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| | Weekly View August 5, 2022 |
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From the desk of Priya Ranade |
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Yesterday we released an Evidence Report assessing the comparative clinical effectiveness and value of AMX0035 (Amylyx Pharmaceuticals, Inc.) and oral edaravone (Mitsubishi Tanabe Pharma Development America, Inc.) for the Treatment of amyotrophic lateral sclerosis (ALS).
ICER’s Chief Medical Officer, David Rind, MD explained:
“There is tremendous need for new therapies in ALS. Current treatment of this devastating disease is largely focused on supportive care. AMX0035 appears to extend life and modestly slow disease progression, but we expect vigorous debate at the public meeting on the strength of evidence, which hinges largely on the findings from a single trial, particularly given the negative vote by the FDA Advisory Committee. We also anticipate a policy discussion on whether, should this Drug receive FDA approval, the degree of uncertainty should be reflected in a price scaled closer to the cost of production pending completion of trials that can confirm the magnitude of the benefit for patients. In this report we also evaluated the evidence on an oral formulation of edaravone. The oral version is much less burdensome to patients than the intravenous formulation, which is likely to lead to increased use, but the price of edaravone appears too high to align reasonably with the benefits it provides.”
This Evidence Report is also the pre-read for our August 19th public meeting. Register here.
Let’s see what else happened this week… |
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bluebird bio Reports Second Quarter 2022 Financial Results and Highlights Operational Progress bluebird bio |
We recently released our Final Evidence Report on the gene therapy beti-cel for beta thalassemia, which bluebird bio covered in their Q2 financial results readout. At our public meeting, the independent appraisal committee unanimously determined that evidence was adequate to demonstrate that beti-cel provided a net health benefit compared to standard clinical management. Given the high costs of standard care, our cost-effectiveness modeling found that beti-cel meets commonly accepted value thresholds at an anticipated price of $2.1 million — if that price is subject to an 80% payback for treatment failure. |
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MUST READS Democrats' backdoor crackdown on pharma "gaming" Axios |
Patient advocates, health policy experts and politicians from both parties have for years decried "thickets" of patents they say let the pharmaceutical industry game the system, flout antitrust laws and keep prices higher for longer. Democrats' drug pricing plan could end drugmakers' practice of taking out overlapping patents around one drug — a strategy which fends off competitors but that the industry argues incentivizes innovation after a drug is approved. |
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AbbVie wins appeal in antitrust case over Humira 'patent thicket' Reuters |
A U.S. appeals court on Monday affirmed that AbbVie Inc did not unlawfully block competition by building up a "thicket" of patents related to its blockbuster arthritis drug Humira. AbbVie's 132 patents related to Humira do not violate antitrust law, U.S. Circuit Judge Frank Easterbrook said Monday, rejecting claims that the patents wrongly kept generic competitors out of the market years after the original patent on Humira expired.
Even after factoring in rebates and other concessions, Americans paid an estimated $1.4 billion more on Humira in 2020 than they would have if the treatment’s net price remained where it was at the end of 2019. Back in 2017, we found that Humira would require at least a 55% discount off its list price to reach common thresholds for cost-effectiveness when used to treat rheumatoid arthritis.
Since we issued that report in 2017, Humira’s net price in the US has continued to climb. And in that timespan, we’ve seen no evidence that the treatment delivers any additional benefits for patients than what we already recognized back in 2017. Over this same three-year window, Humira’s price has been falling steadily in other countries where it faces biosimilar competition. |
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Civica Rx begins selling its first outpatient generic drug as it tries to overhaul the pharmaceutical supply chain STAT |
In the latest bid to address high prescription drug costs, a nonprofit plans to sell a cheaper generic version of an expensive cancer medicine in the U.S. Civica Rx, which was formed four years ago, is working with a generic manufacturer, Hikma Pharmaceuticals, to supply pharmacies with the abiraterone prostate cancer treatment for $160 for a month’s supply. And the nonprofit is suggesting pharmacies sell the medicine to consumers for $171, which is considerably less than what most consumers currently pay for brand-name version, Zytiga. |
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Which drugs may be hit with negotiations if the reconciliation bill passes? Wall St. analysts explain Endpoints News |
If Senate Democrats can rally every last member before next Friday, and the House pulls it together sometime next month, CMS negotiations for drug prices would begin in 2026, but only for 10 Part D drugs (i.e. outpatient drugs) to start. In 2028, Medicare would set prices for both Part D and Part B (physician-administered) drugs. Cumulatively, CMS may end up negotiating about 60 drugs, depending on generic/biosimilar competition, SVB Securities analysts wrote. |
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UnitedHealth-Change Healthcare deal trial has begun. Here are 3 things to know Fierce Healthcare |
The battle over UnitedHealth's acquisition of Change Healthcare is underway. The trial over the $13 billion deal began Monday in District of Columbia federal court. The Department of Justice (DOJ) filed suit to block the merger in February, citing antitrust concerns, a little over a year after it was first announced in January 2021. |
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Sarepta to seek early FDA approval for gene therapy to treat Duchenne muscular dystrophy STAT |
Sarepta is asking the Food and Drug Administration to approve the one-time treatment called SRP-9001 based on its ability to produce large increases in a crucial muscle protein typically missing in children born with Duchenne. Without dystrophin, muscles break down over time, which is why Duchenne patients gradually lose their ability to walk and breathe. The disease is fatal, often by the time boys are in their late teens or early 20s.The company also has preliminary data showing SRP-9001 improves muscle strength and respiratory function in patients. However, a larger and more definitive Phase 3 clinical trial that compares SRP-9001 against a placebo is still underway, with results not expected until the second half of |
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