COMPARATIVE EFFECTIVENESS RESEARCH: ANGELA OSTROM OF THE EPILEPSY FOUNDATION OF AMERICA

By Jessie Gruman

April 2, 2014

This is the twelfth 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.

Angela Ostrom: The Epilepsy Foundation of America® is a national voluntary agency dedicated solely to the welfare of the almost 3 million people with epilepsy in the U.S. and their families. We work to ensure that people with seizures are able to participate in all life experiences; to improve how people with epilepsy are perceived, accepted and valued in society; and to promote research for a cure. In addition to our national programs, we serve epilepsy clients throughout the United States through 48 Epilepsy Foundation affiliates around the country.
I am the senior director of government relations in the Foundation’s Legal and Government Affairs Department.

Gruman: Where do you see comparative effectiveness research (CER) fitting in the effort to improve the effectiveness of health care?

Angela Ostrom: Nationally, the Epilepsy Foundation has supported comparative effectiveness research that seeks to integrate patient, consumer and clinician perspectives. We advocated for the creation of PCORI as an independent body within the health care reform law.
Through PCORI, we support a CER enterprise that we trust will include patients and physicians in evidence-based/CER policies and decisions. We hope that it will maintain transparency in discussion, research and priorities. And we depend on it to facilitate clear communications to policymakers and the public about the importance of and appropriate use of evidence in public policy decisions.

Gruman: Tell me how your organization views the relationship between CER and patient-centered outcomes research (PCOR).

Angela Ostrom: CER is comparative clinical research that can be limited by available studies or literature reviews.In contrast, we view PCOR as an effort to integrate patients, consumers and clinicians in conducting real-world research (such as clinical experience, RCTs, observational studies and systematic reviews) that takes into account patient outcomes and quality of life while resisting the urge to create a “one size fits all” medical decision for all health conditions.

Gruman: Can you give me an example of how your constituents have been affected by CER?

Angela Ostrom: Here is an example of how CER is notPCOR that illustrates the comparative advantages of the latter:In response to a request and recommendations from the Epilepsy Foundation, the Agency for Healthcare Research & Quality (AHRQ) commissioned a report designed to answer some broad key questions on anti-epilepsy medications. However, the approach of the report posed questions that the Foundation, along with other epilepsy patient and provider organizations, saw as contributing little value to patients and providers.

Nevertheless, the report moved forward and while some of the experts recommended by the patient groups were consulted by the lead researcher (who was not a neurologist or an epilepsy expert) they were not involved in the report beyond one-shot interviews. There was no overall discussion of the research project, no interaction among experts and no feedback or discussion among the group that included the patients and caregivers. In essence, the “key informants” who appear to endorse the report had no input in the drafting, discussion, conclusion or recommendations.

This kind of engagement of patients/consumers/stakeholders offers no value. While independence and non-bias is important, it should not take precedence over involving the key patient, research, and provider organizations in discussion and analysis of research and clinical recommendations. We have heard from the field that insurers used this report to deny access to physician prescribed treatments; this is CER gone wrong.

Stakeholder and patient engagement must be meaningful. One big challenge for PCORI will be to enforce such involvement. Clearly, this will not be easy.

Gruman: How do you see the increased attention to CER affecting the care your constituents will receive in the future?

Angela Ostrom: For the epilepsy community, one of our priorities for the research sponsored by PCORI is that it will provide information on different health interventions and how those health interventions affect individual patients. If appropriately defined and implemented, PCOR can enrich our understanding of these variations, rather than ignoring them by focusing on population averages that mean little for any individual patient or subgroup.

Gruman: Some professionals believe that patients are opposed to comparative effectiveness research. Do you think this is accurate? What do you think is behind this view?

Angela Ostrom: It may be accurate for some patients and patient groups but not for the Epilepsy Foundation. I think, however, that some of our concerns about how CER might be misused and thus injure or restrict people’s access to care for epilepsy could leave some with the impression that we oppose it.Epilepsy is a complex disease. An optimal quality of life and seizure control for the person with epilepsy – so that they can be a fully productive member of society – is our goal.Our main concern about CER and our constituents is that one treatment may provide a high quality of life with seizure control and few side effects for many but not for all patients. Another treatment may appear to be equally effective but may have unacceptable side effects for a specific individual, or not be appropriate. For example, a woman with epilepsy should not be on certain medications if she is of child bearing age.The Foundation has long encouraged government and private entities to support clinical studies on epilepsy treatments that focus on efficacy, patient safety and to consider treatment risks and benefits. Given all of the choices of treatment available to a physician, comparative effectiveness studies can provide critical information on which treatment approach will be most effective compared to the others available. This research should be used to assist the provider and patient with making treatment decisions, not make the decision for a physician or be used to deny the treatment that has worked best for an individual.

Gruman: What are your fears and hopes for CER?

Angela Ostrom: Our hope is that CER will be used as a useful tool that helps doctors and patients make good treatment decisions and reach the best possible outcomes based on the individual’s needs and medical condition.Our fear is that pharmacy benefit managers and health plans will use CER recommendations based on research that is insensitive to patient differences, that is limited by the scope of the question or the patient population as the basis for restricting treatment options and access to medication. We saw this with the AHRQ report I described earlier and hear reports of this happening from our constituents.

We are also concerned that limiting access to care in this way cuts off the potential of observing differential effect of medications over time and may limit research on medical devices.

Original blog post by Jessie Gruman. Updated by the GW Cancer Institute June 2016.