Sign up for ICER's Weekly View Newsletter

Weekly View

April 5, 2024

Good morning, everyone.

From ICER’s President and CEO Sarah K. Emond, MPP:

Yesterday Amylyx announced it would remove Relyvrio, a treatment for amyotrophic lateral sclerosis (ALS), from the market. The drug was originally approved in 2022 based on a small phase II trial, well ahead of the conclusion of its phase III trial. The results of that phase III trial were reported last month and unfortunately, the therapy failed to provide any benefit to patients. Historically, a failed trial following FDA approval has not resulted in an automatic revocation of FDA approval or withdrawal of the drug from the market, and post-marketing trial requirements are not consistently used to assess the regulatory status of all approved products. Despite this hole in regulation, Amylyx made the responsible decision to discontinue this drug, and is being rightly lauded for the choice.


Of course, everyone hopes that treatments approved early with limited evidence will prove effective. But when they don’t, this is how it is supposed to play out: patients get early access to a potentially promising treatment, and then when all the data come in and the benefits fall short, the manufacturer removes the drug from the market. What’s missing from this story though is price: since 2022, the health system paid a steep price for a drug with no proven benefit to patients. When ICER reviewed Relyvrio (prior to FDA approval), we recommended that the manufacturer consider setting the launch price, “close to the cost of production until the benefits of treatment can be adequately evaluated." Amylyx priced the drug at $158,000 per year, far beyond ICER’s recommended price of $9,100 to $30,700 per year, a price range based on the benefits shown in the small phase II trial. The fact is, that when our health care system allows pricing of treatments far above any reasonable alignment with the benefits they have demonstrated for patients, we do real harm to unseen people in the health care system. Costs increase for everyone without making anyone healthier. And as costs increase, more people forgo care or drop their health insurance all together.


Even though the story played out as planned this time, the system can be improved to protect all patients and ensure affordability for everyone.

Let's see what else happened this week...

ICER in the News

Winrevair Gains Approval For Pulmonary Arterial Hypertension, But High Price Could Limit Uptake


Merck’s Winrevair was recently approved for pulmonary arterial hypertension and was priced at approximately $240,000 per year. ICER found that the treatment would reach commonly accepted thresholds for cost-effectiveness if priced between $17,900 to $35,400 per year. ICER issued an “Access and Affordability Alert” to signal to policymakers that given the population size and chosen price, the cost of this new treatment may be difficult for the health system to absorb in the short-term.

Healthcare Analyst Joshua Cohen stated:

“Since it’s highly unlikely the net yearly price of Winrevair will be close to the [ICER health-benefit price benchmark of the] $18,000 to $35,000 range, this may be considered problematic by payers and pharmacy benefit managers. To reduce their financial exposure, it’s plausible they will impose a set of utilization tools.”

State Medicaid Agencies Grapple Case By Case With Costly Gene Therapy For MLD

Inside Health Policy

The FDA recently approved Lenmeldy, a gene therapy for metachromatic leukodystrophy (MLD). ICER found the therapy would achieve common thresholds of cost-effectiveness if priced between $2.3M to $3.9M. The list price is $4.25M.

The manufacturer of the gene therapy, Orchard Therapeutics, told Inside Health Policy that it is actively negotiating with multiple states and expects the Medicaid programs to evaluate the rare disease on a case-by-case basis.

Pharmaceutical News


Net prices for medicines fell considerably in 2023’s last quarter, mostly thanks to Humira biosimilars


The net prices that health plans paid for medicines — after subtracting rebates, discounts, and fees — fell by 2.8% in last year’s fourth quarter, the largest decline ever measured by SSR Health, a research firm that tracks the pharmaceutical industry and its pricing trends.

A key reason for the big drop was pricing pressure on the Humira treatment for rheumatoid arthritis and psoriasis. Over the past year, nine biosimilar versions of the medicine were launched by other companies seeking favorable placement on formularies.

US responds to drugmakers' IRA counteroffers as Medicare price negotiations continue

Endpoints News

CMS wants further discussions with pharma companies that are participating in IRA price negotiations, HHS announced on Tuesday. The agency said it has responded to counteroffers proposed by all of the participating manufacturers but declined to comment further.

According to CMS’ guidelines, the agency will now hold up to three negotiation meetings with the companies before negotiations end Aug. 1. Drugmakers have largely kept quiet about the talks. Endpoints News reached out to participating companies on Tuesday, all of which declined to comment or did not respond.

Will Medicare raise your monthly premiums to cover Wegovy?

NBC News

Experts warn that Medicare’s recent move to cover the weight-loss drug Wegovy for some recipients with heart disease risk could drive up the cost of monthly premiums for many of the 65 million adults enrolled in the federal health insurance program.

The premium increase would depend on how many millions qualify for the medication, which has a list price of around $1,300 a month. 

Patient Out-of-Pocket Costs for Biologic Drugs After Biosimilar Competition


Researchers from Harvard's PORTAL examined the relationship between biosimilar competition and patient out-of-pocket (OOP) costs.

They found: "The introduction of biosimilar competition did not systematically lower patient OOP spending on biologics, highlighting the need for targeted policy interventions to ensure that savings from biosimilar competition improves affordability for patients."


Join ICER!

Want to work for a fast-paced, mission-driven organization? Look no further! ICER is looking to expand our team, which includes a diverse set of clinicians, researchers, and policy experts.

Health Technology Assessment

Program and Operations

Key Upcoming Dates
  • 4/10: COPD -- Draft Evidence Report

  • 5/9: MDS -- Draft Evidence Report

  • 5/14: PTSD -- Revised Evidence Report

Learn more about ICER’s ongoing and recently completed reports:


Email for more information.

When you make a gift to ICER, you help keep ICER independent.  

LinkedIn Share This Email