Change the Guide RNA: Is It Still the Same Medicine?
An FDA draft guidance explores the regulatory boundaries of individualized gene-editing products.
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FOLLOW A QUESTION
Regulation & approval
Explore approval standards, regulatory policy, and the evidence that continues to develop after a medicine reaches patients.
Avacopan Update: A $69 Million Securities Settlement Is Approved, but Patients’ Questions Remain
Funds are in escrow, while payment still awaits the required procedures. Patient compensation, accountability, and the FDA withdrawal process must be tracked separately.
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Why Are Approved Drugs Withdrawn?
Unreliable evidence, unconfirmed benefits, and newly identified safety risks can all lead to withdrawal. The reasons behind these decisions need separate examination.
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Avacopan: When a Trial Goes Wrong, Why Do Patients Still Use the Drug?
Patients’ experiences of improvement, the credibility of a trial, and continued market approval each call for different evidence. The avacopan case shows why none can substitute for the others.
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Relyvrio: How Much Certainty Is Enough When Patients Cannot Wait?
When disease leaves little time to wait, how should earlier access to treatment be weighed against the costs of uncertain efficacy?
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Vioxx: Why Withdraw a Drug That Relieves Pain?
Pain relief and new harm can coexist. How should the assessment of benefit and risk change when both become visible?
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Bevacizumab: Why Was Delayed Progression Not Enough to Keep This Approval?
Why does withdrawal of one indication differ from withdrawal of an entire medicine? From progression-free survival to patients’ lives, this case reexamines treatment value.
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Mylotarg: Why Did a Withdrawn Drug Return Seven Years Later?
When the molecule stays the same but the dose, regimen and eligible patients change, what new evidence is needed?
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After a drug is withdrawn, who bears patients’ losses?
Patient relief, investor settlements, and transition supply address different problems. Public records show how losses are handled.
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Amgen’s Tavneos: When Approval No Longer Settles the Evidence
Examine how trial reliability, regulatory proceedings, and patient questions reshape the assessment of an acquired drug.
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J&J’s Rybrevant Beyond Lung Cancer: What the New Approval Changes
An accelerated approval opens a defined new indication. Response durability, confirmatory evidence and real adoption will shape its lasting value.
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