Pharmaceutical manufacturers historically have spearheaded most outcomes research into their products. But specialty pharmacies, faced with complex patient paths and stiffening competition for limited-distribution drugs, increasingly are conducting their own outcomes studies.
The goal, stakeholders say, is for specialty pharmacies — particularly those that are owned by health systems and integrated delivery networks (IDNs) — to show how their services improve patient care and are cost-effective overall.
“Historically, outcomes research has been on the pharma side of things,” says Autumn Zuckerman, Pharm.D., program director of health outcomes and research at Vanderbilt Specialty Pharmacy. “They have the specific health outcomes and research positions and fellowships — a lot of fellowships and training.” The research primarily has focused narrowly on the medication and its results, she says. However, that leaves out “that whole patient journey,” spanning from obtaining the medication, affording the medication, starting treatment and then getting the outcomes, Zuckerman says.
What Is ‘Full Patient Journey’?
Pharma manufacturers still are the experts in outcomes research — and still have most of the funding, she says. “But what we have done and what we continue to hope to do is shed light on the patient journey — that full patient journey from the time even before they decide to start treatment, the time that they are introduced to specialty pharmacy. What does that patient journey look like? What medications have they tried before that brought them here? Should we have found out earlier that they should be on a specialty medication, or should they not be on a specialty medication?”
Zuckerman adds, “So from that very first entry into care, all the way through persistence and outcomes and monitoring for safety and efficacy and everything in between, that’s where I think the specialty pharmacy can do a really good job of studying and scientifically evaluating the gaps in care and where we can improve, and what we need from a resource perspective to help patients get from A to B.”
Life sciences organizations and biotech pharmaceutical companies historically have led the charge in terms of substantiating the value of these products for decision-makers in health plans and in PBMs, says John Linnehan, practice director of health economics and advanced analytics at Avalere Health.
“As specialty pharmaceuticals continue to grow in terms of share of overall cost of pharmaceutical drugs broadly and in terms of innovation and considerations around the value derived within the health care system, there has been an increased focus broadly on defining and articulating the value of specialty pharmaceuticals, because they often have higher price tags, and we’re seeing a lot more innovation in the area,” Linnehan tells AIS Health. This includes new research efforts from more diverse stakeholders, such as health system specialty pharmacies, he says.
These days, specialty pharmacies of all types — PBM-affiliated, independent and health system-owned — are eager to show the value of the services they’re providing, “and so outcomes research is playing a more important role there, although it’s more around substantiating the value of the services that the specialty pharmacies provide and less on the real-world effectiveness of the products,” Linnehan says. “Research is more driven by the investments that specialty pharmacies are making in increasing the depth and intensity and patient touch points of the wraparound services that they’re providing.”
This occurs regardless of what type of entity — insurer, PBM or IDN — owns the specialty pharmacy organization, Linnehan says. Instead, it’s about the imperative for those specialty pharmacies to demonstrate their value within the distribution model that exists, he says. “It’s really the organizations who are looking to innovate beyond standard filling of scripts and wanting to get more involved in long-term financing of cell and gene therapies. It’s those specialty pharmacies who have the incentive to substantiate their value further because they’re asking for more investment from their end customers, which in many cases are life sciences companies.”
Various Entities Are Providing Funding
Investment also is coming from all sides, Linnehan says. “If the outcomes-based contract is tied to payment for a specific drug, the manufacturer is often going to pay for that evidence generation over the course of an outcomes-based contract or up front,” he says. “If the focus of the research is to substantiate the impact of the actual patient interventions that are being provided, oftentimes it’s the specialty pharmacy making that investment, because they can then use that to sell services to other manufacturers and health plans and PBMs.” Still, adds F. Randy Vogenberg, Ph.D., principal for the Institute for Integrated Healthcare, “pharma is where all the money is.”
Partnerships between manufacturers and specialty pharmacies that include outcomes research often arise as a result of a request for proposal from a manufacturer seeking wraparound services, Linnehan says. Manufacturers are incented to find partners who can most efficiently handle distribution, financing, administration and other wraparound services, and they’ll often choose a partner based on that partner’s ability to substantiate their offerings, he says.
When it comes to therapeutic areas, oncology has seen a substantial amount of investment in outcomes research, since there are cell and gene therapy products, along with other high-cost medications, with the potential to drive differentiating value, Linnehan says. “We also see this type of activity in hemophilia, in multiple sclerosis and in a number of rare diseases,” he adds.
