The Milken Institute's Center for Accelerating Medical Solutions hopes to begin operations in Washington, D.C. during the summer.

The newly formed think tank will study the FDA approval process and incentives for innovation. CAMS is currently in the fundraising stages and will be managed out of the Milken Institute's Santa Monica headquarters while it seeks to recruit staff and establish a D.C. presence.

CAMS' mission will be to "mobilize economists, medical researchers, clinicians, biologists, ethicists, genomics specialists, chemists, physicists, mathematicians, computer scientists, legislative analysts and others to evaluate the entire research process from beginning to end and then publish concrete policy recommendations and provide leadership for their implementation," the organization says in an "action plan" circulated to potential donors.

"The center is non-profit, non-partisan and independent of interest groups," the action plan states.

However, CAMS' mission to identify "new efficiencies that can accelerate scientific discovery and suggest changes in misplaced priorities, sometimes inefficient regulations and conflicting incentives that slow progress" is of obvious appeal to the research-based pharmaceutical industry.

Milken Institute Chairman Michael Milken presented the CAMS concept to the Pharmaceutical Research & Manufacturers of America during the association's annual meeting in Naples, Fla. March 28.

The action plan acknowledges the contributions of several current and former pharma executives in formulating the project, including Abbott President Jeff Leiden, MD/PhD; former Bristol Worldwide Development head Stephen Carter, MD; and Celera Genomics Chief Medical Officer Samuel Broder.

The action plan also acknowledges the contribution of FDA Commissioner McClellan (another speaker at the PhRMA meeting).

The CAMS plan quotes approvingly from McClellan's innovation initiative, unveiled Jan. 31 (¹ [A#00650050012], p. 15). McClellan's "attempt to speed up the approval process for drugs" is a "positive sign," the document states. As part of the innovation initiative, McClellan has recently recruited advisors to join the agency from the American Enterprise Institute and the University of Chicago (see ² [A#00650150001]).

McClellan is also quoted in the CAMS report (from a 2001 paper co-authored with Kennedy School Professor of Economics David Cutler, PhD) on the economic importance of improved health care: "The benefits from just lower infant mortality and better treatment of heart attacks have been sufficiently great that they alone are about equal to the entire cost increase for medical care over time."

The paper, written by McClellan before he joined the Bush Administration, was published in a special issue of Health Affairs on the value of medical innovation (³ Also see "NPC Study Says New Drugs Offset Nondrug Spending: Will CBO Agree?" - Pink Sheet, 24 September, 2001., p. 6).

The economic arguments in support of innovation are at the heart of the Milken Institute's approach to the topic. The CAMS action plan cites Milken Institute economists Kevin Murphy and Robert Topel (University of Chicago), who calculate that the economic value of eliminating deaths from heart disease would be $48 trillion.

McClellan's contributions to the CAMS action plan add an extra resonance to some of the FDA-related topics the center plans to study.

Potential research projects include encouraging use of surrogate markers. A more dramatic research question suggested in the action plan is: "should the drug-approval process be modified to require evidence of safety, but not efficacy?"

Suggestions that the 1962 FD&C Act amendments be replaced by market-driven efficacy standards have been a recurring theme in conservative critiques of FDA. After the 1994 Republican election victories, the topic was openly debated, but the more extreme ideas were not included in the 1997 FDA Modernization Act.

The CAMS proposal to study the question is the latest indication that advocates for eliminating the efficacy standard may be seeking to bring the idea back into the mainstream FDA debate.

The Wall Street Journal editorial page offers several recent examples in the context of FDA's review of the AstraZeneca oncologic agent Iressa . In one article, entitled "FDA to Patients: Drop Dead," the Journal argued that, "particularly in cases of terminal disease, any safe drug with even a 'hint' of effectiveness should be brought to market as quickly as possible."

The editorialist for the Journal, Robert Pollock, was a finalist for a Pulitzer Prize in 2003. The Pulitzer committee cited his "clear and compelling editorials on FDA's delay in approval of new cancer drugs" (⁴ 'The Pink Sheet' Dec. 16, 2002, In Brief).

Defenders of the current regulatory standards appear concerned that there may be growing support for the idea of rolling back the efficacy standard.

During the Food & Drug Law Institute annual educational conference April 2 in Washington, D.C. former FDA Bureau of Drugs Director Richard Crout, MD (now president of Crout Consulting) discussed the emergence of "evidence-based medicine" as one of the positive "unintended consequences" of the 1962 amendments.

The statutory standard for "adequate and well-controlled trials" was added "with minimal discussion, almost as an afterthought, as a good thing to do," Crout noted. "It gave FDA the ability to reject drugs lacking in efficacy summarily, by summary judgment, without a hearing. That was upheld by the Supreme Court."

"It forced the drug industry to fund clinical trials," Crout added. "Today, all of medicine is influenced by a whole new enterprise called evidence-based medicine." The standard set by the 1962 act prompted a "revolution in medicine that the Congress, I submit, absolutely had no idea it created," Crout continued.

Other potential research topics for CAMS focus less on FDA and more on intellectual property topics that could influence the Waxman/Hatch reform debate, or, farther down the line, a potential framework for generic biologics.

Possible research questions include: "Will extending patent lives accelerate science?" and, "How can the regulatory process be streamlined while protecting patents?"

CAMS will also look at research incentives more broadly, including tax policy and ideas to "encourage" countries other than the U.S. to "bear more of the burden" of "the global costs for prescription drugs, medical research and drug development."

Milken Institute Center To Study FDA Approval Process, Innovation Strategies

More from Archive

More from Pink Sheet