In one of the first business sessions at Asembia’s AXS23 Summit, a panel discussed findings from Alnylam’s Rare Disease Trend report and offered different perspectives on how the data can be leveraged to improve outcomes.  

Stephen Crowe, VP, industry relations, Emisar, Jeremy Fredell, VP, pharma trade relations, Express Scripts, and Kasey Raetz, vice president, pharma contracting & strategy, Express Scripts, served as panelists for the session titled “The Current State of Rare Disease Therapeutics in the Payer Landscape.” The discussion was moderated by Ed Paiewonsky, director, medical outcomes science liaison, Alnylam Pharmaceuticals, Inc, who noted the session focused on trends and not Alnylam products.

“It's our hope that by sharing these critical insights and perspectives on current and future management trends and considerations, that we can elevate the discussion around rare disease products,” said Mr Paiewonsky.

The report was generated through a series of aggregated, anonymous surveys completed by payers, specifically pharmacy and medical directors, explained Mr Paiewonsky. 

Data in the report suggested similar utilization management levers are employed for both rare and nonrare disease products. When asked about the expected reaction from payers regarding utilization management trends, Mr Fredell said it will be crucial to understand how much attention clients give to total cost of care, including the impact on medical benefit, pharmacy benefit, or potential both depending on the drug. 

“They're going to be more in tune to how we manage both benefits … they don't want one benefit to become the loophole to the other in a drug that might be able to sit on both benefits,” said Mr Fredell. 

On that note, Mr Paiewonsky asked Ms Raetz where payers are from a capability standpoint when it comes to managing both pharmacy and medical benefits. 

Ms Raetz responded that it falls into 3 buckets: (1) Echoing Mr Fredell that payers are trying to avoid loopholes that could potentially create long-term consequences and financial strain, particularly on the pharmacy side; (2) looking at it through therapeutic category lens—“looking to preference the most clinically appropriate, cost-effective product, regardless of benefit”; and (3) payers are investing in technology, like single authorization systems regardless of medical vs pharmacy benefit.

In terms of the rare disease space, Ms Raetz sees complement inhibitors for myasthenia gravis as the best example of an area that has peaked payers interest in the last 6 months to a year and she anticipates it to continue growing. 

According to the Alnylam report, “Most pharmacy and medical directors are generally less aware of proposed policy reforms within the rare disease space and noted little impact to coverage decisions.” 

Mr Fredell polled the room and asked attendees if anyone in the room actually understands the impact of the Inflation Reduction Act and how it will affect new drug regulatory processes. Only one person expressed confidence by raising their hand, illustrating how important understanding the coming changes really is. 

Panelists agreed that regulatory and legislative changes often create a ripple effect. It is common to know change is coming but not truly understand the full impact until its enacted.

Another trend that surfaced in the Anylam report was health technology assessments or HTAs and their impact on payer decision making as it relates to rare disease. Ms Raetz said there is a lot of good information in them but they don’t significantly impact decision making. Mr Crowe agreed they’re not hugely impactful but in single payer systems where “the payer is the government; society does get a direct benefit from the outcome of a gene therapy.” However, in a US setting, employers could be stuck with a $2 million bill which can create issues. 

Mr Fredell compared this topic to feeling the temperature in the room. Therapies benefit from the review but it can be challenging to explain benefits of cost effectiveness relative to a population vs an individual plan. 

Per the report, “Innovative contracting is increasingly valued as a tool to help control the cost of care, especially as more rare disease therapies become available.” Further, “Of stakeholders expecting a shift toward innovative reimbursement models, ~38% anticipate implementation in 2023.”

When asked to identify some of the most significant barriers to creating more value-based contracting for these types of therapies, Mr Paiewonsky explained, “The top three specifically were me difficulty measuring outcomes, challenges with the data collection itself, and difficulty defining a valued metric specifically.” But he continued, that these barriers were often accompanied by optimism that changes are inevitable.