: patient advocacy groups.

Background

In late April 2014, Reps. Fred Upton (R-MI) and Diana DeGette (D-CO) announced the launch of a new initiative called the "21st Century Cures Initiative," which they said would be aimed at accelerating the "pace of cures and medical breakthroughs in the US."

"Health research is moving quickly, but the federal drug and device approval apparatus is in many ways the relic of another era," DeGette said in a statement. "We have dedicated scientists and bold leaders at agencies like the NIH and the FDA, but when our laws don’t keep pace with innovation, we all lose."

"If we want to save more lives and keep this country the leader in medical innovation, we have to make sure there’s not a major gap between the science of cures and the way we regulate these therapies," Upton said in the same statement.

While the nascent initiative is only just beginning to bring together stakeholders, it has already held one roundtable with some of the top regulatory and healthcare officials in the US, including officials from the US Food and Drug Administration (FDA).

New Focus on Patient Groups

But now the initiative is reaching out to patient advocacy groups, a traditionally under-represented segment in regulatory decision-making, for input.

To hear the legislators' explanation, they hope to do at the macro level what FDA is now doing with individual patient populations with its Patient-Centered Drug Development program.

"The committee is seeking input from patients and advocates regarding what cures and treatments are available for individual diseases, how they work with researchers and other patients, their experience with clinical trials, and what role government has played," legislators wrote in a 16 May 2014 statement.

In a new whitepaper accompanying the statement, legislators expanded upon this statement, posing a set of questions that appear to be remarkably similar to those posed by FDA in its patient-centered meetings.

The legislators ask:

  • What is the state of discovery of cures and treatments for your disease? Are there cures and treatments now or on the horizon?
  • What programs or policies have you utilized to support and foster research, such as patient registries, public-private partnerships, and venture philanthropy?
  • How can Congress incentivize, coordinate, and accelerate basic research for diseases we know relatively little about?
  • How can we work together to better translate advances in science into safe and effective new therapies for patients?
  • How do you coordinate your research and outreach with other patients?
  • How do you learn about new treatments and cures? How do you communicate with other patients regarding treatments and cures?
  • What can we learn from your experiences with clinical trials and the drug development process?
  • What is the role of government in your work, including any barriers to achieving your goals and advancing breakthroughs?
  • How should regulators evaluate benefit-risk? How do you work with regulators regarding benefit-risk? Can this process be improved?
  • What is the role of public and private funding in the research and development of cures and treatments?
  • Are there success stories the committee can highlight and best practices we can leverage in other areas?
  • How have you worked with other patients to support one another?
  • What is the financial burden of your disease? How would better treatments and cures help save money for your family and the federal government?
  • How can Congress help?

Also notable is the relatively quick turnaround time. Patient groups will have to submit their responses to the Energy and Commerce committee by 13 June 2014—less than a month from now.

E&C Statement

White Paper

Categories: Regulatory News

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