This is the 19th in a series of interviews between CFAH President and Founder Jessie Gruman and patient and consumer group leaders about their experiences with and attitudes toward comparative effectiveness research (CER).
Gruman: Tell me about yourself and your organization.
Jeff Carroll: My partner at HDBuzz, Ed Wild, and I are research scientists. I study Huntington’s Disease (HD); I am a member of an HD family and a carrier of the gene. My mom died of HD and four of my six siblings are also carriers. I have kids who are product of a genetic screening technology called “pre-implantation genetic diagnosis,” which precludes them from inheriting the mutant gene from me. So while I am not a patient per se, I am definitely a member of the HD community.
HDBuzz works to raise awareness about the scientific facts about HD. HDBuzz is a carefully curated site, designed to be of interest to HD families. It includes some basic science and clinical trials as well as what doesn’t work, what’s hyped, what’s not. We are highly motivated to help patients and people who are at risk to understand the disease and predictive testing. Most people who are at risk are not likely to be seen in clinics: there is no reason for them to go. How can you reach them?
HDBuzz is a patient organization that exists solely to provide news stories about clinical and basic research in the HD field for HD patients and those at risk for HD. We don’t provide patient care or perform other functions that are traditionally carried out by existing lay organizations. We provide an RSS feed for most HD patient organizations, including the UK HDA, Canadian HSC and American HDSA. We launched our 12th language (Chinese) in September. Our Spanish feed is very big and active. We also make use of social media: Twitter and Facebook, for example. We currently get about 110K hits to our front page per month, about 50% looking for English, 50% other languages.
Gruman: Where do you see comparative effectiveness research (CER) fitting in the effort to improve the effectiveness of health care?
Jeff Carroll: This is an issue with HD. We have no disease-modifying treatment, so we can’t afford to be picky about them. That said, there is a lot of treatment for symptoms of HD: cognitive or motor symptoms, for example. We have a poor understanding of which patients are taking what and to what effect. There is some evidence from animal models that some symptomatic treatments may be disease-modifying, but this needs to be looked at in human patients.
Gruman: Tell me how your organization views the relationship between CER and patient-centered outcomes research (PCOR).
Jeff Carroll: I don’t know, but I look forward to learning. We are in the Stone Age in HD, because we are only just now creating large, well-curated databases of HD patients, including the treatments that they’re taking. I’m sure in the Parkinson’s field they have better data. Two large HD patient databases, REGISTRY and COHORT, have recently merged to form ENROLL-HD. Hopefully this will provide a novel source for CER.
Gruman: Can you give me an example of how your constituents have been affected by CER?
Jeff Carroll: They haven’t.
Gruman: How do you see the increased attention to CER affecting the care your constituents will receive in the future?
Jeff Carroll: Just knowing more about the comparative effectiveness of symptomatic treatments would help. In large population centers, we have some centers for HD. In the U.S., many of these are designated as “Centers of Excellence” by the HDSA. Individual clinics have developed standards, but these are not always consistent from clinic to clinic. The Huntington’s Disease Society of America (HDSA) put together a physician guide that can be downloaded for free. Having the guide is one thing; but of course, getting it used is another – many HD patients see primary care doctors that are not part of an established HD clinic.
Gruman: Some professionals believe that patients are opposed to CER. Do you think this is accurate? What do you think is behind this view?
Jeff Carroll: I don’t have any sense that the patient community is resistant. Patient advocates worked with the HDSA to make their physician guide easily accessible online. Many hundreds of patients have signed up for observational studies, providing data on their treatment regimens. My sense is that patients want this to be done so that HD care improves for themselves and for their affected family members.
Gruman: What are your fears and hopes for CER?
Jeff Carroll: As a community, our focus is on the discovery of disease-modifying treatments. This is the burning desire of everyone in the community. Getting there involves early stages, like which clinical trials do I volunteer for, how do can patients be convinced to volunteer their time for the right clinical trial. Right now, anyone can recruit for trials if they have FDA approval. If things heat up, there will be more trials, more competition for patients and people will really need guidelines. Investigating which treatments work best for HD symptoms, however, is something we could do more comprehensively now, while we wait for the initiation of more disease-modifying trials.