Chester Good, MD, MPH, is professor of medicine and pharmacy, University of Pittsburgh School of Medicine and School of Pharmacy. He is also the senior medical director, UPMC Health Plan Insurance Division, as well as the director for the Center for Value-Based Pharmacy Initiatives.
What are the potential benefits of value-based contracting for patients with multiple sclerosis, as well as for health systems and payers?
CHESTER GOOD, MD, MPH: I think for all 3, value-based contracting really is about moving from volume to value. And in doing so, what we're trying to do is recognize the value of pharmaceuticals perhaps in ways that we previously have not.
Patients with conditions such as multiple sclerosis (MS) want to be able to live their lives where they can continue to work, maintain their activities of daily living, and maintain their quality of life. Value-based contracting ideally recognizes those patient values and leverages contracting of pharmaceuticals that have been shown to help maintain those qualities in patients. For health systems, our greatest duty is to our patients in terms of maintaining their quality of life, maintaining their health, and preventing disease progression. By developing value-based contracts in which we recognize these measurable outcomes, we're showing a commitment to our patients and trying to hopefully establish the value of our pharmacy benefit.
For most health plans and payers, MS especially has become an increasingly expensive disease, with MS-related medications representing a significant cost burden. It's no secret that MS drugs have gone up significantly in recent years.
In a paper published in JAMA Neurology,1 we looked at the costs of these self-administered disease-modifying therapies for MS. From 2006 to 2016, the costs more than quadrupled, from a mean of $18,660 a year to more than $75,000, which means they increased at an annual rate of almost 13%. For payers, pharmaceutical spending per 1000 beneficiaries increased tenfold; and out-of-pocket expenses to our patients also increased significantly.
So, that's the reality. But the other reality is that there have been significant advancements in the treatment of MS using pharmaceuticals. And some of these newer treatments are more effective. Moreover, some are easier to take than some of the older therapies, and some are better tolerated from a patient perspective.
As a result of dramatic increase in costs of MS drugs, payers are asking about what they are getting in return for all these dramatic increases in costs. One of the ways to demonstrate that value is to link paying for these drugs with a value-based contract.
Why is a disease such as MS a good fit for value-based contracting?
CHESTER GOOD, MD, MPH: MS is a fairly prevalent disease. Fortunately, pharmacotherapy has gotten better over the years, to the point where it's clear that the pharmacotherapy of MS has really changed the course of the disease for our patients, allowing them to maintain their quality of life and functional abilities.
On a personal level, I had a family member growing up who had MS. And she did not have the benefits of the newer drugs. And through my childhood and early adulthood, I watched as she went from a very productive working person to someone who could no longer work, was unable to take care of her children, and was institutionalized and eventually died from complications of MS. That would be unusual today.
In a value-based contract, what MS outcomes are linked to reimbursement, and how are they measured?
CHESTER GOOD, MD, MPH: Traditionally, outcomes-based agreements focused on things that would be easily measured through administrative data. These are things that either a drug manufacturer or insurer, or both, have identified as being what they think are important outcomes for that disease state, but also things that they think they would be able to measure. Oftentimes, these were intermediate outcomes. Did the patient remain on a drug? Were they adherent? What was the impact on hemoglobin A1c in patients with diabetes? Although these outcomes may be important, they may not be as important to our patients.
So for MS, I would argue that it's really important to try to expand on this idea of which MS-specific outcomes are meaningful to our patients, and then try to link them to reimbursement. One of the things that we do at the University of Pittsburgh Medical Center (UPMC) is survey our stakeholders, and we ask what outcomes are important, using the Delphi method. Our most important stakeholder, of course, is our patient. But we also survey the physicians who care for these patients, other clinicians who care for these patients, our pharmacists, pharmacy benefit managers, our industry partners, and sometimes other stakeholders such as employers.
Sometimes, the things that are important to the patients are easily measured. It may be that patients with heart disease don't want to have another heart attack, and typically with a heart attack you end up going into the hospital. In the case of other disease states, however, it may be that what's most important to our patients differs from what we think is most important. We don't make any assumptions. We measure what our patients tell us.
In the case of MS, before we entered into a value-based contract, we did a Delphi survey including patients, physicians, industry partners, payers, and our pharmacy benefits manager.2 We did several rounds of surveys, and in the second round we had our stakeholders rank outcomes. What we found was that the most important outcome was “worsening physical disability.” One hundred percent of our stakeholders—not just our patients, but everyone—ranked worsening physical disability as important.
