This is the tenth 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.
Perry Cohen: I am a patient advocate who was diagnosed with Parkinson's Disease (PD) in 1996. My advocacy has focused primarily on the role of persons with Parkinson's (PwP) in clinical research and the regulatory evaluation of new PD therapies, but I generalize my advocacy perspectives as they apply to all serious chronic illnesses.
In the spring of 2000, I started serving as a patient representative for the FDA on the Deep Brain Stimulation Advisory Council, and in 2001, I founded the Parkinson's Pipeline Project to develop and support roles for PwP to facilitate approval of new therapies. The pipeline project maintains a tracking database with information on all new therapies that have entered clinical phases of approval and has collaborated with FDA staff on training and informing FDA-PD consultants to provide the patient view.
I am a member of the National Working Group on Evidence-Based Health Care and just finished a two-year stint on the stakeholder advisory group for AHRQ. I was also appointed as a patient representative on the planning committee for PCORI's patient engagement workshop at the end of October, 2012.
I have also participated as a subject in nearly 2 dozen different studies and demonstrations including one shot observations, multi-year longitudinal studies and controlled clinical trials.
Gruman: From your perspective, what constitutes comparative effectiveness research (CER)?
Perry Cohen: CER generally refers to comparing one treatment to another based on a synthesis of the research literature. It is a narrow technical exercise aimed at compiling all of the evidence available for a specific therapy. What is most relevant is how to make a decision under conditions of uncertainty. CER may add new kinds of information to the process, but uncertainty always remains.
A number of years ago, the National Working Group on Evidence-Based Health Care decided to work on getting patients to the table at all stages of the research process. Patient-centered outcomes research takes this a step further, saying that patients' interests should come first. I think this is a gesture in the right direction but would note that patients don't always know how their interests differ from those of other constituencies (clinical scientists, regulators, sponsors) in the research process.
Gruman: Well, who can we depend on to identify patient's interests then?
Perry Cohen: There are plenty of us who are smart enough to learn, and I am still learning as I encounter different situations. I observe how I react to a choice or a challenge versus how others react who are PwP or not. Inevitably, most PwP react similarly. For example, patient advocacy organizations represent patient interests, but they also represent the interests of scientists who conduct the research or other organizations in the community who raise the funds. At the end of the day its PwP ourselves and our close family care partners who you can count on to identify patient interests.
It's incumbent on us to speak up and represent at least our own experience. I've been to many meetings in Washington DC where people claim they represent patients' interest but they actually are representing their own interests that overlap with patients' interests. As a patient with a serious illness, I observed that there were often no other people in the room willing to speak up for the patients' interests independent of the other groups. My motto became 'the missing ingredient in the development of new therapies is the voice of the patient.'
Researchers say they understand and represent patient interests, and it's true that sometimes theirs and ours are aligned, but by no means are they always!
The pharmaceutical industry says it represents patients' interests, and their interests often overlap with ours except when it comes to price. Unfortunately, we pay the price and it's often inflated.
It's the same with regulators to some extent: we and they are interested in safety. But regulators want to go slowly and we patients die while we are waiting for new medicines to make their way through the approval process.
The risk/benefit ratio is different for people with severe chronic disease than it is for those with less-severe diseases and conditions. General patient advocacy and consumer advocacy doesn't represent my interests. Those of us with serious advanced disease evaluate risk differently, i.e., we are willing to take bigger risks because our alternative is the almost certainty that our disease will continue.
Gruman: Can you give me an example of how you and your constituents have been affected by CER?
Perry Cohen: We can see the impact of CER in decisions that health plans make about what's on their drug formulary. I was taking a drug that cost my insurance $11 per pill. My health plan dropped it from the formulary, so now I take the generic that is still expensive, just not quite as expensive.
As a health services researcher I think you have to look at cost. As a patient, I don't mind taking the generic, but I recently came across an article that noted a 10-fold increase in all cancer mortality in those who take the generic. Now what do I do?
We are concerned with getting the best medicine at the same time we are trying to control costs of care. This article gave me pause.
Gruman: How do you see the increased attention to CER affecting the care your constituents will receive in the future?
Perry Cohen: I believe that CER will improve the quality of health care and that better quality care is more cost effective. I think there is a role for patients in here. We have to take responsibility for whether we spend $11 of our own money for a pill not on the formulary or just pay the co-pay for the generic.
Gruman: Some professionals believe that patient groups are opposed to comparative effectiveness research. Do you think this is accurate?
Perry Cohen: I think most patient groups don't really get what CER is. Given this lack of understanding, yes, for many, their fears outweigh their hopes.
Gruman: What do you think the public's opinion is about CER?
Perry Cohen: The public doesn't understand what it is. Further, I think that the public expects that doctors already know which treatments are better ' that's why they are recommending them.
The truth is that evidence is not often unequivocal. For the most part, it is primarily specialists that keep up with all the evidence. Too much of the evidence comes from commercial sources; the standards for quality of the evidence are limited in their applicability; risk-benefit tradeoffs are highly individualized and situational. Informed patients often know as much as physicians about the evidence, and their knowledge comes from different sources'1st hand from daily living with the condition and participation in clinical research.
Gruman: What are your fears and hopes for CER?
Perry Cohen: It would be nice to know if a treatment is totally worthless or that there are truly horrible side effects to a treatment or that a treatment really won't help slow the disease or relieve the symptom it's aimed at.
I've heard experts suggest that one of the barriers to higher quality health care is the gap between knowing and doing. I believe that the function of CER is to try to fill in parts of that know-do gap. It is a critical operational component of the rational, efficient health care delivery system that we need.
My fear is that CER will become a heavy handed, green eye shade tool for arbitrary cuts in service, based on equivocal information without regard to individual differences in response to medicine.
My hope is that the same amount of energy and creativity that has gone into advancing biomedical science for better treatments will be devoted to the management of this innovation. Information and communication technology provide new sources of data to capture patient observations, to compile the data from electronic medical records and personal health records and deliver comparative data to the point of service to aid individuals' choices on treatment options.
More CER Interviews by Jessie Gruman