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December 7, 2007

Dear Chairmen and Ranking Members:

We are writing to ask that as you consider comparative effectiveness research within the context of the upcoming Medicare package you keep the needs of patients, health care providers and other health specialists at the forefront of the discussion. Any provisions related to comparative effectiveness research should:

• be conducted through an open and transparent process that allows for all stakeholders to participate equally in governance and input, starting from the research planning stage;
• design studies and communicate results in ways that reflect variation in individual patient needs and help patient and doctors make informed health care decisions, not encourage a "one-size-fits-all" approach;
• address research questions that matter to health care providers and patients by examining all aspects of health care that affect patient outcomes - including care management and processes, benefit designs, and medical interventions;
• recognize the individual needs of patients and advance the goals of personalized medicine, particularly for minorities and vulnerable populations;
• recognize the unique nature - and value - of targeted therapies that benefit specific groups of patients with rare and life-threatening diseases; and
• examine the clinical value of the treatment intervention to the patient, not cost-effectiveness.

Comparative effectiveness research typically compares average results of one therapy versus another for a study population. These results do not take into account differences between patients due to genetics, co-morbidities and other factors. They may not even reflect gender and age differences. For example, just last week it was reported that the formula used for the National Cancer Institute's Breast Cancer Risk Assessment Tool often underestimates the risk of cancer in older African-American women. The original formula was based on data from about 240,000 Caucasian women. Comparative effectiveness research also rarely takes into account differences in patient preferences, including tolerance for side effects and different willingness to balance risk and benefits. In the end, decisions about the most appropriate care for individuals should be made by patients and their doctors.

Comparative effectiveness research should focus on questions that reflect the interactions among all of the various components of the health care system and have the greatest potential to empower medical specialists and patients to make the most appropriate decisions when faced with "real world" clinical situations. Health system factors such as care coordination strategies and health plan coverage policies and financial incentives play a larger role in determining whether patients receive the care supported by clinical evidence. It is important that these practices also be based on solid evidence and therefore be included in the research agenda on an equal footing with clinical interventions; otherwise, as at present, care indicated by strong evidence may not reach patients.

We are also concerned that the legislation might not only allow comparative effectiveness research but "cost-effectiveness" research. By definition, cost-effectiveness research places a dollar value on a human life and could result in therapeutically inferior treatments being judged as the preferred treatment due to cost, and in the case of rare diseases limiting access to any treatment.

Patients deserve the medical care they and their doctors think is best for them. We look forward to working with you to ensure that all patients continue to have access to lifesaving medications and the tools necessary to advance a better quality of life for all Americans. Thank you for your consideration.

Sincerely,

Abigail Alliance for Better Access to Developmental Drug
Alliance for Aging Research
Alpha-1 Association
Alstrom Syndrome International Alzheimer's Association
American Academy of Physician Assistants
American Association of Neurological Surgeons
American Association of Occupational Health Nurses
American Association of Orthopedic Surgeons
American Association of People with Disabilities
American Autoimmune Related Diseases
Association American Brain Coalition
American Pain Foundation
American Psychiatric Association
American Public Health Association
American Sickle Cell Anemia Association
American Society for Therapeutic Radiology & Oncology
American Urological Association
APS Foundation of America
Association of American Medical Colleges
C3: Colorectal Cancer Coalition
Coalition of Heritable Disorders of Connective Tissue
Congress of Neurological Surgeons
Cornelia de Lange Syndrome Foundation
Crohn's and Colitis Foundation of America
Cutaneous Lymphoma Foundation
Detroit Medical Reserve Corps
Dystonia Medical Research Foundation
Easter Seals
Families of Spinal Muscular Atrophy FasterCures
Foundation for Allergy & Immunology Research
Friends of Cancer Research
Genetic Alliance
Genetic Alliance BioBank
Hereditary AngioEdema Association
Histiocytosis Association of America
Huntington's Disease Society of America
Hydrocephalus Association
Incontinentia Pigmenti International Foundation
Inflammatory Breast Cancer Research Foundation
International Mosaic Down Syndrome Association
International Pemphigus & Pemphigoid Foundation
Kennedy's Disease Association
Kidney Cancer Association
Lupus Foundation of America
Lymphangiomatosis & Gorham's Disease Alliance
Marti Nelson Cancer Foundation
Mental Health America
Moebius Syndrome Foundation
Myasthenia Gravis Foundation of America
Myelodysplastic Syndromes Foundation
Myotonic Dystrophy Assistance and Awareness Support Group
Myotonic Dystrophy Foundation
National Alliance on Mental Illness
National Ataxia Foundation
National Foundation for Ectodermal Dysplasias
National Hemophilia Foundation
National Hispanic Medical Association
National Kidney Foundation
National Marfan Foundation
National Multiple Sclerosis Society
National Organization for Rare Disorders
National Tay-Sachs & Allied Diseases Association
New Jersey Association for Biomedical Research
Oregon Metabolic Disease Foundation
Parent Project Muscular Dystroph
Parkinson Pipeline Project
Parkinson's Action Network
Physician Assistant Education Association
Plasma Protein Therapeutics Association Project DOCC - Delivery of Chronic Care Pulmonary Hypertension Association PXE International
RetireSafe
Sickle Cell Disease Association of America
Sjogren's Syndrome Foundation
Society for Investigative Dermatology
Society for Women's Health Research
Sturge Weber Foundation
TII CANN - Title II Community AIDS National Network
Tourette Syndrome Association
Trimethylaminuria Foundation
United Spinal Association
VHL Family Alliance
Vietnam Veterans of America
Vital Options International
Wilson's Disease Association