Because specialty pharmacies are now more and more involved in distribution and also financing of wraparound services for these products, “there’s a substantial opportunity for specialty pharmacies to drive value in this model,” Linnehan says. Driving value can occur in the context of “getting the drugs from the pharmaceutical company or from distributors to patients, but also supporting those patients in education around how to physically take the product, and other high-touch wraparound offerings,” he says. “So there is more of an imperative for specialty pharmacies to demonstrate the stand-alone value that they’re contributing as part of this process.”
Industry Disruption Drives Efforts
Disruption and consolidation in the health care industry overall play a significant role in propelling specialty pharmacies to increase their outcomes research efforts, says Vogenberg.
New and accelerating competitive pressures abound, he tells AIS Health. In some cases, pharma companies may need to contract with only one large specialty pharmacy organization, or with a couple of major players, for a particular specialty drug with a small potential population, he says. In other cases, the manufacturer might want one distributor but has a network of either IDNs or specialty pharmacies that would receive the drug. Competition is significant to win access to new specialty products.
To counter these dynamics, health systems’ specialty pharmacy units are trying to differentiate themselves by showing, via their outcomes research efforts, that they provide superior care, says Zuckerman. They’re eager to demonstrate value and are actively investing in new roles and organizations designed to meet that goal, she says.
Individual IDNs are ramping up investments specifically in outcomes research, she states. Vanderbilt’s specialty pharmacy outcomes research program, a leader in the field, was established for three reasons, according to its mission statement: “To improve patient care through sound research, measure and expand the value of Vanderbilt Specialty Pharmacy, and foster research training in the field of specialty pharmacy.”
IDNs Hope to Get More of Specialty Pie
Vanderbilt started the research institution for obvious competitive reasons, Zuckerman explains: “Health systems’ specialty pharmacies are about 20% of the [overall specialty pharmacy] pie — definitely increasing, but we are the small fry with the payer groups. And because of that, we’re often cut out of being able to service our patients, mainly due to payer contracts, but still sometimes due to manufacturer limited-distribution drug networks. So in 2015, we got together at Vanderbilt and said, ‘We really need to produce research that shows we are providing the best possible care to the patient and the best value for all stakeholders.’”
With that research showing superior outcomes in hand, Zuckerman says, Vanderbilt can go to payers and manufacturers and ask for access to patients and products. She says there haven’t been specific instances where a study led to access to a drug, but “we do know that [the research] has been very instrumental, not only in specific conversations that Vanderbilt has had, but for health systems’ specialty pharmacies in general. My goal is for it to be full advocacy for health systems’ specialty pharmacies, not just Vanderbilt.”
Health system specialty pharmacies are working more closely together in general. In October, for example, eight of the nation’s leading health systems and other network providers of specialty pharmacy patient care announced the formation of the Health System Owned Specialty Pharmacy Alliance (HOSP), which will focus on advocating for the interests of integrated specialty pharmacies and promoting best practices that enable them to deliver the best patient care and patient outcomes. HOSP will “act as the ‘face and voice’ of the integrated specialty pharmacy industry,” said Tanya Menchi, the new organization’s executive director, in a statement.
In addition, for the past two years, Vanderbilt has hosted a Specialty Pharmacy Outcomes Research Summit, designed to bring stakeholders from all sides together to share views and research.
The movement has grown well beyond Vanderbilt’s initial effort, Zuckerman says. A consortium has formed to support specialty pharmacy outcomes research that includes 44 different health systems, and there are studies coming out of UIC Specialty Pharmacy Services, the University of Utah Specialty Pharmacy and Johns Hopkins Specialty Pharmacy, she says. In addition, the University of Kentucky’s specialty pharmacy program recently developed a position similar to Zuckerman’s Vanderbilt post to focus on outcomes research and value.
The first research to be presented from Vanderbilt’s program debuted at the Academy of Managed Care Pharmacy Nexus meeting in October and showed superior outcomes in adherence in rheumatoid arthritis, Zuckerman says. The second study, which the team is working on now, is prospective research around multiple sclerosis.
Pharma Finances Most Research
Vanderbilt recently has signed five different research contracts with pharma companies to conduct outcomes studies. Some of this research involves tailored monitoring, she says: “If I take care of your patient in a different way, by calling them more frequently or sending starter kits, does that help them stay on the treatment?” Other pharma-supported research at Vanderbilt has involved prescribing pathways — what brings a patient to a certain treatment, how often they change treatment and why they changed treatment, she says.
It’s difficult to say what percentage of pharmaceutical companies’ research budgets is dedicated specifically to post-approval outcomes research, but companies are spending “millions of dollars specifically on health economics and outcomes research” per year, says Linnehan. But they’re not the only stakeholders doing work in this area, he notes.
