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Grand Rounds February 2, 2024: Strategies for Improving Public Understanding of FDA and the Products It Regulates…Why Should We Care, and What Might We Do? (Susan C. Winckler, RPh, Esq)

Rethinking Clinical Trials

Food and Drug Administration (FDA) Commissioner Robert Califf asked the Reagan-Udall Foundation to conduct research and consult with experts to better understand how consumers find, consume, and perceive health information, especially regarding FDA-regulated products.

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First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

The clinical trial will be led by Professor Rob Wynn, Consultant Paediatric Haematologist at Royal Manchester Children’s Hospital (RMCH), together with Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease at Saint Mary’s Hospital, and Professor Bigger.

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Grand Rounds January 12, 2024: Design and Implementation of a Weighted Lottery to Equitably Allocate Scarce Covid-19 Resources (Erin K. McCreary, PharmD, BCIDP)

Rethinking Clinical Trials

Clinicians wrote a consult for mABs and the pharmacy delivered whichever mAB that was available. Tags #pctGR, @Collaboratory1 The post Grand Rounds January 12, 2024: Design and Implementation of a Weighted Lottery to Equitably Allocate Scarce Covid-19 Resources (Erin K. Many patients did not answer the phone.

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Grand Rounds December 8, 2023: A Pragmatic Randomized Trial of the Jumpstart Intervention to Promote Communication about Goals of Care for Hospitalized Patients with Serious Illness (Ruth Engelberg, PhD; Erin Kross, MD; Robert Lee, MD, MS)

Rethinking Clinical Trials

Tags #pctGR, @Collaboratory1 The post Grand Rounds December 8, 2023: A Pragmatic Randomized Trial of the Jumpstart Intervention to Promote Communication about Goals of Care for Hospitalized Patients with Serious Illness (Ruth Engelberg, PhD; Erin Kross, MD; Robert Lee, MD, MS) appeared first on Rethinking Clinical Trials.

Trials 130
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bluebird bio to split into oncology and gene therapy specialists

pharmaphorum

million price tag. Ibrahim is currently serving as a consultant for the Parker Institute for Cancer Immunotherapy (PICI) and built the clinical capabilities within the institute. Zynteglo is already in beta-thalassemia in Europe, where the company will seek to expand access despite its hefty $1.8

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After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

pharmaphorum

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S. Professional reception of the findings.

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NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

pharmaphorum

” NICE asks for comments on its Appraisal Consultation Document (ACD) for #Risdiplam : [link] pic.twitter.com/SILXW9QFhZ. million price tag make it is the most expensive treatment ever approved for NHS funding, as it provides “exceptional benefit” to babies with the disease.