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2015 policy landscape: A biomedical R&D planning guide

01/29/2015

policy Webinar

The legislative and regulatory calendars for 2015 are already filling up with opportunities to impact the biomedical research and development landscape in the United States – from 21st Century Cures to PDUFA VI negotiations to the impact of the omnibus on appropriations. In this Webinar, experts in federal budgetary, legislative, and regulatory matters talked about what to expect from Washington, when and how to plug in, and where to direct patient input for greatest policy impact this year.

  • Andrew Emmett, Managing Director, Science & Regulatory Affairs, Biotechnology Industry Organization
  • David Zook, Partner, FaegreBD Consulting
  • Moderator: Margaret Anderson, Executive Director, FasterCures

Summary

“2015 is an important year for what is possible in biomedical R&D,” commented Margaret Anderson while introducing a FasterCures-hosted Webinar on Jan. 29 about the outlook for medical research policy this year.

Along with guests Andrew Emmett of the Biotechnology Industry Organization (BIO) and David Zook of FaegreBD Consulting, the panel offered insights about what legislative and regulatory milestones to expect from Washington, and where to direct input for greatest policy impact this year.

A Banner Year for R&D Policy?

If the first weeks of 2015 are any indication, medical research is poised to have an elevated role on the national agenda this year. Between the President’s remarks at the State of the Union, the release of the 21stCentury Cures Act discussion draft, the announcement of the White House’s Precision Medicine Initiative, the health investment proposed in the FY2016 budget, and the upcoming Prescription Drug User Fee Act (PDUFA) VI negotiations, this stands to be a particularly busy year for advancing policies that further biomedical innovation. 

Opportunities for input from stakeholders are plentiful, and reflect a move towards greater inclusivity in the policy making process and acknowledgement of patients as vital partners in their care.

 

Congressional Outlook for 2015 – Common Ground Still Possible to Find

Zook addressed the concern that with a Republican majority in both the House and Senate, political friction with the White House could stymie progress. “Common ground is necessary to move forward on major legislative changes,” he said. He remains optimistic that many members, regardless of party affiliation, are showing the potential to work together on medical research issues and get things done for the benefit of the American people.

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Budget—Threading the Needle

The President’s budget request released Feb. 2 includes more than $70 billion in new spending above the caps set by the Budget Control Act of 2011. Zook noted that the unified control of the House and Senate by Republicans increases the chances that a regular order budget will be passed by Congress this year. However, he also cautioned that the continued trend of rising mandatory spending means that “discretionary spending, which includes the National Institutes of Health (NIH) budget, will continue to get squeezed.”

Another concern, he pointed out, is the potential for across-the-board cuts to all federal agencies, including the NIH and U.S. Food and Drug Administration (FDA), in a process known as sequestration. “We’re heading into difficult territory on the appropriations front,” he cautioned.

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21st Century Cures – Discussion Draft Takes on R&D System Reform

After almost a year’s worth of listening to and learning from the biomedical and patient communities, the U.S. House Energy and Commerce Committee, under the leadership of Committee Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO), released the 21st Century Cures Act discussion draft on Jan. 27 with the aim of modernizing the discovery, development, and delivery of new therapies to patients. Zook called the 2014 listening process that led up to the release of the draft “amazing and impressive,” and Emmett echoed that “the amount of public engagement has just been unprecedented.”

Emmett was particularly enthusiastic about the overarching emphasis on incorporating patient perspectives throughout the R&D process, and the unique effort to gather input around FDA reforms outside of the PDUFA cycle. Since 1992, he explained, PDUFA reauthorizations must pass Congress every five years and have become lynchpins of FDA reform legislation. Instead, the 21st Century Cures process is “looking at the full cycle of drug development, including delivery, health information technology, and regulatory science.”

The draft bill also recommends the creation of a “21st Century Cures Consortium” modeled after the European Union’s Innovative Medicines Initiative that would bring together federal agencies — including the NIH, FDA, and Centers for Medicare & Medicaid Services — with leaders from the biomedical industry, academics, patient groups, health plans, and others to “foster collaboration, establish a strategic agenda, identify gaps and opportunities, facilitate interoperability, award grants/contracts to accelerate discovery, development of cures, treatments, prevention.” Emmett believes that the proposed consortium will “facilitate regulatory approval through better pooling of data and leverage for supporting drug development.”

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With the goal of passing a bill before Memorial Day, the Committee has its work cut out for it, both speakers agreed. But while many changes may occur to the current 400-page draft, they are optimistic about the probability of the bill becoming law this year. 

  • The House Energy and Commerce committee is currently seeking feedback on the proposals outlined in the draft. All interested stakeholders are encouraged to submit specific suggestions about how to improve the legislation to cures@mail.house.gov.
  •  The Senate has launched a complementary process to seek bipartisan input on reforming the drug discovery cycle with a report released last week, Innovation for Healthier Americans, by Chairman of the Senate HELP Committee Sen. Lamar Alexander (R-TN) and Sen. Richard Burr (R-NC). They are seeking comments on the report by Feb. 23, and will begin hearings in March.


PDUFA VI

Many believe that the 21st Century Cures initiative will dovetail into the next major opportunity for stakeholders to come together to address challenges facing the FDA—the PDUFA VI negotiation process. As Emmett explained, regulated industry will have a series of meetings with the FDA beginning in June 2015, with parallel meetings between the FDA and other stakeholders (including patient advocacy organizations), leading to an FDA-industry agreement by March 2016.

Emmett pointed to efforts to expand the feedback collected under the PDUFA V-initiated Patient-Focused Drug Development Initiative, which invites patients to share their input on benefits and risks with the FDA. 21st Century Cures includes, he explained, a new section that explicitly links the statutory benefit-risk framework to a process for submitting more quantitative patient experience data. 

“Safety and efficacy are not absolutes,” he said. “It’s a process of balancing benefits and risks in the context of unmet medical needs. And as FDA makes those very difficult value judgments, it is critical that it is done through the lens of the patient experience.” 

Emmett complimented the FDA for its recent successes in improving the regulatory approval process, noting that the 41 novel new drugs approved in 2014 was an 18-year high for the agency. He also pointed out that the PDUFA discussions are singularly focused on FDA processes, not the entire drug development cycle the way the 21st Century Cures is.

FasterCures is hopeful that with this strong early momentum, 2015 will prove to be a productive year for medical R&D policy. We look forward to continuing our efforts to pave a path of meaningful engagement between patients and policymakers as the year unfolds.