A Q&A With Derek van Amerongen, MD, MS
AJMC: What are some of the current unmet needs in the treatment and management of PAH?
VAN AMERONGEN: I think any patient would say that the number 1 unmet need in the treatment and management of PAH is a cure. As payers and providers, we always need to keep that in mind. The probability of finding a cure in the near future may be unrealistic right now, so the focus should be to help maximize patients’ ability to accomplish activities of daily living, to be productive, and to maximize their quality of life (QOL). That is tremendously important. A prime focus for health plans is determining how to help individuals age in place and maintain a positive level of QOL in their homes or other preferred settings. Many patients do not want to be forced into any kind of long-term care facility, and there is great concern over losing independence. Ensuring that patients have access to the right treatment to help them function in their daily lives meets that need. The treatments that are available today are far better than [are those that were available], say, 20 or 30 years ago, but there’s still a large opportunity for improvement.
AJMC: How can health plans help promote patient adherence to treatment plans that may include specialty drugs and medications with complicated dosage and administration requirements?
VAN AMERONGEN: One of the most important things that health plans bring to the table is the ability to understand their populations—to identify members who have these types of conditions and who are at risk for potential adverse events. Health plans can get these members into disease management programs and case management programs and act as coordinators for their care. Health plans don’t treat patients, and that’s an important point I always want to make. But, at the same time, health plans can frequently be the “air traffic controllers,” as I’ve heard people say, to ensure that the member is connected with the right team of health care professionals to meet their complex care needs and to help ensure that all members of the health care team are talking to each other. Health plans can help ensure that the member understands their benefits and has opportunities for accessing the care that they need. Health plans can coordinate with the pharmacy benefit manager or the dispensing pharmacy, which is tremendously important; most health plans have pharmacists who are trained in specific disease areas and, therefore, they can track patients’ use of medications and counsel patients on their medication regimens, which can be very complex. Health plans have an important supportive role, and care management is something that I think has made a big impact in medical care over the last 20 years.
AJMC: How can payers work to minimize barriers such as those associated with social determinants of health?
VAN AMERONGEN: I’m glad you mentioned social determinants of health. That’s something that plans have been focused on quite a bit in the last 5 to 6 years, perhaps even before providers were focused on it. Health plans realized, while reviewing the data, that although there are facilities, hospitals, and providers that are all connected in the plan, people still were not receiving preventive services. So that led to the obvious question—why? Why aren’t members following up and filling their prescriptions for diabetes or heart disease, for example? And when you start peeling back the layers of the onion, you will find that it’s very often related to an issue of the social determinants of health. The availability of transportation is a great example—you can have the greatest medical center in the world, but if a patient cannot get there, it doesn’t matter. And for the patient, medical care may be fifth or sixth [entry] on their list of priorities, because they are worried about paying their rent. They may not have access to healthy food, because they live in a food desert. There are many other factors that can keep people from being able to access care, and we should address those. We cannot just say that it’s the patient’s responsibility to show up and do whatever the doctor tells him or her, and that’s the end of the story. It’s so much more complex.
When viewed from a more holistic point of view, there have been many positive signs of improved outcomes in the last several years. Health plans have been instrumental in ensuring that considerations of social determinants of health are an important part of the routine conversation about optimizing health outcomes overall as opposed to just optimizing medical outcomes. Some examples of how health plans have approached care in a more holistic way include connecting members who do not have access to a car or those with disabilities with access to free transportation services provided by the local government; providing support services in the home for older adult members, such as housekeepers, to prevent them from being moved to long-term care; and using services like app-based transportation companies to deliver medications to members who may be homebound to ensure they continue to receive their treatments. This can be especially important after hospital discharge.
AJMC: How are members connected with the many resources available through their health plans?
