Remove tag value-based-pricing
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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing.

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Amylyx ALS drug draws criticism over $158,000 price tag

pharmaphorum

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

Drugs 52
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Grand Rounds June 2, 2023: PROACT Xa and The Wizard of Oz: Behind the Curtain of a Pragmatic Decentralized Clinical Trial (John Alexander, MD, MHS)

Rethinking Clinical Trials

Sites were selected based on their On-X AVR volume, and the study leveraged the surgeon/patient relationship for recruitment. Several sites could enroll patients from outside of their system, based on regional or state requirements from local IRBs. The price for Apixaban was $600 per bottle/$6M, and the price for warfarin was $50K.

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Why Rao’s Sauce Is Worth Billions, According to Campbell’s

XTalks

In August, Campbell Soup acquired Rao’s sauce owner Sovos Brands for $23 per share, reflecting a total value of a staggering $2.7 A noteworthy achievement is volume-driven growth, particularly in an era where growth is often price-driven. At $8 per jar, Rao’s sauces come at a much higher price point than fellow Campell’s brand Prego.

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FDA Trims Broad Label for Biogen’s Alzheimer’s Drug Amidst Harsh Criticism

XTalks

The FDA based the decision on the fact that there is “no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.”. The FDA’s rationale for granting the approval was based on the function of the drug as an amyloid beta targeting antibody.

Drugs 98
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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US. Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. Vertex-CRISPR’s Casgevy has a US list price of $2.2 Vertex-CRISPR’s Casgevy has a US list price of $2.2

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What challenges does the advanced therapies sector face? 

Drug Discovery World

He said: “The industry, as a whole, needs to carefully consider the true value of curing lifelong genetic or degenerative diseases, particularly when evaluating novel therapeutics that present unique treatment opportunities.” Pricing and reimbursement continues to be a challenge we address as a field.