Value Viewpoint: May 3, 2024

On Tuesday, Health Affairs Forefront published a commentary about establishing an HTA-like framework for the CMS Drug Price Negotiation Program (DPNP). In the commentary, Dan Ollendorf, Chief Scientific Officer and Director of HTA Methods and Engagement at ICER, and co-authors from Harvard PORTAL and UW CHOICE point out that the program currently lacks:

• openness and transparency,

• the involvement of stakeholders and public deliberation, and

• the use of credible and evidence-based methodologies.

While I’m not on board with CMS using price controls under the guise of negotiation or the commentary’s call for a national, ICER-like advisory committee, I do agree that value assessments should be conducted in an open, credible, and comprehensive way. I also applaud the inclusion of multi-criteria decision analysis (MCDA) in the commentary’s proposed framework to capture important value factors beyond what would normally be considered in a value assessment. I especially liked this quote from the commentary,

“Our approach is borne of a conviction that negotiation for the sole purpose [of] achieving the lowest possible price will send the wrong signals to industry and the wider public about the benefits of pharmaceutical innovation, and that the appropriate tool for negotiation is an ascertainment of the value these drugs provide.”

A new study in Value in Health investigated shifting away from traditional cost-effectiveness analysis (CEA) based on the quality-adjusted life-year (QALY) to a generalized cost-effectiveness analysis (GCEA), which seeks to address issues with the QALY and incorporate novel value elements. The study's authors aimed to better understand how to measure and incorporate these new value elements in CEA and how CEA could inform the DPNP.

Although GCEA could be a useful approach, it could easily be brushed off by policymakers who are more interested in political soundbites than good policy. As the authors conclude,

“Of course, depending on the political environment, GCEA might not end up informing Medicare negotiation at all. It’s possible the policymakers will maintain a simple model of price controlling the highest-priced drugs, which would discourage investment in orphan disease research and development, or the drugs with the highest sales, which would discourage all investment in research and development – consider the absurdity of a race in which no one wants to be in the first ten to finish. So, in the end, doing GCEA might merely serve to alert us all to the consequences of policy that fails to take societal value into account.”

The QALY challenges and ongoing debate were front and center in at least two other places this week:

• On Wednesday, the U.S. Department of Health and Human Services (HHS) announced the final rule governing Section 504 of the Rehabilitation Act. This section addresses people with disabilities and how to protect their rights in federal programs and activities. Concerning value assessment, the final rule contains language that prohibits the use of: “any measure, assessment, or tool that discounts the value of life extension on the basis of disability to deny or afford an unequal opportunity to qualified individuals with disabilities with respect to the eligibility or referral for, or provision or withdrawal of any aid, benefit, or service, including the terms or conditions under which they are made available.” While the interpretation may be in the eye of the beholder, this ban most likely includes measures such as the QALY.

• In a timely response to legislation and regulations aimed at prohibiting the use of the QALY, a new editorial in Value in Health (paywalled) identified the scientific support of the QALY and laid out an argument for how the QALY can be used in healthcare decision-making in a non-discriminatory way. The authors do acknowledge that, “When used in CEA for treatments targeted for disabled and chronically ill populations, health economists must exercise caution when treatment extends survival in that state; use of “ex-post” utilities, for patients experienced in those conditions, should be considered. Other QALY-like approaches may help to address these issues.”

• On Friday, Health Affairs Forefront published an article defending the QALY. Although it came out too late for this issue of the newsletter, it's topical and authored by luminaries in the field including Peter Neumann and Marthe Gold, so I'm editing the newsletter to add it post-production.

Pivoting to ICER, a new study in Value in Health (paywalled) this week compared value assessments conducted by ICER with those conducted by the German Federal Joint Committee (G-BA) for 15 drug assessment pairs matched on indication, patient subgroup, and comparator treatment. Interestingly, none of the assessments analyzed in the study showed agreement between ICER and G-BA. From the study's conclusion,

“The findings demonstrate that even under conditions where populations and comparators are identical, and the evidence base is consistent, different assessors may arrive at divergent conclusions about comparative effectiveness, thus underscoring the presence of value judgments within assessments of clinical effectiveness.”

Why does this matter?

There is no single source of truth when it comes to value assessment; it is as much art as science. This study provides further evidence that value assessments should be viewed as a tool and not a rule, as there will always be significant variations in how different countries, experts, patient sub-populations, and healthcare systems view and value the same treatment. It also highlights the inadvisability of blindly “importing” assessments from other countries without considering the broader context and nuance. 

 On Thursday, ICER announced plans to assess the comparative clinical effectiveness and value of tabelecleucel (“tab-cel”) for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD). ICER is accepting stakeholder comments on the Draft Scoping Document through May 22, and the topic will be reviewed by the New England CEPAC in November 2024. 

Here are two quick hits to round out this update:

• This week, I spoke with The Evidence Base® about value assessment topics, including the role of patient experience data (PED) in HTA and the ICER/NEWDIGS white paper on financing new gene therapies; read the full interview here.

• If you're heading to Atlanta, please check out NPC’s activities at #ISPORAnnual next week. I look forward to connecting!