Susan Manzi, Lupus Foundation of America
On April 3, 2009, public testimony on comparative effectiveness
research was given at a meeting of the National Advisory Council
for Healthcare Research and Quality. The testimony represents
the views of the presenter and not necessarily those of the Agency
for Healthcare Research and Quality (AHRQ) or the Department
of Health and Human Services (HHS).
The Council provides advice and recommendations to the Director,
AHRQ, and to the Secretary, HHS, on priorities for a national
health services research agenda.
April 1, 2009
Carolyn M. Clancy, MD
Agency for Healthcare Research and Quality
540 Gaither Road, Suite 2000
Rockville, MD 20850
Dear Dr. Clancy:
On behalf of the Lupus Foundation of America, I am writing to comment on the Comparative Effectiveness Research (CER) program at the Agency for Healthcare Research and Quality. We have particular concerns about the impact of CER on the treatment recommendations for individuals with lupus.
Lupus is a prototypical autoimmune disease that affects virtually every major organ system in the body including the skin, joints, heart, kidneys, lungs, blood and brain. The treatments presently used to treat the disease, i.e., chemotherapies, glucocorticoids and anti-virals, cause side effects that may be worse than the disease itself. These side effects include bone loss and osteoporosis leading to joint replacements, loss of fertility, organ transplant, infections and more. The chemotherapy which is said to be the "standard of care" for lupus kidney disease, which affects more than 30% of lupus patients, has never been approved by the FDA and carries with it short and long term side effects that are well known and devastating to people with lupus.
The last drug approved by the FDA for lupus occurred during the Eisenhower Administration - 50 years ago! The federal investment in lupus research has not been commensurate with the prevalence, incidence and impact of the disease. While we support increased research to find the causes of lupus to hasten the day when safe, effective and tolerable treatments for the disease can be developed, the CER legislative language appears to focus on short-term cost implications rather than what is the best treatment for individuals over their lifetimes.
In lupus, that is especially important, since young women in their child-bearing years are most at risk for developing the disease. In addition, the drugs that presently exist to treat lupus, while they may reduce symptoms, have side effects that cause other serious conditions which profoundly diminish the quality of life for most lupus patients.
We urge you to consider the unique medical needs of people with lupus and the need to incorporate into any analysis the effects of life-long treatment. In lupus, it is our hope that newer drugs in development will demonstrate fewer side effects than the drugs presently prescribed and we believe that even if these therapies are equally effective, they should be seen as superior to the older, less tolerable treatments.
For example, less expensive drugs, like steroids (glucocorticoids), are presently used in the treatment of lupus, usually at high doses for prolonged periods of time. The harsh side effects over a lifetime of use include damaged joints, serious infections and more. The result - costs are initially low but as hips, knees and other joints require replacement surgeries, the costs dramatically rise. This is but one example of the intolerable situation people with lupus endure. Comparative effectiveness research on many chronic diseases should clearly include quantification of the long term effects of treatment on an individual's quality of life and the burden associated with other conditions that develop as a result of the treatment. Diseases such as lupus which are systemic and have effects that vary significantly from patient to patient should be placed in a special classification or we run the risk of lowering costs in the short term but greatly increasing healthcare costs in the long term.
In addition, we respectfully would like to nominate Susan Manzi, MD, MPH, who is an LFA Board Member and lupus expert to serve on future councils or committees regarding CER. Dr. Manzi can be reached at:
Susan Manzi, MD, MPH, Director Lupus Center of Excellence
University of Pittsburgh Schools of the Health Sciences
S722 Biomedical Science Tower
3500 Terrace Street, Pittsburgh, PA 15261
We very much appreciate your attention to our concerns, and thank you for considering our recommendations. Please feel to contact me or Julie Venners who directs our Government Relations Department at email@example.com, or 202- 349-1150.
Sandra C. Raymond
President & CEO
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