Last week, the Institute for Clinical and Economic Review (ICER) published its draft evidence report for sickle cell disease. The report assesses initial cost-effectiveness thresholds for two potential gene therapies against existing standard of care.

Despite ICER not having authority over the final list prices set by manufacturers, payers increasingly take these assessments into consideration when determining coverage, reimbursement and access. Media often picks up on final ICER assessments, which can influence how stakeholders feel about the value of a product prior to approval. While initial media coverage has been minimal, Endpoints and Fierce Pharma noted that both gene therapies offer promise of a potential cure, adding to pricing speculation in the lead up to price disclosure. 

Why is this moment noteworthy?

While the final assessment won’t come out until July, ICER’s value assessment of two gene therapy drug candidates for sickle cell disease provides some additional signals about how ICER will value gene therapies going forward. This is significant since we’re on the precipice of a wave of gene therapies entering the market. The science is exciting, but the multimillion-dollar price tags raise questions from health assessors like ICER and payers.

A closer look – What does the ICER report signal for future gene therapies?

The drug pricing watchdog group reaffirmed that an appetite remains for high-priced therapies that demonstrate incredible efficacy. In this case, both gene therapies exa-cel (Vertex/CRISPR) and lovo-cel (bluebird bio) demonstrated high efficacy for 97% of patients (this is essentially a cure for a very difficult, painful and life-shortening disease).

However, there are several notable trends that will be critical for cell and gene therapy manufacturers moving forward:

  • Health equity – With the emergence of health equity inclusion criteria, manufacturers should be prepared to show how their therapy works across subpopulations that are reflective of the real-world populations. As signaled by ICER’s chief medical officer, the two gene therapies may be considered within a higher range of cost-effectiveness (beyond $2M) due to historical discrimination and health inequities reported with sickle cell disease. 
  • Broad access – ICER says only 15.4% of patients with sickle cell anemia/disease can be treated at $2M, but 34% could be treated if priced at $900k. This is likely ICER’s way of saying to manufacturers, “Lower your price and more patients who desperately need these therapies can benefit.” In turn, manufacturers should be prepared to speak to any collaborative efforts with payers (e.g., innovative contracting or pay-over-time schemes) and contextualize potential cost offsets that may result with treatment. 
  • Patient voice – This was more prominent in the report than ever before. Notably, the “patient voice” was included within the cost-effectiveness methodology and featured prominently throughout the draft evidence report. Detailed accounts from patients, caregivers and advocates – discussing topics from stigmas associated with a sickle cell diagnosis, the painful burden of the disease and the historical lack of treatment options – were placed between datasets. This raises the stakes for manufacturers in the development of value messaging and, equally important, that they closely align with the unmet need(s) of the intended patient population.

Manufacturers of cell and gene therapies should pay close attention to the upcoming final report and public meeting scheduled on July 27, 2023, to pick up any additional signals of how their future cell and gene therapies are likely to be assessed.

About the Author:

Patrick Rigby joined Syneos from the private sector where he specialized in corporate communication, government relations, and public affairs across the healthcare industry. Patrick served as Director of Communications and later as Chief of Staff for the New Jersey Office of Homeland Security and Preparedness under two administrations. He also served as an Advisor to the Chairman of the U.S. Senate Foreign Relations Committee. Prior to government service, Patrick held positions with Bloomberg L.P., the Council on Foreign Relations, U.S. House of Representatives, and in the financial services industry. Patrick brings with him over 15 years of managing large teams and directing complex communications and reputation management programs for businesses and government.