Verona prices lung disease therapy above expectations at $2,950/month

Reuters

With FDA nod, Verona's Ohtuvayre is set to Jack up the COPD market

FiercePharma

On Wednesday, the FDA approved ensifentrine (branded Ohtuvayre) for chronic obstructive pulmonary disease (COPD). ICER recently released an Evidence Report on ensifentrine, and ICER's Chief Medical Officer David Rind, MD stated:

“COPD is a common cause of severe respiratory problems. People with COPD can experience persistent shortness of breath and fatigue that can significantly affect their daily activities. Current evidence shows that ensifentrine decreases exacerbations when used in combination with some current inhaled therapies and is well-tolerated by patients, but there are uncertainties about how much benefit it may add to unstudied combinations of inhaled treatments.”

We found that the therapy would meet traditional thresholds of cost-effectiveness if priced between $7,500 to $12,700 per year. Ohtuvayre's list price is $35,400 per year.

The Next Drug Price Dilemma

Boston Business Journal

Boston Business Journal’s Hannah Green analyzed gene therapies designed for smaller patient populations and the potential impact on insurers.

Last year, ICER published a Final Evidence Report for a gene therapy indicated for a rare disease. We assessed the comparative clinical effectiveness and value of atidarsagene autotemcel (branded Lenmeldy) 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.

 MUST READS

Peter Marks single-handedly approved Sarepta’s gene therapy — what does that mean for everyone else?

Endpoints News

The FDA recently announced it was expanding the allowed use of Sarepta’s gene therapy for Duchenne muscular dystrophy to a far wider group of patients. The treatment, Elevidys, will now be available to those ages 4 and older, an expansion that’s expected to make it available to 80% of patients with the disease, according to the company.

But the move by the FDA, in which CBER Director Peter Marks overruled his own staff, is prompting new questions about just how the agency will approach similar drugs in the future.

Biden administration touts lower costs for 64 prescription drugs

The Hill

The Biden administration on Wednesday announced some Medicare beneficiaries will save money on 64 prescription drugs for the third quarter of this year. The Inflation Reduction Act (IRA) requires drug companies to pay rebates to Medicare when prices increase faster than the rate of inflation for certain drugs.

FDA publishes long-awaited guidance on clinical trial diversity

Endpoints News

As the White House kicked off a public forum on clinical trials on Wednesday, the FDA simultaneously published a delayed draft guidance on ensuring clinical trials become diverse. The draft guidance outlines how biopharma companies can meet a new requirement to submit diversity action plans to increase enrollment of historically underrepresented populations.

The plans, which were created by the passage of the Food and Drug Omnibus Reform Act in 2022, must include the sponsor’s goals for enrollment, disaggregated by race, ethnicity, sex, and age of clinically relevant study populations; a rationale for such goals; and an explanation of how the sponsor intends to meet such goals.

Separately, as a part of ICER’s Value Assessment Framework Update in September 2023, ICER also pledged to develop methods to evaluate the demographic diversity of clinical trials. Now, the ICER-developed Clinical Trial Diversity Rating (CDR) tool is available to be downloaded on ICER.org. The CDR tool shares ICER’s framework for evaluating the demographic diversity of clinical trial populations in a consistent, transparent manner leading to an overall rating.

Mail-Order Drugs Were Supposed to Keep Costs Down. It’s Doing the Opposite.

The Wall Street Journal

A key tool that businesses have counted on to keep a lid on employees’ drug spending—filling workers’ prescriptions by mail—is now driving up their costs. 

Unity Care NW, a nonprofit health clinic in Washington state, forecasts the cost of medical and drug benefits for its 365 employees and their family members will increase this year by 25% to more than $3 million. A big reason: drugs delivered by mail are costing multiples more than those picked up at a store counter. Markups were as much as 35 times higher than what other pharmacies charged, according to a recent analysis of millions of prescriptions in Washington state.

US Medicare Agency Reconsidering Proposed Accelerated Approval Payment Demo

Pink Sheet

CMMI proposed paying less for drugs that have not yet confirmed clinical benefit after accelerated approval as one of a series of demos unveiled in 2023 in response to a White House executive order calling for further actions to curb drug pricing.

However, CMMI Director Elizabeth Fowler indicated that the new authorities granted to the FDA as part of the 2022 FDA Omnibus Reform Act prompted the two agencies to reconsider whether a payment mechanism still is needed to encourage the completion of confirmatory trials.

OTHER HEADLINES

• Axios: Smallest businesses feel crush of health care costs

• STAT: House panel proposes new strategy for Medicare to cover weight loss drugs Wegovy, Zepbound

• The New York Times: In States That Won’t Pay for Obesity Drugs, ‘They May as Well Have Never Been Created’

• The Associated Press: Arkansas sues 2 pharmacy benefit managers, accusing them of fueling opioid epidemic in state

• Bloomberg: Boehringer Faces Skeptical Judge in US Drug Price Plan Challenge

• The Hill: Democratic senator calls on HHS to investigate PBMs blocking access to generic cancer drug

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