Intended for payers and specialty pharmacists, Matt Dickinson, PharmD, MBA, Alnylam Pharmaceuticals, led a panel to highlight payer strategies for managing rare and specialty disease products and how they continue to evolve based on Alnylam Pharmaceuticals’ 2023 Rare & Specialty Trend Report. 

Dr Dickinson detailed the basis of the report, stating, “The contents of the report were generated through quantitative surveys to capture payer management as well as perspectives and practices for rare and specialty diseases.” To capture additional insights, Alnylam Pharmaceuticals provided follow-up qualitative surveys with payers. While the report covers changes and consistencies among trends year-over-year, Dr Dickinson explained there were “new sections in this edition on employer involvement and benefit design, strategic finding, and 340B trends.” This article highlights key insights discussed during the panel.

Dr Dickinson teed off the discussion outlining the data identified around the impact of value drivers on access, noting previous versions of the report where Alnylam explored the effect of budget pressures and the desire for “aggressive” approaches to management vs the investment in the practicality of implementation. While it is common knowledge that payers want to prioritize patient outcomes over financial burdens, understanding that eventually there will be more high-cost therapies in the market, in 2023, payers still ranked economic and contracting priorities among “4 of the 9 highest value drivers in pharmacy and therapeutics.”

In 2022, economic contracting and priorities only accounted for “one of the 9 highest value drives in pharmacy,” according to Dr Dickinson. Based on this data, Dr Dickinson posed a question specifically for Kasey Raetz, VP, Product + Pharma Strategy & Contracting, Express Scripts, “do you think that an increase in contracting and economic-focused value drivers, such as the treatment cost versus standard of care, is due to improved tools in the paired toolbox, or that budget pressures have resulted in an increased focus on these components?”

When considering payers and the tools they have at their disposal, Miss Raetz expressed, “We have better data [that is] more readily available.” She described that the system is more connected than it used to be, outlining that she believes that there could be more “tools in the payer toolbox,” but its issues like increased economic pressures that payers are having to balance—the financial burden of a lot of these high-cost products is what Miss Raetz believes is “driving them to think more innovatively.”

Different cost management tools and strategies are available, and payers are committed to finding a delicate balance while utilizing the tools at their disposal. Previous versions of the Rare & Specialty Trend Report support this. They detail that payers already considering tools such as manufacturer-generated cost-effectiveness models as less important for the evaluation of rare diseases. This begs the question, “What tools do payers need to optimize [to have an] economic-focused evaluation, and how can manufacturers partner?”

Miss Raetz emphasized that she had previous conversations that same day regarding various cost-effective models and how payers do or don’t use them. She said that when done appropriately and in partnership, “they can be effective, and payers must utilize that.” However, she noted hurdles payers have often faced when looking at typical cost-effective models, specifically because the models are both “the inputs and the outputs.” This means that stakeholders created some cost-effective models. Still, some of the inputs associated with those models are not easily transferable to how most payers make formulary decisions.

“When those inputs and outputs aren’t aligned, it’s really hard for us to leverage models in their current state to really make effective management decisions,” Miss Raetz said. Miss Raetz reiterated the need for partnerships to hone in on prevalence and incidence by asking the right questions, eg, “can we leverage real world data to truly understand the patients that are ultimately going to be on the drug?”

Regarding implementing strategic financing in response to policy changes, the 2023 report revealed that 34% of PBM respondents already implemented strategic financing, but 66% will not consider it. 8% of national plans have already implemented strategic financing, 25% are considering it, and 67% will not. 17% of managed Medicaid implemented strategic financing, but 83% will not consider it. Only 9% of regional plans have considered it, but 91% will not.

Another avenue for assistance with value and coverage, Dr Dickinson switched gears and asked the panel about specifics regarding specialty pharmacies, first asking Josh Parker, VP, Pharma Trade Relations, Accredo, whether there is reporting from specialty pharmacies that payers are utilizing to forecast the long-term economic impacts of rare diseases. Mr Parker explained that modeling tools are available, especially when new drugs, new gene therapies, and new rare diseases cost a lot of money. He highlighted that “[payers] want to know what impacts they have on their book of business.” Mr Parker added that specialty pharmacies and payers are partnering to review the drug pipeline to identify what would impact their book of business, which helps payers from a “predictability perspective [and] a budgeting perspective.”

Alnylam Pharmaceuticals' 2023 Rare & Specialty Trend Report investigated this dynamic and found that, according to its survey, specialty pharmacy integration remains a priority for payers as they look for ways to medicate patients and keep high pharmacy spending neutral. According to the report, “payers with specialty pharmacy services is a priority.” Its involvement in the product journey results in more cost-effective and efficient care. The report also details that among those surveyed, pharmacy benefit managers, national payers, and regional payers reported being vertically integrated with a specialty pharmacy.

Dr Dickinson followed-up with Mr Parker, asking “in rare disease where patient care is so complicated, nuanced, and high-touch, how do payers integrate in specialty pharmacy to maximize the quality of care?” From the pharmacy perspective, Mr Parker explained that they specialize in rare disease, which allows for specialization. If there are a lot of patients within a category, there are different therapeutic resource centers within the organization that allows them to specialize treatment, allowing them to understand the patient and “work with our former partners out there [regarding] drug interactions and other things specific to [the] therapy.”

Dr Dickinson shared additional insights from the report and mentioned that some questions were related to strategic financing because “payers have historically relied on strategic financing techniques, which allow clinicians to be trained in risk mitigation options to control traditional costs within their organizations.” However, Dr Dickinson noted that some large-scale pharmacy benefit managers (PBMs) and national health plans are beginning to use these techniques to “manage the financial impacts of discounted channels and expanding policies for things like 340B or patient assistance programs.” Despite this information, the survey revealed that most payers said they’re not considering strategic financing initiatives in response to changes in policy, such as annuity, subscription, and capitation models.

Mr Parker believed the uptake has been slow “because the volume is not there.” He indicated that payers have not seen this mass entry of expensive gene therapies because of slow volume. As more gene therapies are approved, which are expensive, Mr Parker believes that “over time, our peers will adopt those and see their value.” He also noted the high-interest rate related to pay-over-time models. He used the example of a patient who may go from one payer to another within a year. He explained that “payers don’t want to sign up for paying over time” because a patient may come in, and then, by the following year, they may move to another payer. He described the payer over time model as “fuzzy.”

Mr Parker believes that another solution is needed for how some of these programs follow the patient. “When it comes to the capitation models on per patient per month, I think those are very valuable,” said Mr Parker.

It’s clear the industry needs to revisit partnerships and payment models to best support every stakeholder’s needs, and perhaps committees can be a step in the right direction. In the 2020 version of the report, “only 25% of survey respondents anticipated creating pharmacy and therapeutics subcommittees focused on rare disease management within the next 5 years.”

In the 2023 report, while subcommittees in rare and specialty remained limited to the largest payers, “survey results showed 60% of national plans currently have a subcommittee dedicated to rare and/or specialty drug evaluation and coverage.” While that number is an improvement, it does demonstrate that further work needs to be done to provide value in this treatment landscape.

For more information on the 2023 Rare & Specialty Trend Report, click here.

Reference

Dickinson M, Crowe S, Parker J, Raetz K. The current state of rare disease therapeutics in the payer landscape. Presented at: Asembia 2024; April 30, 2024; Las Vegas, NV.