A few weeks after finishing
negotiations with FDA over a PDUFA V deal that seeks to tweak drug
oversight with changes to the review process, BIO now plans to propose
sweeping changes to the U.S. regulatory system.
This week the biotech trade
association will call for the creation of a provisional approval pathway,
mandates for FDA to promote medical innovation, and a complete restructuring of
the agency's relationships with Congress and the White House.
BIO's FDA innovation agenda is
coupled with proposals for congressional action to bolster capital formation (see
"Talking Capital in the Capitol," A14).
Although it is separate from
the PDUFA deal, BIO's package of regulatory innovation proposals is certain to
provide food for thought for congressional committees that are just starting to
consider legislation that could accompany the reauthorization of user fees.
Underscoring the connection between regulatory innovation and
PDUFA, a member of BIO's board of directors will present the organization's
recommendations at a July 7 House Energy and Commerce Committee hearing about
PDUFA. The committee, which has jurisdiction over FDA, has also invited the National
Venture Capital Association to testify about recommendations developed
by its Medical Innovation and Competitiveness Coalition (MedIC).
MedIC includes life science VCs
and their portfolio companies.
Separately, patient and consumer groups are lobbying Congress
with ideas for changing the way medical products are regulated. Some of the
patient proposals have been incorporated into the National
Health Council's Modernizing Our Drug & Diagnostics
Evaluation and Regulatory Network (MODDERN) Cures Act (see BioCentury,
Monday, May 30).
BIO plans to first release
concept papers describing its ideas for revitalizing the U.S. biotech industry,
and then seek congressional champions to introduce comprehensive legislation in
both houses of Congress, BIO President and CEO Jim Greenwood told BioCentury.
Several of BIO's and NVCA's
highest impact proposals, including creation of a progressive approval pathway,
could be accomplished without congressional intervention, but BIO has concluded
public debate and legislation are needed to provide an impetus for action.
Progressive or conditional
approval has been discussed at FDA and among regulatory experts for years. The
idea is to move away from a binary approval decision to a gradual easing of new
drugs onto the market for defined patient populations, with restrictions on use
modulated based on real-world data.
Conditional approval was at the
heart of proposals for regulatory innovation presented in BioCentury's 2010
Back to School report (see BioCentury, Sept. 13, 2010).
On the June 12 broadcast of BioCentury
This Week, BioCentury's public affairs television program, FDA
Commissioner Margaret Hamburg said she has discussed the possibility of
creating a progressive approval pathway with Janet Woodcock, director of the
agency's Center for Drug Evaluation and Research (CDER).
Hamburg has been briefed on BIO's
proposals, Greenwood said.
Under BIO's version of
progressive approval, FDA would allow sponsors to market therapies for unmet
medical needs that provide significant advances to standard of care, or for
targeted therapies, based on solid safety data and evidence that such a therapy
is "more likely than not to provide favorable benefit-risk to its intended
patient population," according to briefing slides BIO presented to patient
groups last week.
This could mean allowing drugs
on the market after Phase II trials and while Phase III trials were under way,
Sara Radcliff, EVP for health at BIO, told BioCentury.
products approved under the
progressive pathway could be restricted using FDA's authority to impose REMS
that limit which patients and physicians can receive or prescribe a drug, and
with requirements for postmarket registries.
Rather than propose a new
pathway, NVCA is calling for FDA to "expand its use of the accelerated
approval pathway for new drugs." It thinks "Congress should make
clear that the agency has the authority and the public mandate to behave
flexibly, without a 'one-size-fits-all' standard of evidence, in approving
breakthrough drugs for unmet needs where evidence suggests a reasonable
likelihood that the benefits outweigh the risks."
NVCA and BIO also want Congress
to prod FDA into weighing unmet need more heavily when deciding whether and
when products should be approved under existing pathways.
"FDA should update its
regulatory standard for balancing the benefits and risks of breakthrough
products by emphasizing that the evidence of benefit required of a new product
should depend on the level of unmet medical need it addresses and its likely
safety risk," NVCA proposes.
On a mission
Both BIO and NVCA want Congress
to redefine FDA's mission so the agency is formally charged with advancing
"Congress should amend and
strengthen the FDA's statutory mission to include promoting public health
through acceleration of access to novel therapies and technologies and require
the FDA to routinely assess the impact of its decisions, policies, and
priorities on unmet medical needs and medical innovation using agreed upon
metrics," according to an NVCA memo outlining MedIC's proposals.
BIO is also calling for a fixed term of office for the FDA
commissioner, echoing a recommendation from a 2006 Institute
of Medicine report, "The Future of Drug Safety: Promoting and
Protecting the Health of the Public."
The commissioner position is "a
somewhat thankless job. Job security will help attract the best candidates,"
Greenwood told BioCentury. A fixed term is also intended to provide some
insulation from political pressure.
BIO takes political
independence a step further, calling for FDA to be removed from HHS and turned
into an independent agency with a head who reports directly to the president,
like the Environmental Protection Agency. This would "eliminate a level of
bureaucracy," Greenwood said.
BIO is also calling for an
external management review board to advise the commissioner.
To bolster FDA's capacity to
advance scientific innovation, BIO wants Congress to lift restrictions on
funding the Reagan-Udall Foundation. Congress created the foundation in 2007 as
a public-private partnership to oversee and fund precompetitive research to
advance drug development and regulation. Rep. Rosa DeLauro (D-Conn.) has
blocked the funding, saying it is dominated by drug companies.
Another BIO proposal for
turning FDA's attention to advancing science and medicine is the creation of a
chief innovation officer with "authority to pilot and develop strategies
for implementation of promising scientific and regulatory approaches in review
Both BIO and NVCA also want to
improve the advisory committee process.
restrictions have severely restricted the pool of experts who can serve on
advisory committees. NVCA is calling for those COI rules to be amended, and to
replace restrictions on committee membership with requirements for disclosure
BIO wants Congress to create a
chief medical policy officer who would "oversee and improve the FDA
advisory committee process." This officer also would have authority to
resolve broad medical policy disputes within and across FDA's divisions and
Also, BIO wants Congress to
require that advisory committees include a medical or scientific expert
appointed by patient organizations.
NVCA thinks the committees
should include an "innovation advocate" representing investors and
entrepreneurs who finance medical innovation.
BIO has created concept papers
describing its proposals, and has discussed them with "key members of
Congress and their staff," Greenwood told BioCentury. It hopes to have its
regulatory and capital formation proposals combined in one bill that would be
introduced in the Senate.
Because of the House's
different jurisdictional structure, Greenwood envisions two bills in the House,
one focusing on FDA, and the other on tax and economic issues.
Greenwood doesn't expect the
bills to be enacted, but feels that presenting all of the ideas as a package
illustrates the need for comprehensive changes.
BIO's hope is that members of
Congress will pick out and champion various elements of its plan and
incorporate them into bigger legislative initiatives.
Industry Organization (BIO), Washington, D.C.
of Medicine (IOM), Washington, D.C.
Venture Capital Association (NVCA), Arlington, Va.
Health Council, Washington, D.C.
Food and Drug Administration (FDA), Silver Spring, Md.