ISSUE BRIEF
Definition
Comparative Effectiveness Research (CER) is the process utilized to evaluate and assess the effectiveness of clinical services and therapies for a given medical condition. The intent of CER is to assess the effectiveness of various clinical services, interventions and therapies for a given disease to determine what works best. According to the Agency for Healthcare Quality and Research (AHRQ), CER usually compares two or more types of treatment, surgeries or medical procedures allowing patients and clinicians to review research results and make informed health care decisions. The limited availability of data makes it difficult to understand how different medical interventions to treat a given medical condition compare.
Community Impact
The Obama Administration suggests the “keys to eliminating waste and missed opportunities is to increase our investment in comparative effectiveness reviews and research”. Congressional leaders continue to express a need for more medical evidence to guide healthcare decision-making. CER is a critical component to the reduction of skyrocketing health care cost. Accordingly, high-cost rare disorders, such as Hemophilia, should be considered when drafting CER legislation and during implementation of policies.
The Hemophilia Federation of America (HFA) believes that individuals with rare bleeding disorders deserve high quality healthcare and access to the full range of treatment options. A “one size fits all” approach to medical interventions for rare bleeding disorders results in unfavorable health outcomes. We recognize that treatment options for rare bleeding disorders are expensive, however each patient responds differently to the same treatment. What works for one patient may not work for another. To this extent, the following MUST be considered:
- Separate specialized review panels for rare diseases
- Prohibition to use CER to make coverage determinations and deny coverage
- Differences in patients, patient co-morbidities, and legitimate patient preferences
- Appropriate use of CER studies by clinicians and patients
- Establishment of a transparent and open process at each stage of CER planning and implementation
- Participation of ALL stakeholders in each stage of CER
CER, when used appropriately can provide significant advantages:
- Reduction in health care spending and substantial cost savings
- Generation of essential information about cost and benefits
- Improvement in health care quality and outcomes
Education, active involvement and collaborative efforts are critical during discussion of CER program development, design and implementation. The HFA continues to work with the National Organization for Rare Disorders (NORD), the Alpha-1 Foundation and other stakeholder groups to advocate for an “expert advisory panel for rare diseases,” when conducting CER studies on rare diseases. Additionally, we support designation of a “Rare Disease Ombudsman” to serve as the single point of contact to patients with rare diseases. The HFA will continue to work together with legislators, rare and chronic disorder advocacy groups, and other stakeholders to ensure that CER legislation and policies do not impede access to clinical services and the full range of treatment options.
Legislation
111th Congress:
- Patient-Centered Outcomes Research Act of 2009 (S.1213)
- Comparative Effectiveness Research Act of 2009 (H.R. 2502)
110th Congress:
- Comparative Effectiveness Research Act of 2008 (S.3408)
- Children’s Health and Medicare Protection ACT (H.R. 3162, Section 904)
- Enhanced Health Care Value for All Act (H.R. 2184)
- Medicare Prescription Drug Price Negotiation Act (S. 3, Section 4&5)
The American Recovery and Reinvestment Act, signed into law on February 17, 2009 included a provision that would distribute $1.1 billion between the Agency for Health Care Research and Quality, National Institutes of Health, and Department of Health and Human Services. The funding was provided “to accelerate the development and dissemination of research assessing the comparative effectiveness of health care treatments and strategies”. The provision calls for development of a database available to the public, a federal coordinating council, and an Institute of Medicine (IOM) report to establish national CER priorities. The IOM report can be found at http://www.iom.edu/CMS/3809/63608/71025.aspx.
Comparative Effectiveness Research (CER) Resources
For more information on CER and a list of sources for this brief, please visit the following links:
Advanced Medical Technology Association
AHIP Center for Policy and Research
AHRQ Effective Health Care Program
Alliance for Better Health Care
American Recovery and Reinvestment Act
BCBS Office of Policy and Representation
Coalition for Health Services Research
Consumer Reports Best Buy Drugs
National Institute for Health and Clinical Excellence (UK)
Oregon Evidence-Based Practice Center
