The American Journal of Managed Care® interviewed 2 presenters at Asembia 2023, gaining insight on how the specialty pharmacy pipeline is growing and how health systems and payers can affect the accessibility and affordability of new specialty therapies.
At Asembia 2023, The American Journal of Managed Care® spoke to 2 presenters about the ever-changing specialty pharmacy space. Between 2015 and 2021, the number of specialty pharmacies has increased by 315%, with hospital- or health system–owned specialty pharmacies accounting for about a third of the total growth. Additionally, the specialty drug pipeline is expected to grow about 8% annually through 2025.
Ray Tancredi, divisional vice president at Walgreens, presented on the current specialty pharmacy pipeline and provided insight into how payers can make a difference in the space. Naveen Mansukhani, the director of operations and account management for retail pharmacy services at Cardinal Health, discussed some of the strategies (ie, value-based pricing tactics and leveraging data analytics) health systems are using to address the rising costs of specialty therapies.
Which specialty drug approvals do you expect to have the largest impact in managed care in 2023, and what are these impacts?
Tancredi: Yeah, that's a great question. I think the unmet need is NASH [nonalcoholic steatohepatitis]. So, there are 2 products, [obeticholic acid] Ocaliva, currently approved for primary biliary cholangitis, and there's also a product from Madrigal Pharmaceuticals called resmetirom. Ocaliva could be approved for NASH in June, so I'm looking forward to that, and then Madrigal's product could be approved in September. So, I know from a managed care perspective, 5% to 6% of the US population could actually have NASH. It's a significant patient population—12% of those could progress to cirrhosis. So, these drugs are very important.
I think from a managed care perspective, pharmacy directors and medical directors have been awaiting these products. I don't think it's a surprise. I'm sure they've factored it into their per-member per-month costs. But nevertheless, very exciting from a patient perspective. It gives physicians the ability to treat this subset of the patient population and have some successful therapies so patients don't progress to cirrhosis of the liver.
Additionally, we could see a couple gene and cell therapies approved this year. And as you know, gene and cell therapies can cost in the millions of dollars. I think there's 2 drugs that are really important to follow that we could see approved. I think the first one is SRP-9001 for Duchenne muscular dystrophy. When you look at Duchenne, 1 in every 3500 to 5000 male births have Duchenne muscular dystrophy. So, this really has an unmet need. I think secondarily, there's a product called valoctocogene roxaparvovec [Roctavian], which is for severe hemophilia A, so it's a subset of patients with hemophilia A. The product would be very expensive, but would really fill an unmet need in hemophilia. I think those 4 products, for the remainder of this year, I know that managed care will have their eye on.
What are some other drugs and biologics in development? Do you anticipate any specific outcomes for these, regarding their approval/use?
Tancredi: A lot of the products that I named are in development. I think, though, if you look at the pipeline, there are a significant number of drugs in general. Last year, there were 37 drugs approved and I think 20 of them were first in class. So, if you're a managed care organization, I think that's something to keep an eye on.
There are drugs like [mavacamten] Camzyos, [deucravacitinib] Sotyktu for plaque psoriasis, and [nivolumab/relatlimab] Opdualag for metastatic melanoma that were all significant improvements, first-in-class products. And again, I think even this year, we'll see that again. Twenty out of 37, or 54%, were first-in-class products and I know the pharmacy directors will keep their eyes on those drugs.
Are there any overarching trends or stark differences you see in the pipeline each year?
Tancredi: It's remarkably consistent, the pipeline. However, cell and gene products are really beginning to take over the pipeline. In fact, when I did my presentation at Asembia, I broke it out into 5 sections. So, the first section was rare and orphan diseases. We know there are 7000 rare and orphan diseases, and only 5% of those actually have an FDA-approved drug. I think I covered over 20 drugs in rare and orphan diseases that'll make an impact. I think, secondarily, you have traditional specialty pharmacy drugs—still significant. I covered another 20 drugs in that area.
Oncology remains a large part of the business and in oncology, we had [approximately] 25 drugs that we were able to cover. And then, cell and gene therapies were an additional section.
