An approach to data collection in compassionate use/managed access

Review Medicine, General & Internal

Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals

Tobias B. Polak et al.

Summary: This study aims to quantify and characterize the usage of expanded access (EA) data in National Institute for Health and Care Excellence (NICE) technology appraisals. The study found that over one in five appraisals used EA data, which can meaningfully inform safety, efficacy, and resource use analyses in various disease areas.

BMJ OPEN (2022)

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Article Pharmacology & Pharmacy

Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations

Tobias B. Polak et al.

Summary: This article discusses the potential value of expanded access (or compassionate use) for patients with rare diseases and its supportive role in decision-making. The authors recommend harmonization of expanded access regulations across European countries to reduce burden on manufacturers, improve patient access, and obtain better data.

FRONTIERS IN PHARMACOLOGY (2022)

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Article Health Care Sciences & Services

International Country-Level Trends, Factors, and Disparities in Compassionate Use Access to Unlicensed Products for Patients With Serious Medical Conditions

Paul Aliu et al.

Summary: The presence and public availability of compassionate use regulations, as well as an increase in local clinical trial activity, are associated with higher rates of compassionate use requests across countries. Despite the free provision of unlicensed therapeutic products, there is a correlation between a country's economic development and its request activity for unlicensed compounds via compassionate use.

JAMA HEALTH FORUM (2022)

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Editorial Material Pharmacology & Pharmacy

Increasing Use of Compassionate Use/Managed Access Channels to Obtain Medicines for Use in COVID-19

Paul Aliu et al.

Summary: The COVID-19 pandemic has created a demand for new therapeutics, leading to the widespread use of unapproved/repurposed medicines. Some countries have regulations for access to these medicines, but a better understanding of these channels is needed as they continue to expand.

CLINICAL PHARMACOLOGY & THERAPEUTICS (2021)

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Article Medicine, General & Internal

JAK Inhibition with Ruxolitinib in Patients with COVID-19 and Severe Pneumonia: Multicenter Clinical Experience from a Compassionate Use Program in Italy

Alessandro Maria Vannucchi et al.

Summary: This study reviewed the clinical outcomes of 218 severe COVID-19 patients treated with ruxolitinib, a Jak inhibitor. The results showed that patients experienced improvements in symptoms and reduced oxygen support requirements after treatment. Additionally, significant decreases in C-reactive protein and increases in lymphocyte total count were observed, with an overall survival rate of 87.2% and no unexpected safety findings.

JOURNAL OF CLINICAL MEDICINE (2021)

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Article Pharmacology & Pharmacy