“Pharmaceutical manufacturers want to conduct outcomes research to demonstrate both the clinical cost resource use and the humanistic impact of the products that they’re developing,” Linnehan explains. “Specialty pharmacies are investing more in outcomes research because they want to substantiate the value, not specifically of the products themselves, but of the model of specialty pharmacy they’re providing.” This includes going beyond managing filling of scripts to demonstrate value based on such factors as time to fill and adherence rate, he says.
Still, negotiating contracts with pharmaceutical manufacturers can be challenging, Zuckerman maintains. In fact, her institute has worked to bring representatives of pharma and health system specialty pharmacies together at its summit to talk about how to execute partnerships.
Pharmaceutical manufacturers can provide financing and help with data, she says, but it has taken Vanderbilt an average of about 12 months to negotiate an outcomes research contract. “So even when we’re on the same page and we’re all interested in the same thing, it’s a long process.” In one situation, Vanderbilt spent three years on a project and then the pharmaceutical manufacturer backed out, citing changed priorities, she notes.
Meanwhile, payers have been reluctant to engage in outcomes-related projects at all, says Zuckerman. Vanderbilt has been trying to interest payers in projects that could show how to keep patients out of the hospital but has found reluctance, particularly from those closely aligned with a different specialty pharmacy. Still, Zuckerman says she’ll keep trying: “Our goals are aligned if we could all come together.”
Data is key, of course. Zuckerman points out that the data needed for outcomes research spanning the entire patient journey isn’t available as part of a claims data set, “so it’s really reliant on health systems and health systems’ specialty pharmacies to do that type of outcomes research.”
Researchers need both claims-based and clinical-based data, preferably integrated and as timely as possible, Linnehan says, noting, “it’s really critical to utilize real-world data sources that are as timely as possible, so that this value can be demonstrated as quickly as possible as therapies initiate and then be able to track over time. So specialty pharmacies owned by, or closely aligned with, other stakeholders, whether it’s the health system or a health plan, often have an advantage from the perspective of data.”
There’s also some innovation occurring in which disparate sources of health care data such as claims and electronic medical records can be combined in ways that preserve patient privacy but allow additional insight into outcomes, Linnehan says. “So there are ways that standalone specialty pharmacies can take advantage of this, although the integration [found in specialty pharmacies owned by or aligned with other stakeholders] certainly gives a head start.”
Even if data is available from various sources, combining all of it can be a challenge, Zuckerman says. Her team found this to be true in the rheumatoid arthritis study, which involved looking at data from multiple institutions. Still, granular chart data provides “unique insight into the patient journey vs. just a claims-based study,” she says. The health systems involved paid for that study, without help from any manufacturers.
Of course, there are multiple payment models in which long-term financing for cell and gene therapies is being tied to outcomes, with more to come, Linnehan says. This likely will drive further investment in the field of outcomes research generally, he says. “Think of it more as ongoing outcomes research where payers have the opportunity to pay over a number of years, but the degree to which they’re being asked to pay is tied to the durability of the outcomes.”
For example, Cambridge, Mass.-based bluebird bio, Inc., has been open about putting these models in place, Linnehan points out. The company’s one-time gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), which was approved to launch in Germany in early 2020 to treat beta thalassemia, is priced at around $1.77 million, amortized over five years. The firm is paid most of that money only if the treatment works, “and that’s where outcomes research plays a role over time to help substantiate the durability of the response,” Linnehan says.
Area Is Emerging Discipline
Overall, outcomes research in the context of specialty pharmacy is an emerging discipline, Linnehan says. “Traditional outcomes measures that have been used to assess the impact of specialty pharmacy include things like time to sell, adherence data and patient touch-point-based metrics. But a focus on outcomes-based contracting — and paying for outcomes and financing products over time with a tie to outcomes — that’s what’s really driving this focus on substantiating the value of specialty pharmacy services more broadly.”
Over the next five years, he says, gene and chimeric antigen receptor T-cell (CAR-T) therapies will demonstrate with more clarity what real-world value they deliver. “We would expect outcomes research to come out alongside that. The next five years is really going to follow the timeline of the impact of many of these therapies in the real world. And so I would expect it to increase,” he says.
Meanwhile, specialty pharmacies within health systems are looking for research showing the value of their services, Zuckerman says. “I hope that those elements spur pharma and payers to work with us, because we really think that our goals are aligned: providing the best patient care at the lowest possible cost. So I hope it leads to a bigger piece of the pie.”
The first fruits of this outcomes research, Zuckerman believes, “will be additional insight into the patient journey. Specialty pharmacy is still such a new and evolving field that we really need more information about what patients go through and how to support them in the process.”
by Jane Anderson