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A broad range of unmet needs remains in the immuno-oncology space

Pharmaceutical Technology

Immuno-oncology (IO) agents have transformed the cancer therapeutics landscape, driving long-term remissions in a subset of patients who historically had limited options. Efficacy-related unmet needs included the need for a greater long-term benefit, higher response rates, and mechanisms for overcoming resistance to IO therapies.

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Grand Rounds June 16, 2023: BeatPain Utah: Partnering With Community Health Centers Within a Socio-Technical Framework (Julie Fritz, PT, PhD, FAPTA; Guilherme Del Fiol, MD, PhD)

Rethinking Clinical Trials

The interventions include a telehealth strategy that provides a brief pain teleconsult along with phone-based physical therapy, and an adaptive strategy that provides the brief pain teleconsult first, followed by phone-based physical therapy among patients who are nonresponsive to treatment.

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Grand Rounds Biostatistics Series January 5, 2024: Methods for Handling Missing Data in Cluster Randomized Trials (Rui Wang, PhD; Moderator: Fan Li, PhD)

Rethinking Clinical Trials

When you have a non-identity link, you get that non-collapsibility issue so that other models can be better in terms of estimating. It’s important to note that these approaches are not wedded to logistic regression, as long as we have a sensible model for the missingness.

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Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. With a price tag of over $2.5 The researchers conducted a long-term follow-up study of the START trial that included patients who completed phase I of the study.

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

XTalks

Related: Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod Casgevy and Lyfgenia Pricing and Warnings The price tags of both gene therapies are steep, given the complexity of developing gene therapies and as therapies meant to be one-time treatments. Vertex-CRISPR’s Casgevy has a US list price of $2.2

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Biogen updates on its confirmatory Aduhelm trial

pharmaphorum

Aduhelm (aducanumab) was approved by the FDA in June, but has been held back by a glacial rollout as clinicians have questioned the data on which the decision to clear the drug was based, plus a $56,000 per year price tag, which has in turn led to pushback by payers.

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What challenges does the advanced therapies sector face? 

Drug Discovery World

He said: “Increasing the level of automation and standardisation is not only the way to reduce manufacturing costs, but also paves the way for follow-on approvals once a platform technology is part of an approval. Developing value-based payment models that consider long-term benefits could improve access.