Sunday, June 23, 2024

Elevidys in DMD: Approved Despite Trial Failed in Primary Efficacy Endpoint

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The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy ( Du...
Wednesday, June 19, 2024

Functional Unblinding in Double-blind, Randomized, Controlled Trials (RCTs)

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Earlier this month,  FDA advisory committee declined to endorse Lykos Therapeutics’ application to market its psychedelic drug MDNA, also k...
Sunday, May 19, 2024

Master protocol: umbrella-trial-based platform trial versus basket-trial-based platform trial

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Biotech company Aerami Therapeutics announced that they are going to design a platform trial to test their pulmonary hypertension drug simul...
1 comment:
Thursday, May 16, 2024

Forward: When Worlds Collide: The Theory of Real-World Evidence Meets Reality

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 A good article from The FDA Law Blog :                When Worlds Collide: The Theory of Real-World Evidence Meets Reality
1 comment:
Monday, May 06, 2024

eIND: exploratory IND versus emergency IND

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eIND may be referred to as either exploratory Investigational New Drug (IND) or emergency Investigational New Drug (IND). An "Explorato...
Monday, April 29, 2024

Phased clinical trials, seamless clinical trials, phaseless clinical development process

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The drug development process encompasses a series of phased clinical trials, typically categorized as phases 0 (optional), 1, 2, 3, and 4. P...
Thursday, April 25, 2024

Phased Clinical Trials - Phases 0, 1, 2, 3, 4

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Clinical development programs for drugs and biological products include phased clinical trials ranging from Phase 0, 1, 2, 3, and 4.  Phase ...
Sunday, April 21, 2024

Expanded access, compassionate use, and eINDs

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Traditionally, drug approvals relied on at least two adequate and well-controlled studies, each convincing on its own, to establish effectiv...