This is the 23rd interview in a series between CFAH President and Founder Jessie Gruman and patient and consumer group leaders about their experiences with and attitudes toward comparative effectiveness research (CER). The full series of Jessie's past CER interviews can be found here.
Gruman: Tell me about your organization.
Louise Vetter: The Huntington’s Disease Society of America (HDSA) was founded 1968 by the widow of folk singer Woody Guthrie to bring Huntington’s’ Disease (HD) families together to champion better care and drive forward research. Originally, it was just families sitting at kitchen tables to address the fact that no one knew about this disease. The organization gave these families a voice. It helped push through the orphan drug act in the 80s and has advocated strongly for more research. HDSA is one of the oldest rare disease organizations and is the product of families coming together to acknowledge a need in the absolute absence of attention.
There are 30,000 Americans alive today with symptoms of HD, and an additional 200,000 are at risk. HD is a genetically dominant disease: If one of your parents has HD or has died from it, you have a 50-50 chance of getting it. It is one of only a few diseases where a definitive diagnostic test can tell you your risk prior to the onset of symptoms.
Gruman: Where do you see comparative effectiveness research (CER) fitting in the effort to improve the effectiveness of health care?
Louise Vetter: Generally, we see CER as an important priority to inform clinician decision making. The fears that many have that this will eliminate treatment options is well-founded, but if there is good head-to-head evidence that will inform good clinical decisions, we believe there is a positive potential for health care overall.
Gruman: Tell me how your organization views the relationship between CER and patient-centered outcomes research (PCOR).
Louise Vetter: We are excited about PCOR because it is more personalized and it takes into account quality of life issues which are so very important for HD families. What is meaningful for patients is often very different than for researchers and doctors, so looking at outcomes that bear on what really constitutes good care for patients is important.
For HD, there is currently only one FDA-approved therapeutic, and it is not disease-modifying. It treats a primary movement symptom of the condition. Other symptom management for HD, such as depression or anxiety, utilizes other non-HD specific treatments. Clinical practice is now guided mostly through an open, though largely informal, sharing of clinical expertise and clinician surveys of prescribing practices. It’s not data driven. In this context, CER is tough for HD, but we really see PCOR as an area of opportunity.
Gruman: Can you give me an example of how your constituents have been affected by CER?
Louise Vetter: Unfortunately, we have no examples at this time.
Gruman: How do you see the increased attention to CER affecting the care your constituents will receive in the future?
Louise Vetter: Our constituents will continue to be frustrated until there are therapeutic options that CER can be applied to. We are optimistic about disease-modifying treatments for HD coming online in the next 5-10 years, so ask me again then.
Gruman: Some professionals believe that patients are opposed to CER. Do you think this is accurate?
Louise Vetter: I don’t think HD families understand CER. Those who have some level of awareness are likely to think of CER as a barrier to good treatment and not as an informer of good treatment. They associate it with death panels, for example.
Gruman: What are your fears and hopes for CER?
Louise Vetter: I hope that CER is used to guide clinician decision making and not insurance coverage, and that it can be a meaningful addition to medical knowledge and not a hurdle or a barrier.
My fear is that we will get to a place where CER must be conducted in order to make treatment decisions. At that point, it would be getting in the way of good care, especially in rare diseases like HD where there are so few specific resources.