And ultimately, I covered some approvals. There were some blockbusters last year in specialty pharmacy, maybe drugs like [efgartigimod alfa] Vyvgart for generalized myasthenia gravis, [mavacamten] from BMS [Bristol Myers Squibb], and even [deucravacitinib] from BMS that are really meeting unmet needs and could help patients from a large scale.
So far in 2023, we've had about 15 overall drugs approved. Of those, 12 are considered specialty pharmacy products. So, that's well over 80%. And looking out for the rest of the year, there could be 39 more drug approvals from the FDA, of which 28 could be specialty. So, the pipeline is certainly robust. Even in oncology, I know we've only had 2 drugs approved so far this year, but over the last 10 years, we've seen about 13 drugs per year approved in oncology. When this year is over, I think we'll be very close to that number again.
What roles do payers play in specialty pharmacy?
Tancredi: Payers play a big role. When you think about what a payer will do for the specialty pharmacy industry, I look at a couple things. They'll create a specialty pharmacy network that has the ability to dispense drugs to their plan sponsors and insurers. I think that's so important. They evaluate, they vet the pharmacies, they ensure they can dispense the drugs that are required, and meet all the needs of that payer.
Secondarily, they have the ability to adjudicate the claims for accuracy and the appropriateness of therapy, which is so important to the physicians, to the patients, and even to the pharmacists to ensure that the claims that they're submitting are paid in an accurate manner. So, payers continue to be an important part of that specialty continuum.
What strategies are health systems employing to combat the increasing costs of specialty drugs within their specialty pharmacy programs?
Mansukhani: It's really about employing a collaborative approach. It's not just a pharmacy problem of controlling costs. It's also patient navigators, the pharmacy department, the billing department, ensuring that things are getting built properly before it's administered to a patient, where it ends up being a cost to the hospital.
How are health systems leveraging data and analytics to identify cost-saving opportunities and make informed decisions regarding specialty drug pricing and procurement?
Mansukhani: This is where the importance of a specialty GPO [group purchasing organization] comes into play. Specialty GPOs can help with these data analytics and ensure that the hospitals are buying the best possible drug at the lowest possible cost.
Are there any best practices or success stories of health system specialty pharmacy strategies that have effectively tackled the challenge of escalating specialty drug costs?
Mansukhani: Yeah, there's many hospitals that have employed strategies that [can help with], from a retail perspective, getting specialty drugs into the community. So—from the hospital into the community—ordering these drugs, and now, they become a revenue to the hospital vs just simply a cost. And that's where a specialty pharmacy strategy really makes sense and where hospitals are finding success. It also helps with the patient end-to-end approach where the hospital is able to work with patients on the outside when they become outpatients. It improves compliance, it improves outcomes, and it controls overall health care costs.
What considerations should health systems keep in mind when implementing strategies to address rising specialty drug prices, such as patient access, quality of care, and patient outcomes?
Mansukhani: So, all of these [things I mentioned before] are part of that collaborative approach. It's a collaborative approach of the patient navigator on the floor down to the person that's billing the claim down to the specialty pharmacy to ensure the patient has the drug when they need it. And collaboration with a manufacturer or a co-pay savings card can ensure the patient is getting [their medication] at the lowest possible price.
How can health systems balance the need for cost containment with the imperative of ensuring patient access to life-saving specialty drugs?
Mansukhani: Yeah, so again, this collaborative approach and usage of the specialty GPO is very important for hospitals to collaborate to ensure that they're giving the patient the right drug with the best cost containment strategy and also employing a biosimilar approach when those drugs are covered.
What impact are emerging trends in specialty pharmacy, such as biosimilars and value-based pricing, having on health system strategies for combating rising drug prices?
Mansukhani: Biosimilars are very important. They're going to play an important role in the in the upcoming future in controlling costs. But payers also determine what drugs are going to be covered on a patient's health plan. Hospitals are really employing these specialty retail pharmacies to get the patient the right drug at the right time at the right price.