Remove tag patient-access
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Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

Historically, the pharmaceutical industry has failed to meet the needs of this patient population. Most famously, the US passed its Orphan Drug Act in 1983, providing innovators with financial motivation to develop orphan drugs and meet the needs of these forgotten patients. Efforts have been made to change this.

Drugs 147
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Risk-sharing agreements are growing at a rate of 24%

Pharmaceutical Technology

This is not the first treatment to come with a high price tag. The three agreed on a confidential price, and the TLV determined whether the drug was cost effective for the eligible patient population. million, according to GlobalData’s Price Intelligence (POLI), making it the most expensive drug in the world.

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Free access to Zolgensma curbed, says Novartis

pharmaphorum

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 Pause in UK access.

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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

UPMC did not allow patients to receive experimental COVID-19 therapies outside of the context of a clinical trial and used the REMAP-CAP platform, a global pragmatic adaptive trial platform, in all clinic sites. Every patient with COVID-19 was automatically considered. Patients were screened by local teams at each site.

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Grand Rounds Ethics and Regulatory Series November 11, 2022: Data Sharing and Pragmatic Clinical Trials: Law & Ethics Amidst a Changing Policy Landscape (Stephanie Morain, PhD, MPH; Kayte Spector-Bagdady, JD, MBioethics)

Rethinking Clinical Trials

Additionally, in August 2022, the latest federal data-sharing memorandum, Ensuring Free, Immediate, and Equitable Access to Federally Funded Research Memorandum (2022 OSTP Memo), was released requiring articles resulting from federal funding be made “freely available and publicly accessible” without embargo or delay.

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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. Although a subset of patients derives long-term benefits from IO therapies, a high percentage of patients still fail to respond or develop resistance to IO therapy.

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

Pharmaceutical Technology

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. AZ: What is the ISCT doing to tackle supply and access-related obstacles in the CAR-T field?