Moderator Margaret Anderson, COO, FasterCures/The Center for Accelerating Medical Solutions
Panelists:
Jeffrey Chamberlain, Professor, Neurology, Medicine and Biochemistry, University of Washington (glasses)
Director, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center
David Meltzer, Associate Professor of Medicine, School of Medicine, University of Chicago
We all want faster cures for diseases but what is the thought process in determining which research studies are funded? This panel looked at muscular dystrophy as an example in terms of evaluating the larger problem of medical research and rising costs. How do we know which studies should be done and how can researchers present their case in terms of getting the most funding for the greater good?
Jeffrey Chamberlain: Muscular Dystrophy Therapies
What are muscular dystrophies? There are 20 different types of this disease which causes muscle weaknesses. The disease is genetic which means that it often causes concerns for families. There are 500,000 to 1,000,000 cases worldwide (mainly in males) making it the most common genetically inherited disease that involves a gene mutation but at only 500 births per year it is still considered a rare disease. There are not a lot of treatment strategies for this disease currently. Current methods being studied include gene therapy, stem cell therapy and drug treatments. Drug therapies tend to move along most quickly. One gene therapy technique on 10 or 12 people has been relatively successful but it also caused leukemia in two patients. Chamberlain believes that, "the pharmaceuticals are going to have the biggest impact in the short term" but that "stem cell therapies have the greatest long term potential."
David Meltzer: Medical Research Spending DecisionsMeltzer works on applying economical models to medical research spending.
Health care costs have been on the rise. Since 1960, health care spending has grown by 2.5% more per year than the rest of the economy. The price of treating a heart attack in the U.S. has gone up four percent because the quality of care has gone up. Health care costs are rising because we are able to do more. How do you assign a value to health benefits? The question for research studies is often is it worth it? Do the cost and effectiveness balance out? What is the dollar value versus life saved/quality of life? Where do you draw the line? A study of pap smears found that having them once every three years is nearly as effective and costs far less over the course of a lifetime (several thousand dollars versus over $800,000).
What is a year of life worth? A study found that people place a value of $50,000 to $160,000 on a year of life. What is the value of research in a narrower area such as a rare illness? Most people with muscular dystrophy die by age 30 and many by age 20. The total of a cure for MD is $38 billion, combining both prevention and cure over the long term.
The value of the economic modeling is in applications for grants and research funding. Technology assessment will be an increasing part of getting research funds for studies. The NIH is investing $20 million per year for muscular dystrophy. This same type of economic model when applied to more prevalent diseases has all sorts of ramifications for determining the value of certain treatments and prevention techniques. What is the value of cure versus prevention? In terms of muscular dystrophy, prevention is further along than potential cures currently and also offers the greater dollar value in terms of the research done by Meltzer. This will vary depending on the disease studied.
