Regulatory & Medical Writing

Meet Pharma Transparency Mandates & Engage Study Participants

Behtash Bahador

Most clinical study participants want to know the results of trials they participated in. However, few are actually informed. Lack of communication and transparency jeopardizes the success of the clinical research enterprise. In this blog post, I’ll discuss a new model for communicating trial results in lay language with minimum added burden on sponsors and […]

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Topics: Regulatory & Medical Writing

Can QSP Save Lives? Lessons from a Trial Debacle

Neil Benson

The notion that volunteers could be harmed in a clinical trial is every drug developer’s worst nightmare. Earlier this year, the drug company, Bial, investigated inhibitors of the enzyme fatty acid amide hydrolase (FAAH) in clinical trials as a treatment for pain. Tragically, one person in the volunteer group died, and six patients were hospitalized. […]

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Topics: Systems Pharmacology

How to Expedite FDA Approvals of Orphan Drugs

Thomas Peyret

350 million patients worldwide suffer from 7,000 rare diseases, yet only 300 of these diseases have approved treatments. This gap, impacting 95% of rare disease patients, represents a huge unmet medical need. Developing drugs for rare diseases poses a range of clinical, regulatory and commercial challenges. The small number of patients are difficult to identify […]

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Topics: PK/PD Modeling & Simulation

FDA’s PDUFA VI Goals Highlight Model-informed Drug Development

Ellen Leinfuss

On July 15, the US FDA published its goals and commitment letters for the re-authorization of its Prescription Drug User Fee Act (PDUFA) for fiscal years 2018-2022, known as PDUFA VI. The document reflects the agency’s performance and procedural goals to expedite bringing safer therapies to patients.  It also reflects and incorporates the advances in […]

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Topics: PBPK Modeling & Simulation