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 organizations.

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 pharmaceutical companies.

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 manufacturer.

"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 diagnostics.

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 quarterly basis.

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 eligible.

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.


American Cancer Society, Washington, D.C.

Biotechnology Industry Organization (BIO), Washington, D.C.

Centers for Medicare and Medicaid Services (CMS), Baltimore, Md.

American Lung Association, Washington, D.C.

National Health Council, Washington, D.C.

National Multiple Sclerosis Society, New York, N.Y.

Pharmaceutical Research and Manufacturers of America (PhRMA), Washington, D.C.