Comparative Research Poses Challenges - Pharmaceutical Technology

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Comparative Research Poses Challenges
A new center may provide evidence for improving care, but could discourage coverage of treatments.


Pharmaceutical Technology
pp. 32-40


Jill Wechsler
There is great optimism throughout the healthcare community that comparative-effectiveness research (CER) will enhance the nation's healthcare system and curb unwarranted spending. To this end, the US federal government is poised to invest some $500 million a year on research for how to prevent, diagnose, treat, monitor and manage disease, as authorized by the Affordable Care Act (ACA) enacted last March.

Although Republicans initially supported comparative research as a way to improve healthcare decision-making, last year some conservative groups raised the specter of rationing and "death squads" in an effort to end healthcare reform. Even among CER backers, there is concern that the initative could limit access to certain treatments and skepticism about just how big the savings will be. Pharmaceutical and medical product companies, as well as some patient groups, are wary that government-funded CER will steer health coverage toward low-cost remedies, and away from more expensive new products often accused of driving up healthcare costs. Personalized medicine advocates, moreover, are pressing for CER to consider treatment effects on patient subpopulations, including minorities, children, and individuals with uncommon health problems—as opposed to researching what works best in the average patient. Yet, designing studies able to detect such differences is complicated and may increase the scope and cost of research.

The government met a Sept. 23, 2010 deadline for naming a board of governors for the new Patient-Centered Outcomes Research Institute (PCORI). The board now must set the national CER agenda, develop systems for funding research, establish standards and methods for CER studies, and support programs to disseminate results to practitioners and the public. Gail Wilensky, senior fellow at Project Hope and admittedly an outspoken advocate for CER, says she's "cautiously optimistic" about the progress so far, but acknowledged at a recent comparative-effectiveness summit in Washington, DC, that the program remains a "very fragile concept" that "a lot of people still want to torpedo."

Stimulating CER

The CER bandwagon has been rolling for several years under the guise of "evidence-based medicine," "health technology assessment," and "relative effectiveness" research. All promote the use of rigorous, unbiased assessments of alternative treatments as a way to identify the most effective medical therapies and practices, as well as those that are inappropriate or even harmful (such as routine use of estrogen replacement therapy for post-menopausal women).

To date, most CER initiatives focus on the comparative costs and effectiveness of new drugs and medical technology. The United Kingdom's National Institute for Health and Clinical Excellence (NICE) is recognized worldwide for its recommendations—pro and con—on new drug coverage. Blue Cross/Blue Shield insurance companies have supported technology assessments of drugs for more than a decade. The University of Oregon-based Drug Effectiveness Review Project (DERP) evaluates drugs for state Medicaid programs and other payers. The Veterans Administration's vast database supports comparisons of therapies and treatments such as oral diabetes medications and care for patients with acute kidney injury.

With the establishment of the Medicare drug benefit in 2003, Congress provided more support for this field by expanding the Effective Healthcare Program at the Agency for Healthcare Research and Quality (AHRQ). The agency has received nearly $150 million for CER during the past five years to solicit systematic reviews and literature syntheses related to prevalent conditions affecting Medicare beneficiaries.

The federal stimulus legislation enacted in 2009 (American Recovery and Reinvestment Act, or ARRA) dramatically advanced federal funding of CER by providing $1.1 billion for the Department of Health and Human Services (HHS) to set priorities for and support comparative studies. Much of the money went to AHRQ and the National Institutes of Health (NIH) to provide for CER projects and infrastructure development.

This year's healthcare-reform legislation built on ARRA by establishing PCORI as an independent, nonprofit organization with resources to support clinical trials and outcomes studies on effective treatments for common medical conditions. By 2014, this nongovernmental institution is slated to have a $500–600 million annual budget, funded largely by a 1% tax on health insurance premiums—a strategy designed to insulate the program from the highly political annual Congressional appropriations process and provide more stability and predictability.


In Washington this Month
PCORI's 21 board members, who were named by the Government Accountability Office (GAO) in September, represent payers, providers, patients and industry, with an emphasis on women and minorities. NIH Director Francis Collins and AHRQ Chief Carolyn Clancy are on the panel, but do not chair it; that honor goes to University of California at Los Angeles Vice-Chancellor and Dean Eugene Washington. Steven Lipstein, president of the nonprofit BJC Healthcare system, was named vice-chair. The industry representatives on the board include Pfizer's chief medical officer and senior vice-president, Freda Lewis-Hall; Johnson & Johnson's head of medical devices and diagnostics, Harlan Weisman; and Medtronics senior-vice president, Richard Kuntz.

"This is not the list of usual suspects," said National Pharmaceutical Council President Dan Leonard regarding the PCORI board assignments. All members are highly qualified and reflect diverse backgrounds, but have not been regulars on the CER circuit.

PCORI is expected to award many CER grants through NIH and AHRQ to take advantage of existing peer-review and research infrastructure. But before it can start funding research, the board has to hire a staff, locate offices, and issue a charter.


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