To Reverse Alzheimer's Disease, Biotechs Betting on Next-Gen Cell, Gene Therapies

Precision Medicine Online

Precision Medicine Online’s Jessica Kim Cohen interviewed ICER’s CSO and Director of HTA Methods and Engagement, Dan Ollendorf, about the potential for gene therapies in the Alzheimer’s disease space:

“Gene therapies have hit the market with extremely high price tags in rare disease settings but likely won't be able to demand similar prices in Alzheimer's, said Dan Ollendorf, CSO and director of health technology assessment methods and engagement at the Institute for Clinical and Economic Review (ICER), a nonprofit that conducts independent value assessments of healthcare interventions… the first treatment that shows it can halt or reverse dementia — rather than slowing cognitive decline — could warrant a higher price, Ollendorf said.”

 MUST READS

Trump demands pharma CEOs implement 'most favored nation' pricing within 60 days

Endpoints News

President Donald Trump stepped up his campaign to get large drugmakers to lower their prices, posting letters to more than a dozen major pharma companies demanding that they bring their US prices in line with what they charge abroad, as well as other steps.

Trump will give companies 60 days to comply or he will “deploy every tool in our arsenal to protect American families from continued abusive drug pricing practices.”

Sarepta resumes shipping of gene therapy Elevidys to patients who can walk

Reuters

Sarepta said on Monday it will resume shipping its gene therapy, Elevidys, to patients who can walk. The announcement came after a probe that showed an eight-year-old boy's death in Brazil was unrelated to the treatment.

 Vinay Prasad, a powerful FDA official, departs after controversy over rare disease drug

STAT

Vinay Prasad, a top official at the Food and Drug Administration departed shortly after the decision to resume sales of Elevidys was announced. Prasad was the head of the FDA division that regulates vaccines, gene therapies, and blood products. He was also the agency’s chief medical and scientific officer, making him a top adviser to Commissioner Marty Makary.

A cut to Medicare that could affect millions is buried in Trump’s tax law

STAT

Millions of older Americans living in poverty are entitled to free or heavily subsidized Medicare coverage. But the new Republican tax law will keep that benefit out of reach for many by reintroducing onerous paperwork requirements. People who get subsidized coverage through what’s known as Medicare Savings Programs get to keep hundreds of dollars per month that they can use toward rent, food, or other necessities. They can also go see their doctor or refill prescriptions more readily if they know they won’t owe anything.

The new law doesn’t eliminate these Medicare Savings Programs. But it rolls back a regulation that made it a lot easier for people to sign up.

Federal judge blocks Arkansas law barring pharmacy benefit managers from owning pharmacies in state

The Associated Press

On Monday, a federal judge temporarily blocked Arkansas’ first-in-the-nation law that would have prohibited pharmacy benefit managers from owning pharmacies in the state.

U.S. District Judge Brian Miller issued a preliminary injunction against the law restricting pharmacy benefit managers, who run prescription drug coverage for big clients that include health insurers and employers that provide coverage.

Maryland PDAB Votes Farxiga, Jardiance As Unaffordable

Inside Health Policy

The Maryland Prescription Drug Affordability Board (PDAB) voted Monday that AstraZeneca’s Farxiga and Boehringer Ingelheim’s Jardiance are both unaffordable, a step toward limiting the amount state and local governments can pay for the two popular drugs. The PDAB will next vote on whether to set an upper payment limit. 

OTHER HEADLINES

• MedPage Today: FDA 'Expert Panels' Raise Concerns of Evading Regulations, Ethics
• CNBC: Trump’s pharmaceutical tariffs could affect some drugmakers more than others
• Reuters: US Senate Democrats to investigate Kennedy's firing of vaccine expert panel
• The Washington Post: Senate confirms Susan Monarez to lead CDC
• The Wall Street Journal: The Patients Forced to Switch From Zepbound to Wegovy
• JAMA: Lowering Drug Prices in the US—Look Within, Not Abroad
• JAMA: Reducing Drug Prices—The Most Favored Nation Policy vs Price Negotiation
• Bloomberg: Astra CEO Breaks With Peers on Aligning Drug Prices Globally
• BioSpace: Drug Pricing Just One Point of Confusion Over New FDA Vouchers
• The Wall Street Journal: Trump Administration Weighs Patent System Overhaul to Raise Revenue
• Axios: Why GLP-1s could become the "everything drug"
• JAMA Health Forum: Potential Trade-Offs of Proposed Cuts to the US National Institutes of Health

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.

Program and Operations

• Operations and Program Coordinator

• 8/1: Spinal Muscular Atrophy -- Public Meeting (Register here)
• 9/2: Spinal Muscular Atrophy -- Final Evidence Report
• 9/8: Non-Cystic Fibrosis Bronchiectasis -- Evidence Report
• 9/9: Obesity Management -- Draft Evidence Report

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

• IgA Nephropathy
• Non-Cystic Fibrosis Bronchiectasis
• Obesity Management
• Smoking Cessation
• Spinal Muscular Atrophy

Email info@icer.org for more information.

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