CMOs from some of the leading patient advocacy groups, acting
under the auspices of the National
Health Council, have spent two years crafting a legislative proposal
they hope will jumpstart personalized medicine and revive promising therapies
that have been abandoned because they lack adequate IP protection.
The proposed language for the
Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network
(MODDERN) Cures Act will be the basis for legislation to be introduced in the
House as a stand-alone bill by Rep. Leonard Lance (R-N.J.). But its best
prospect for enactment is to get folded into next year's PDUFA reauthorization.
Lance is a member of the Energy
and Commerce Committee, which has PDUFA jurisdiction, and FDA has invited the
National Health Council to participate in patient and consumer group
discussions about the PDUFA V authorization.
NHC is an association for patient advocacy groups involved
with chronic diseases and conditions. Its 49 patient group members include the American
Cancer Society, American
Lung Association, National
Multiple Sclerosis Society, as well as many rare disease advocacy
Its membership also includes professional societies and
associations such as the Biotechnology
Industry Organization and Pharmaceutical
Research and Manufacturers of America, as well as about 24 biotech and
The NHC's policymaking remains
in the hands of patient advocacy groups, COO Marc Boutin said on BioCentury
This Week, BioCentury's public affairs television program.
On the personalized medicines
front, MODDERN Cures includes provisions intended to create incentives for both
drug sponsors and diagnostics companies.
Under the legislative proposal,
drug manufacturers that collaborate with diagnostic companies on tests to
identify which patients to treat would be eligible for either six or 12 months
of data exclusivity. The additional exclusivity would kick in when a drug's
patent expires and is intended to offset the potential for companion
diagnostics to reduce market size, according to NHC.
A collaboration that yields a
diagnostic to determine which patients should receive an already marketed drug
or biologic would merit six months of additional data exclusivity. A
collaboration that yields a diagnostic at the time the drug is approved would
give the drug company 12 months of data exclusivity.
NHC's proposed language applies
only to companion diagnostics developed by a company other than the drug
"What we're trying to do
is incentivize pharmaceutical companies to develop these diagnostics as they
develop their medicines," Boutin said.
As the same time, diagnostic
companies would benefit from provisions in the proposed law that would update
Medicare policies created long before the advent of advanced molecular
Boutin told BioCentury This
Week that an outmoded Medicare reimbursement system has stifled diagnostics
development: "It is difficult to get [advanced diagnostics] approved,
challenging to get them a code so doctors can order them, and then they are
often reimbursed at a rate set in the early '80s."
MODDERN Cures seeks to move
from today's Medicare diagnostics payment paradigm, which sets payment based on
the process used to perform a test, to a system that pays based on value (see
BioCentury, Dec. 6, 2010).
When setting reimbursement
rates for new tests, CMS would be instructed to take into account the
impact on patient care; technical characteristics of tests including resources
needed to develop, validate and perform them; and recommendations from an
independent advisory panel created by the legislation.
NHC also wants to streamline
the cumbersome processes for establishing codes that must be in place before
tests can be used in the Medicare population.
To speed the coverage process,
the bill would require CMS to assign temporary national reimbursement
(Healthcare Common Procedure Coding System) codes for diagnostic tests on a
MODDERN Cures also would
provide incentives to develop "dormant" therapies that could treat
unmet medical needs, but have been abandoned or ignored by commercial sponsors
because of insufficient patent protection.
"We define dormant
therapies as having two requirements. First, and most important to us, it has
to address an unmet need," Boutin told BioCentury This Week.
Second, "it does not have sufficient patent protection to take it through
the FDA approval process."
As one example, NHC believes
there are compounds that failed to gain FDA approval, but after their
patent life expired, subsequent research indicated they could be useful for the
same or a different indication.
Under MODDERN Cures, sponsors
who could demonstrate that the lack of IP protection was preventing
commercialization could request a compound be designated as a dormant
therapy. Only treatments for unmet medical needs, including those that improve
outcomes or reduce risk compared to FDA-approved therapies, would be eligible.
The dormant drugs provisions
would only apply new compounds. Drugs that are already marketed would not be
NHC suggests dormant drugs
should be granted a period of data exclusivity, but would leave it to Congress
to grapple with the contentious issue of how long the exclusivity should last.
Boutin said NHC is hoping to
provide a period exclusivity to drug sponsors that is "roughly equivalent
to what they would have had, had they had a patent."
Whatever period is agreed
should be extended by six months if a sponsor conducts an FDA-requested study
in a pediatric population, according to the council.
The proposed bill prevents "double
dipping" by requiring that sponsors give up all other IP protections,
including patents and Orphan exclusivity, for indications protected under
dormant therapy exclusivity.
Cancer Society, Washington, D.C.
Industry Organization (BIO), Washington, D.C.
for Medicare and Medicaid Services (CMS), Baltimore, Md.
Lung Association, Washington, D.C.
Health Council, Washington, D.C.
Multiple Sclerosis Society, New York, N.Y.
Research and Manufacturers of America (PhRMA), Washington, D.C.