The big question is whether investment in CER will steer providers and patients to more effective care and reduce inappropriate
expenditures. Instead of drug-to-drug cost assessments, pharma companies want CER to compare medicines to surgery or other
treatments, and assess the value of disease management, care coordination, and benefit design. Focusing on drugs and devices
"misses the point," according to Gail Wilensky of Project HOPE. "The real explosion in costs is in medical procedures."
However, pharmaceuticals are an easy CER target because so much drug data is available from clinical trials, outcomes studies,
and adverse-event reports. The clamor for information on pharmaceutical value and quality ensures that more studies will be
conducted in this area. AHRQ's research program began in 2005 with an emphasis on pharmaceuticals to support the then-new
Medicare drug benefit, along with instructions from Congress not to use CE information in making coverage decisions. But at
an AHRQ advisory meeting, Anthony Wisniewski, head of health policy for the US Chamber of Commerce, said that it is unrealistic
to ignore cost-effectiveness issues in CER. Analysis of costs should not be the first priority, he noted, but "it must remain
a critical component" of CER activity.
If the past is prologue, costs will indeed be a regular CER component. Several large, multi-center trials sponsored in recent
years by NIH have made headlines by concluding that newer, more costly drugs for blood pressure control and schizophrenia
treatment, (among others) may not perform any better than older, cheaper medicines. All eyes (pun intended) are now on the
National Eye Institute's assessment of treatment for age-related macular degeneration (AMD) with Lucentis (ranibizumab) or
Avastin (bevacizumab). Both drugs (produced by Genentech) derive from the same monoclonal antibody. However, Avastin is less
than one-tenth the cost of Lucentis.
The Future of CER
Analysis of drug effectiveness and cost-effectiveness is nothing new for insurers and payers. Blue Cross and Blue Shield Association's
Technology Evaluation Center has reviewed clinical evidence since 1985 to determine the effectiveness of drugs and medical
technologies. The Drug Effectiveness Review Project (DERP) at the Oregon Health and Science University provides comparative
efficacy and safety information on high-cost, heavily used medicines, as well as drugs frequently used off-label. Consumer Reports' Best Buy Drugs program uses the DERP analyses to provide advice on medical appropriateness and affordability of widely used
Foreign CER programs have spurred interest in US initiatives. Most prominent is the National Institute for Health and Clinical
Excellence (NICE) in the United Kingdom, which provides Britain's National Health Service with recommendations on the coverage
of new drugs and diagnostics. NICE sets a clear cost-effectiveness threshold that has led to controversial no-coverage decisions
on a number of new, but costly, therapies.
Pharma companies have also been sponsoring more of their own comparative studies to meet regulatory and reimbursement requirements.
Payers and formulary committees increasingly want to see data indicating superior efficacy or safety of new drugs compared
to available treatments. There are more postapproval comparator studies to meet FDA requirements, and comparative clinical
information is increasingly important for gaining market access, particularly in crowded therapeutic classes or for new products
that raise serious safety issues. There, of course, sponsors write the protocols, select the comparators, and control the
The future promises more oversight of industry studies, along with more CER. Pharma is lobbying hard for such research to
focus on clinical value and outcomes, as opposed to cost-effectiveness. Drug and device makers have enlisted support from
patient and medical groups to form the Partnership to Improve Patient Care, which has signed up former Congressman Tony Coelho
to carry the banner for ensuring that CE studies are well-designed, promote continued medical innovation, and protect patient
access to "advanced treatment options." The National Pharmaceutical Council has narrowed its agenda to focus on CER issues
and plans a mid-June symposium on the CER landscape in the US and abroad.
Policymakers insist that CER will not lead to coverage denials, but will steer doctors and providers to treatments offering
greater benefits for patients and will support flexibility to address special needs. According to Wilensky, the important
questions are whether CE data has credibility, whether research practices are open and transparent, and whether studies are
objective and not politically motivated.
One positive result of a broader CER program is to develop standards and improve trial designs for real-world assessment of
medical treatments. AHRQ plans more investment in methods for designing CER studies, particularly those involving under-represented
populations, and is holding a symposium June 1-2 on the subject. NIH is offering grants to improve CER research practices,
assess quality of life measures and establish patient registries. At the IOM public meeting, Bryan Luce, senior vice president
of UBC, urged "true transformational thinking" in designing CE research studies; otherwise, he said, we will "waste vast amounts
of money answering the wrong questions, or the right questions too late."
Now NIH is proposing a number of studies addressing pharmaceutical efficacy and costs for ARRA-funded challenge grants. Research
officials would like to assess costs along with risks and benefits of commonly used cancer treatments, and compare those costs
and other factors for treating autoimmune rheumatic and skin diseases. The NIH "wish list" includes comparisons of efficacy,
outcomes and costs of treatments for kidney stones, for fibromyalgia and for patients newly diagnosed with type 2 diabetes.
Interest is high in the IOM committee's deliberations, as dozens lined up at a March public meeting to propose CER study methods
and topics to explore. Similarly, there was standing-room-only at a meeting of AHRQ's National Advisory Council, which heard
presentations on how the agency should invest its ARRA funds.
Jill Wechsler is Pharmaceutical Executive's Washington correspondent. She can be reached at firstname.lastname@example.org