Value Viewpoint: June 8, 2024

In celebration of a significant milestone, the 3-year anniversary of John O’Brien ’s tenure as NPC President and CEO (Happy Anniversary, John!), we’re releasing a deck highlighting the value we provide to NPC members and the benefits member companies experience. To meet the challenges of today and tomorrow, NPC has built new capabilities that allow us to build on our 70-year legacy as we conduct rigorous, policy-relevant research and communicate it with impact. We invite you to learn more about us and becoming a member!

On Wednesday, a new article published in Health Affairs Forefront from my NPC colleagues Julie Patterson , James Motyka , Jon Campbell , and John O’Brien provided an in-depth overview of the uncertainties, challenges, and unintended consequences for patients of state prescription drug affordability boards (PDABs). The article focuses on PDABs in four states (Colorado, Minnesota, Maryland, and Washington), which have been given the authority to set upper payment limits (UPLs) for drugs that the respective boards classify as “unaffordable.” Here are a few key takeaways:

1. There is high variance and ambiguity between states including in board structure, drug identification and selection, conducting reviews, and setting UPLs (see the table below).
2. There is an almost singular focus on price-related factors and “affordability” while ignoring the important roles of benefit design, as well as clinical and value-related elements.
3. Patient access could be harmed from upstream effects, such as changing economics at the wholesale level. For example, due to UPLs, wholesalers may be unable to contract with state-level pharmacies, and impacts on reimbursement for physician-administered drugs could lead to consolidation of smaller practices or shifts to more expensive treatments.
4. Drugs are eligible for affordability review at launch in many states, which has the potential to reduce research and development, particularly post-approval research on obtaining new evidence and studying new indications.

As the article concludes,

“Overall, the singular focus on price-related factors in the absence of statutory requirements to prioritize value-related elements amplifies concerns about potential unintended consequences while providing no assurance that PDABs will achieve their intended goals of improving affordability for patients.”

And shoutout to Brian Reid who sums the issue up as only he could, “If this all seems like a loosey-goosey approach to an incredibly vital issue, well ... that's exactly the point (and the problem).”

A letter to the editor published in the Journal of Managed Care & Specialty Pharmacy (JMCP) earlier this week discusses the role that real-world evidence (RWE) could play in the Medicare Drug Price Negotiation Program (DPNP). The letter rightly points out that RWE is likely to play a more important role in CMS decision-making moving forward as they will be evaluating drugs that have been on the market for 7 to 11 years and are seeking to understand comparative effectiveness against therapeutic alternatives as well as the impact on special populations of interest – all topics well suited for RWE studies.

The letter authors provide several recommendations for CMS in an effort to improve the utility of RWE studies in their decision-making including:

• Commit to using RWE for comparative effectiveness and provide clarity on how CMS will evaluate RWE studies and weigh new RWE evidence against other sources.
• Publicize their process for evaluating RWE study quality and build upon the published guidance of others such as from NICE, FDA, PCORI, and ISPOR as to how to conduct a methodologically rigorous and valid RWE study.
• Focus on developing their RWD infrastructure to ensure data are high quality and accessible for RWE studies while also identifying outside RWD sources suitable for linking.

These recommendations echo much of what we have previously published at NPC about RWE and recommendations to CMS. In our Guiding Practices for Patient-Centered Value Assessment updated in January of this year, we write,

“…real-world evidence may provide an additional understanding of how a treatment is used for typical patients, and its comparative assessment to alternative patient care options. It is uncommon that clinical trials – designed for regulatory review of efficacy and safety– yield adequate information on healthcare utilization and costs to support a full value assessment. This is a gap that RWE is best suited to fill.”

NPC’s comment letter to CMS?on the DPNP in 2023 includes similar recommendations to those in the JMCP letter.?

This week, ICER released the protocol for its fourth annual “Barriers to Fair Access” assessment. The report examines how payer coverage policies align with a subset of fair access criteria that ICER previously identified in a 2020 white paper .

One of my primary frustrations with ICER's past "Fair Access" reports is the disproportionate focus on the more payer-friendly criteria (e.g. when it is “reasonable” for payers to implement access barriers) rather than the patient-focused criteria (e.g. holding payers accountable for alleviating the cost burden on patients).

This lopsided focus is partially driven by data challenges, but there is a bright spot in the new protocol. This year’s report will include a new exploratory analysis in partnership with IQVIA, focused on cost-sharing and prior authorization using real-world claims data.?

Also this week, the Innovation and Value Initiative (IVI) has launched a call for experts to join in Phase II of its Rare Disease Initiative . The initiative aims to make sure rare disease patient needs and perspectives are included in HTA and the healthcare decision-making process. The deadline to apply to become an Advisory Board Member is June 21 and the application can be found here .