VAN AMERONGEN: Great question. It must be a multifactorial process. On the back of the health care plan card, there is a number that members can call, and members can also go to the health plan’s website for information. The real opportunity, however, is in proactive outreach. Larger national plans, in particular, have very sophisticated predictive models and algorithms to identify members [at risk], ideally before they get into trouble—before they end up in the hospital or in an emergency department. Someone from the plan will then reach out to the member, engage with them, and tell them about the resources that are available….These services are free to the member. Health plans should leverage these data and their internal expertise in terms of case managers, pharmacists, and physicians to help coordinate care and ensure that the member understands a) [what] issues may be impacting their access to care; b) where they need to go to get their clinical care; and c) how to use their benefits effectively. Frequently, people do not understand all that is available in terms of what [their] coverage provides for them. We see this as a way to make investments early to avoid longer term clinical adverse outcomes. And, of course, if you decrease the complications, the adverse events, that will have an impact on cost as well.
AJMC: From the payer perspective, what do you see as the biggest challenge in PAH?
VAN AMERONGEN: One of the biggest challenges is identifying people who are at risk and then diagnosing PAH. There’s no question in my mind that there are many people who have PAH who don’t know it. PAH is associated with a complex set of symptoms, and it can be a challenging condition to diagnose. Therefore, the first step is to ensure that the patients who have PAH have been properly identified and that they have received a diagnosis. Once they have a diagnosis, then the challenge is connecting them with the proper care systems.
PAH is a complex disease that may require a higher level of care provided by specialists at tertiary care centers. These care centers are not available everywhere. Furthermore, the process of scheduling an appointment, and even following through with the appointment, at a tertiary care center can be very daunting. The medical establishment can be very intimidating for people who are not in medicine. That is an area where health plans can help. Plans can help members identify care centers, make the appointments, and make sure members get to the appointments.
There are also opportunities related to treatments. Again, I think everyone would agree that the treatments for PAH today are far superior to those that were available 20 [or] 30 years ago, but there is a lot of room for improvement. Cost considerations for patients with rare diseases such as PAH cover 2 broad areas: direct clinical treatment costs (eg, provider visits, hospitalizations, testing, and other interventions) and drug costs. In the last 10 years, the focus has shifted mainly onto drug costs. Part of this is because we now have medications that are more effectively addressing signs of symptoms of these diseases and, thus, leading to a reduction in direct clinical treatment costs, partly because the costs for orphan drugs have substantially increased. Ideally, health plans also can address the issue of affordability, because it is concerning that there are patients who could, and should, be in treatment [but] who are not, because they cannot afford it. In some cases, patients do not have health insurance. However, even among those who are insured, the robustness of insurance coverage varies tremendously. There is no question in my mind that there are people who are insured, but, for various reasons, cannot afford the level of care that they need and deserve. I think all of those are challenges. The good news is, I think that they are all fixable. I am relatively optimistic about where we will be, say, in the next 5 to 10 years.
The pipeline is robust in so many therapeutic areas, including PAH. It’s full. There are a lot of novel treatments coming. The challenge is always going to be affordability—not just on the individual level, but on a societal level, as well. That being said, I think that affordability and cost not only to the individual, but to society as well, are being addressed. The Inflation Reduction Act1 is an important first step in that direction, I think. And I also see it as an important step forward.
I am hopeful that maybe in the next few years, we will have an inflection of care moment for PAH, meaning that we have either a treatment to cure the disease or one that is associated with a considerable increase in survival, [ability to perform] activities of daily living, and QOL. The alignment of incentives between providers, health plans, and other stakeholders in the last few years has meant that there has been increasing use of care and interventions that support the long-term well-being of members, regardless of their conditions or health status. A growing emphasis on prevention, expanding access to all the elements that promote better clinical outcomes, and a desire to help people continue to live independently will help individuals experience the optimal health status they deserve.
1. Inflation Reduction Act of 2022, HR 5376, 117th Congress (2022). Accessed March 20, 2023. https://www.congress.gov/bill/117th-congress/house-bill/5376/text