I would've thought that patients would've said, “Well, I don't want an MS flare.” And we could argue that MS flares may result in worsening physical disability, but not always. When we asked what would be the easiest thing to measure, of course MS flares are the easiest thing, because they require an emergency department visit or use of corticosteroids or hospitalization.
Based on our survey, we picked disability progression as our outcome of interest and the basis for our value-based contract. We entered into a contract with Biogen for a couple of their drugs, dimethyl fumarate (Tecfidera) and interferon beta-1a (Avonex), 2 very important MS drugs. This is a classic patient-reported outcome, and of course that poses a lot of interesting challenges about how you actually measure it. So, we had to work with our subject matter experts in the clinics to identify and externally validate a patient-reported outcome that measured disability progression. We worked very hard so that we’re able to collect that in a way that doesn’t inconvenience our physicians or our patients.
There are other tools that can be used, one of which is the PROMIS tool developed through the National Institutes of Health (NIH). That's a way to quantify changes in patient-reported outcomes. The interesting thing is NIH specifically developed this tool with certain disease states in mind, one of which was MS. So, some of the PROMIS domains include pain, fatigue, physical functioning, emotional distress, cognitive functioning, and social functioning. And all these things could be very important to our MS patients.
We actually do use PROMIS measures and PROMIS questionnaires routinely with our patients who come to our neurology clinic. It's not part of our value-based contracts at this point, but it's something that is a potentially interesting way to measure patient-reported outcomes.
What are the key challenges to implementing a value-based contract?
CHESTER GOOD, MD, MPH: Value-based contracting has been far more laborious than I ever imagined. These are legal documents that are very long and complex, and quite daunting. We've tried our best to incorporate figures and diagrams to simplify issues and ensure that we all are understanding things similarly. But at the end of the day, despite our best efforts, these contracts remain very complex, and questions and unexpected situations develop as we implement them. So, that's been one of the challenges.
The pandemic has also posed some challenges. It has affected everything. That includes fewer patient clinic encounters, which is a challenge when some of our value-based contracts require vital signs, lab work, or other things dependent on patient visits. There are also fewer patients presenting with disease states. It’s been well documented that hospitalizations and patient visits to the emergency departments for various conditions have really plummeted in the face of the coronavirus. So, the pandemic is a clear confounder as we try to identify the value of these pharmaceuticals and how they've impacted care. That's been a huge challenge for us.
Another thing to mention is that we’ve tried to be very innovative in our value-based outcomes. But it's been very challenging to do the analyses. What seems to be clear in a contract may not be clear once you start to analyze things, as there are all sorts of scenarios that one cannot anticipate. That has required going back and forth with our industry partners. Fortunately, we have good working relationships with them, and we've been able to work out those wrinkles; but that's been a challenge.
I mentioned the importance of patient-reported outcomes, but they are a challenge to measure. How do you collect patient-reported outcomes in a way that does not pose a burden on our patients or clinicians?
Finally, a significant challenge for health care organizations and insurers that hope to do value-based contracting is that drug companies do not typically place a lot of risk on the table. While we've been very pleased with our value-based contracts thus far, in the future we hope to see greater risk in these contracts, especially for areas such as gene therapy.
What are the next steps in value-based contracting for MS?
CHESTER GOOD, MD, MPH: Multiple sclerosis can be a progressive disease, and it's over a lifetime. It can affect people's quality of life. It can affect their ability to work. It can affect the quality of their work and what sort of things that they can do.
Because MS can affect every aspect of life, it's not enough to simply measure things like disease flares or MRI lesions. Rather, we need to figure out ways to understand how these drugs impact our patients’ day-to-day lives, the quality of their lives, and their ability to function as normally as possible. I think these other patient-reported outcomes would represent important next steps in MS outcomes measurement. Our current value-based contract has 1 simple, albeit very important, outcome that we're looking at, but are there other ways to measure patients’ quality of life? Perhaps this will involve using PROMIS measures or other innovative ways to measure patient outcomes. We’ve discussed with some patient interest groups how to incorporate other measures that are very important to our patients.
From a payer perspective, it may not show up in terms of impacting total cost of care. Depending on the disease states, oftentimes if things progress slowly over years, it is difficult to demonstrate an impact. But it's not all only about decreasing our costs because we decrease hospitalizations; it’s also about improving the quality of our patients’ lives.
The next step for us at UPMC is to continue to gather our data and to analyze our outcomes. And based on that, we're hoping to expand our portfolio of drugs for the MS disease state and expand outcomes that we're measuring.