Institute for Clinical and Economic Review Releases Draft Evidence Report on Treatments for Friedreich’s Ataxia

ICER released a Draft Evidence Report assessing the comparative clinical effectiveness and value of two new treatments: nomlabofusp (Larimar Therapeutics, Inc.) and omaveloxolone (Skyclarys®, Biogen Inc.) for Friedreich’s Ataxia, a rare, genetic disorder.  ICER will be accepting public comments on this draft until November 11th.

This preliminary draft marks the midpoint of ICER’s eight-month process of assessing this treatment, and the findings within this document are not ICER’s final conclusions.

The COVID Vaccine for Pregnant Women

Sensible Medicine

After Dr. Vinay Prasad stated on Sensible Medicine that pregnant women should “absolutely not” get the Covid-19 vaccine, ICER’s Chief Medical Officer, Dr. David Rind, wrote a commentary for Sensible Medicine disagreeing with Dr. Prasad. Dr. Rind argued that currently, the best available evidence suggests infants from ages 0-6 months are best-protected from Covid-19 when pregnant women receive the Covid-19 vaccine in the third trimester.

ICER completed a full comparative clinical and cost- effectiveness assessment of the Covid-19 vaccine this year. When explaining ICER’s report findings, Dr. Rind, stated: “For any preventive care, the goal is always to understand whether the potential benefits outweigh the potential harms. ICER evaluated the evidence for Covid-19 vaccines in 12 US sub-populations. Current evidence suggests that the greatest net benefit of Covid-19 vaccination is in pregnant women; infants aged 6 months to one year; and adults over the age of 65, with the net benefit increasing further with increasing age.”

Drs. Prasad and Rind then posted rebuttals. You can check out the back and forth, here. 

Can Real-World Evidence Reliably Fill Evidence Gaps In Accelerated Approval?

Health Affairs

Researchers from Tufts CEVR examined the role of RWE in accelerated approval:

"More attention by regulators and payers is warranted around how the growing volume of RWE can most effectively address critical evidence gaps. Sponsors of RWE studies should be held to best practice recommendations reflected in numerous and aligned guidelines from consensus panels and the FDA’s own guidance documents for transparency, credibility, and reproducibility. These include clearly defining research questions so that they target the uncertainties that are most important to clinicians and patients; pre-specifying and publicly posting study designs and analysis plans; using fit-for-purpose data; using appropriate analytic and validation approaches; conducting sensitivity analyses; and carefully anticipating and addressing questions around bias, confounding, and generalizability. "

They also refer to a white paper that ICER and Verdant Research published earlier this year titled, “Strengthening the FDA's Accelerated Approval Pathway: Progress and Unfinished Business,” which reviews recent regulatory and market changes and identifies several policies that could be adopted to further improve the pathway. 

Biotechs Are Betting on Gene Therapy for Heart Disease, Obesity; Will Patients?

Precision Medicine

Precision Medicine’s Jessica Kim Cohen investigated the possibility of developing gene therapies for more prevalent conditions, like cardiovascular disease. She spoke with ICER’s Chief Medical Officer, David Rind, MD, MSc:

“For a gene therapy, ‘often the cost is upfront,’ Rind said. That can present a budget problem for payors if many people are seeking treatment, especially since benefits are accrued over the long run, maybe decades into the future. That challenge has already been observed with GLP-1 drugs, where health plans have rolled back coverage as they look to trim budgets.

There are lots of options for addressing cardiovascular risk factors, so patients will want to be confident that the benefits they expect to glean from a gene therapy outweigh not only potential adverse events that could be seen long into the future, but also standard care that’s already available. That makes running large clinical trials to characterize long-term outcomes critical, Rind said.

‘It’s not enough to show that your gene therapy reduces cholesterol,’ he said. The gene therapy will need to be significantly better than statins to justify a significantly higher cost.”

MUST READS

Could Medicare Price Negotiation Tip The Scales Toward Part B Drug Payment Reform?

Pink Sheet

Implementing Medicare negotiated maximum fair prices for physician administered drugs covered under Part B in 2028 may finally drive lawmakers to revise the 22-year-old reimbursement approach that pays providers more when they use higher-cost drugs.

Beginning in 2028, the average sales price plus 6% formula will be replaced with a Medicare maximum fair price plus 6% formula for negotiated Part B drugs. The reimbursement decrease could mean providers avoid prescribing drugs with negotiated prices in favor of more expensive products considered therapeutically equivalent to maintain their margins.

More than 3 million Medicare Advantage enrollees need to find new plans for 2027 in third year of turmoil

CNN

Medicare Advantage, which covers just over half of Medicare’s roughly 71 million enrollees, is going through its third year of turmoil as health insurers overhaul their offerings. The changes are largely being spurred by medical costs outpacing reimbursements from the federal government, which pays insurers to provide coverage to Medicare enrollees – though critics say that insurers are overbilling for patient care.

Many patient, consumer reps on FDA advisory panels have ties to groups funded by pharma, analysis finds

STAT

Nearly 40% of Food and Drug Administration advisory committee meetings held over a recent 20-year period included either a patient or consumer representative with active ties to an advocacy group funded by pharmaceutical companies, according to a new analysis. And for several committees, the number of such reps exceeded 50%.

Poll: 43 percent support ‘Medicare for all’

The Hill

Nearly 43 percent of Americans support the idea of “Medicare for all,” according to an Axios/Ipsos poll released Tuesday, as concerns over health costs mount ahead of the midterm elections. 

People who said they support Medicare for all outnumbered its opponents by a nearly 2-to-1 margin, while a bipartisan majority of Americans said they think the federal government should be willing to spend more tax dollars to lower healthcare costs. 

OTHER HEADLINES

• Annals of Internal Medicine: Out-of-Pocket Savings Among Medicare Part D Beneficiaries Under the Medicare Drug Price Negotiation Program
• The Wall Street Journal: FTC, States Ask Court to Hold ‘Pharma Bro’ Shkreli in Contempt for Trading Drug Stocks

Key Upcoming Dates

• 10/20: ICER, West Health Launch Price and Access — Final Report
• 10/27: Launch Price and Access — Webinar (Register here)
• 10/29: Hypertension — Public meeting (Register here)

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

• Friedreich’s Ataxia
• Huntington's disease
• Hypertension
• Parkinson's Disease

Email info@icer.org for more information.

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