In this interview, Eleanor Perfetto (National Health Council, DC, USA) discusses her presentation from ISPOR (18–22 May, New Orleans, LA, USA) on the importance of patient engagement.
Please could you introduce yourself and your institution?
I am the Executive Vice President for Strategic Initiatives at the National Health Council (DC, USA); we are a not-for-profit, membership organization predominantly focused on the patient voice, be that in health policy, value assessment or clinical outcome assessment. Our membership predominantly includes patient advocacy organizations; we have 60 such organizations in our membership, including the American Heart Association, American Diabetes Association and American Cancer Society. Other membership categories include business and industry, including pharmaceutical and health insurance companies, other types of non-profits, professional and trade associations, and affiliates, which includes vendors who typically provide services to our membership.
In the USA, the patient advocacy movement seems to be a lot bigger than it is in the UK. Why do you think that may be?
It’s hard to say; I think, in part, it has to do with the long history of patient advocacy in the USA. For example, the National Health Council was founded in 1920. One of the things that we were created for, and developed over the years to do, is to strengthen patient advocacy organizations. A lot of our work involves supporting, guiding and educating our members, to help them be better at what they do.
I also think the U.S. is a very vocal population; when something happens, they organize, become activated, vocal and tap into all the resources that they can to get attention for their issue.
“…we are potentially making an access decision based on an incorrect endpoint.”
Your talk focuses on the controversial quality-adjusted life year (QALY) measurement. Why is this controversial?
Health economists and researchers are used to thinking about, and dealing with, the QALY; it is a common tool for them, and they know and recognize its limitations. They try to find ways around these limitations, to reduce them and the inherent flaws, but, ultimately, they advocate for the QALY as a standard for value assessment.
The patient community, on the other hand, is concerned by the use of the QALY to make critical decisions about health care access when we are so aware of its inherent flaws and limitations, particularly its typical lack of consideration of patient input.
Outcomes from clinical trials, for example, which have often been incorporated into the QALY calculation, may not be the outcomes that patients care about. Therefore, we are potentially making an access decision based on an incorrect endpoint. I see this in my work; decision makers, who have access to this information, might rely on it, but they might also be motivated to restrict patient access to save themselves money. It could be used for convenience, to back a money-saving decision. This is what we want to avoid.
“Is it the methodology of calculating the QALY, or the way the QALY is implemented? Which is the problem? I believe it is both.”
You may ask: what is the problem? Is it the methodology of calculating the QALY, or the way the QALY is implemented? Which is the problem? I believe it is both. The methodology is problematic because we know that there are inherent limitations to it, and that some poor or flawed data is often incorporated into calculating it. The implementation of the QALY is also an issue, as some may use it to make an access decision without correct context.
What is the alternative?
We do need alternatives, and the health economics community is starting to delve into them, such as multi-criteria decision analysis (MDCA). However, this also has its own limitations. If we are going to use tools like the QALY or MDCA, we must engage patients from the outset and understand what outcomes and trade-offs are most important to them. There may be some illnesses, for example, where a patient is willing to take on more risks and downsides to achieve a particular outcome. There may be other cases where a patient is unwilling to do so. Without having the patient at the table to have those conversations and for stakeholders to know from the beginning what outcomes the patient is looking for, we cannot do a good job of following that through this mathematical formula and calculation.
“If we are going to use tools like the QALY or MDCA, we must engage patients from the outset and understand what outcomes and trade-offs are most important to them.”
What are some of the challenges in trying to represent a whole patient population?
One of our biggest challenges is finding the correct ‘representativeness’; the representativeness of the group that you bring to the table has to be in alignment with your objective. Sometimes, this can be accomplished by having a conversation with one patient only. Sometimes, this can only be accomplished by bringing in groups of patients that represent diverse populations. These groups can tell you, more broadly, what is going on in the patient community, as opposed to an individual who will tell you what is happening to ‘me’, and what ‘I’ am experiencing. Other times, you may need to have patients’ caregivers at the table, for example when dealing with Alzheimer’s disease. There is no one-size-fits-all model. It’s dependent on the patient’s or caregiver’s objective.
What do you think are some of the obstacles to getting more patient input?
There are lots of obstacles and barriers. For me, the cultural obstacle is one of the greatest; someone must get out of their comfort zone and do things differently than they have in the past. In the past, doctors did not talk to patients, scientists developing medical products did not talk to patients and payers did not talk to patients. All these groups must overcome this barrier of having never done this before. Once this is done, they will realize that the experience was actually worthwhile and they have acquired new, useful information that can improve their work.
“Without having the patient at the table…to know from the beginning what outcomes the patient is looking for, we cannot do a good job of following that through this mathematical formula and calculation.”
Where we are going to be in the next few years — are we moving in the right direction?
I think so; it is going to get easier and better. The clinical research side is changing. Right now, we have to use the data that we have and we are stuck with years’ worth of studies with irrelevant endpoints to patients. Now that we have entered an era of patient-focused medical product development, and patients are being brought into the medical product development and research sides as early as possible, we are going to produce better studies that will give us better inputs for our decision making.
“Now that we have entered an era of patient-focused medical product development…we are going to produce better studies that will give us better inputs for our decision making. “