Remove tag topical-treatment
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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

Despite their potential multi-million-dollar sticker price, if haemophilia treatments by BioMarin and CSL Behring prove durable, they could help patients save money compared to current treatments, experts note. A fair price for CSL Behring’s haemophilia B treatment etranacogene dezaparvovec would be between $2.93–2.96

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Cartherics grants licence for CTH-004 to Shunxi

Pharmaceutical Technology

CTH-004 is developed by genetically altering patient T cells for inserting a chimeric antigen receptor (CAR) to target a marker (TAG-72) on ovarian cancer cells and delete genes which are involved in T cell function suppression. Topic sponsors are not involved in the creation of editorial content.

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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. As per ICER, if a treatment improves quality of life or extends a patient’s life, then this can be represented in additional QALYs provided by the therapy.

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Grand Rounds September 29, 2023: Navigating the Use of Patient-Reported Outcomes in Research and Practice: The PROTEUS Consortium (Claire Snyder, PhD; Norah Crossnohere, PhD; Anne Schuster, PhD)

Rethinking Clinical Trials

The PROTEUS Consortium’s objective is to ensure that patients, clinicians, and other decision-makers have high-quality PRO data from clinical trials and clinical practice to make the best decisions they can about treatment options. Can you elaborate on the incentives and marketing recommendation from the Underserved Advisory Group?

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Almirall and IRB Barcelona team up to tackle skin disease

pharmaphorum

Two Spanish pharma companies are joining forces to identify new oral treatments for immune-inflammatory skin diseases with high unmet medical needs. Almirall and IRB Barcelona (the Institute for Research in Biomedicine), will conduct research using molecular glue degraders, a new approach to the skin diseases under scrutiny.

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Grand Rounds September 23, 2022: Effect Of An Intensive Nurse Home Visiting Program On Adverse Birth Outcomes In A Medicaid-Eligible Population (Margaret McConnell, PhD)

Rethinking Clinical Trials

98% of participants enrolled in the treatment group received at least one visit from NFP. Visits were mostly spent on personal health and maternal role topics. The median home visit length was 65 minutes. Some referrals were made for general services (25%) and health care (23%). pctGR, @Collaboratory1.

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Q&A: A decade on, what’s next for CAR-T therapies?

Pharmaceutical Technology

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. This approach remains logistically complex, which builds the desire for allogeneic treatments that can be manufactured from unrelated donors on a larger scale.