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Expediting innovation in the rare disease domain

pharmaphorum

Here are the challenges that the medical community faces, most of which relate to the lack of research data, high cost of developing treatments or orphan drugs and greater chances of failure. . Small sample size in trials: Finding eligible patients for clinical trials of orphan medicines is a big challenge.

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Rare Disease Day and a Focus on Quality

PQA

The economic burden of rare disease reached nearly $1 trillion in 2019, and the average drug approved under the Orphan Drug Act costs nearly $120,000 per year. A measure developer, researcher, educator and convener, PQA’s quality initiatives support better medication use and high-quality care.

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Why Is Nobody Using Your Guidelines? – Insights On Designing Useful Guidelines For Infectious Diseases

IDStewardship

Additionally, the Centers for Medicaid & Medicare Service (CMS) Center for Clinical Standards and Quality/Quality, Safety & Oversight Group released updates to interpretive guidance for hospital requirements, making their antimicrobial stewardship regulations live now.

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