America will struggle to pay for ultra-expensive gene therapies

The Economist

The Economist examined several highly-priced gene therapies recently launched in the US market, including Zolgensma, Zynteglo, and Hemgenix -- all of which have been reviewed by ICER. They also cited ICER's Draft Evidence Report on treatments for sickle cell disease.

ICER estimates offer warning over NASH Drug prices

The Pharma Letter

ICER’s Final NASH Report Urges Step Therapy, Specialist Prescribing, Equitable Access

Scrip

Last week, ICER released a Final Evidence Report assessing the comparative clinical effectiveness and value of resmetirom (Madrigal Pharmaceuticals, Inc.) and obeticholic acid (Ocaliva®, Intercept Pharmaceuticals, Inc.) for non-alcoholic steatohepatitis (NASH).

ICER analyses suggested resmetirom would achieve common thresholds for cost-effectiveness if priced between $39,600 – $50,100 per year if short-term effects on liver fibrosis translate into longer-term reductions in cirrhosis. Under the same assumptions, obeticholic acid would achieve common thresholds for cost-effectiveness if priced between $32,600 – $40,400 per year.

MUST READS

Just how vulnerable is Medicare drug negotiation to legal challenges?

STAT

Drugmakers have already hinted at suing Medicare over its new efforts to negotiate prescription drug prices. A new report by nonpartisan congressional researchers suggests there are at least some parts of the law that can’t be challenged — but they caution it would ultimately be up to the courts to decide.

When they laid out the new rules for Medicare drug price negotiation, Democrats tried to get the program up and running before a future Republican administration could dismantle it. To speed things up, the law directs government officials to forgo the usual public rulemaking that allows for industry feedback, and it restricts the courts from reviewing aspects of that process, including the Drugs chosen for negotiation and the maximum price that Medicare will pay for them.

The Congressional Research Service, the research arm of Congress, said some challenges to Medicare’s actions could be exempt from judicial review, but it is not clear that all actions will be exempt.

Four ways to make drug price negotiations work for everyone  

The Hill

Researchers from the USC Schaeffer Center for Health Policy & Economics provided several recommendations for CMS’ upcoming drug price negotiations -- including encouraging the use of evidence-based pricing right after launch:

“The IRA orders drug manufacturers to pay a rebate to CMS if their average prices increase faster than the consumer price index. One likely result will be companies setting the highest possible prices at launch…To avoid this and make negotiations easier and fairer, CMS could exempt new drugs from the inflation rebate provision in favor of a three-part pricing framework. New drugs would first undergo an ‘evaluation phase’ characterized by a low launch price which would improve uptake in the short term and accelerate the gathering of real-world evidence regarding the drug’s effectiveness.

During a subsequent ‘reward phase,’ the drug’s price would adjust depending on what the new evidence shows. Finally, an ‘access phase’ would utilize robust generic competition to accomplish the IRA’s intended goal of lower drug prices and improved patient access in the long term.”

Health Insurer Strategies to Reduce Specialty Drug Spending-Copayment Adjustment and Alternative Funding Programs

JAMA

Researchers from Vanderbilt University analyzed the issues with copayment accumulators and maximizers for pricey specialty therapies. They stated:

“Payers have responded to high drug prices by requiring patients to pay a greater share of drug costs through deductibles or coinsurance…Both copayment accumulators and copayment maximizers are designed to ensure that copayment support funds are not used to pay the patient’s deductible or count toward their out-of-pocket maximum while also ensuring that the PBM obtains the full financial value of the copayment assistance program. These copay adjustment savings accrue to the PBMs, health plans, and employers, not to patients…

Given the increase in the use of copay adjustment and alternative funding programs and the manufacturers’ responses, patients with commercial insurance who need specialty drugs may have difficulty obtaining coverage for them or be unable to obtain them at all.”

A short supply of cancer drugs has doctors and patients worried: 'We're at a critical juncture'

USA Today

A drug shortage is forcing doctors to reduce doses of chemotherapy. Experts want the U.S. government to protect the drug supply so that over 100,000 cancer patients can get life-saving drugs on time. Fourteen cancer drugs have been in shortage in recent months, mostly because of supply chain issues. Those in the shortest supply include cisplatin and carboplatin, platinum-based drugs used to treat gynecologic, breast, testicular, bladder, head and neck, and non-small cell lung cancers.

The American Society of Clinical Oncology (ASCO) has been working to resolve the shortage and saw the first glimmers of hope last week after a national delivery of carboplatin, which has been in shortage for a month. 

OTHER HEADLINES

• STAT: Coherus works with Mark Cuban to sell biosimilar Humira at steep discounts

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• The Washington Post: Eli Lilly settles insulin suit for $13.5 million, agrees to keep price cap

• Kaiser Family Foundation: Many People Living in the ‘Diabetes Belt’ Are Plagued With Medical Debt

• The Hill: Millions had Medicaid coverage tied to the pandemic. Now they stand to lose it

• Pink Sheet: Big Pharma Companies Have Higher US FDA Approval Percentage Than Everybody Else

• Axios: Court ruling casts long shadow over future opioid lawsuits