Patient-Focused Drug Development Gets a Boost From the 21st Century Cures Act


Depression and Bipolar Support Alliance

Congress gave mental health advocates a year-end present by passing mental health reform legislation as part of the 21st Century Cures Act and sending it to President Obama for signature. This is the third of three CFYM posts that highlight key pieces of the legislation that benefit individuals living with mood disorders and their families.

The December 13 and December 20 CFYM posts focused on the mental health reform package that became part of the 21st Century Cures Act. This bipartisan legislation passed the U.S. House in July, had support in the Senate and from the President, and was well-positioned for a successful journey to becoming law. What most advocates had not foreseen, however, was that the mental health reform legislation that had been in advancing in varying degrees in both Chambers would be included in that bill.

While this action was indeed a major victory for mental health advocates, this win should not overshadow the significant policy gains for individuals living with mood disorders that was the intent of the original 21st Century Cures bill.  According to the U.S. House of Representatives Committee on Energy and Commerce (E&C) fact sheet (download here) the legislation is “an innovative game changer, a once in a generation, transformational opportunity to change the way we treat disease.”

The E&C fact sheet further states that the legislation was the result of numerous conversations with stakeholders including patients, researchers, innovators, and health care providers with the intent of identifying steps to “expedite the discovery, development and delivery of new treatments and cures.”

Act recognizes value of patient-centered care
During 2016, CFYM shined a spotlight on the value of patient-centered care in the delivery of mental health services. Most recently, former member of Congress Tony Coelho shared in the October 18 post “that in order to create a system that better treats patients, we need legislators and decision makers to create laws and regulations that value and support patient input.”

In many respects this vision was realized when the 21st Century Cures Act became law. In three significant areas, the legislation advances the idea that the patient experience must be at the center of innovation in new treatment development.

First, the Act requires the Secretary of Health and Human Services (HHS) to take certain actions within six months of approval of a 505(b) application. This application, subject to review and determination by the Food and Drug Administration (FDA), is used to seek approval of a new drug (for clinical use) whose active ingredient has not previously been approved. The Act requires a statement by the agency that articulates how patient experience data was used during the review and approval process. The Act defines this data as information about a patient’s experience with a disease, including “the impact of such disease on patients’ lives” and the “patient preferences with respect to treatment of such disease or condition.”

It is encouraging to note that the FDA has already shown openness to this type of patient engagement as highlighted in the CFYM April 5 post. That post reported on the testimony of DBSA President Allen Doederlein during an FDA hearing on the impact of cognitive dysfunction for people living with depression. It is heartening to see that this predilection towards patient-reported experiences will now be required by law.

The bill also requires the FDA to publish guidance on the collection and use of patient experience data in drug development. In particular, the guidance will provide detail around the processes for

  • Ensuring patient experience data is “accurate and representative of the intended population”
  • Identifying what is most important to patients with respect to
    • “burden of disease,
    • burden of treatment, and
    • benefits and risks in the management of patients’ disease”
  • Measuring patient impact around the collection of the patient experience data in clinical trials
  • Analyzing clinical outcome assessments for purposes of regulatory decision making
  • Describing how HHS intends to respond to submissions of patient experience data
  • Determining the impact of patient experience data with respect to the structured risk-benefit assessment

Shedding light on the drug development process
The need for FDA’s transparency to the patient community is recognized with requirements to analyze the agency’s processes and procedures. Over the course of ten years, the FDA is required to publish three different reports that assess the use of the patient experience data in regulatory decision making. Emphasis will be given to how patient experience data influences the review of proposed treatments. All of these reports will be available to the public for online review and comment.

As CFYM has reported over the past year, shared decision making with clinicians leads to better outcomes. It is encouraging that our government is promoting the idea that shared decision making also has a role in the development of new treatments. It is our hope that this approach of learning from patients about which treatment outcomes are important—as well as the risks and benefits patients are willing to accept to achieve those outcomes—ushers in a new age of treating the whole health of the individual.

Your Turn

  • What results do you foresee from the FDA’s greater use of patient experience data in the treatment review process?
  • What changes to the mental health care system do you think will support the goal of “treating the whole health” of people with mood disorders?

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