Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction
UsernamePublicRestriction

Sickle Cell Patients Weigh Clinical Trial Participation At FDA Meeting

Executive Summary

At the fifth disease area meeting under PDUFA V’s patient-focused drug development initiative, patients with sickle cell disease talk about how they want their physicians to be more aware of potential studies.

You may also be interested in...



Case Studies: How Patient Input Is Changing R&D

US FDA's patient-focused drug development meeting on sickle cell disease inspired Global Blood Therapeutics to develop a new patient-reported outcomes instrument, while an externally led meeting on amyloidosis confirmed for Alnylam that its pivotal trial was studying clinically meaningful secondary endpoints.

“Patient Voice” Report Puts Chronic Fatigue Symptoms, Treatments In Benefit/Risk Framework

FDA report on CFS/myalgic encephalomyelitis is first of its kind from the patient-focused drug development initiative under PDUFA V. Incorporating comments from hundreds of patients and caregivers into a structured framework shows how the input may support a benefit/risk assessment for new products.

US FDA Offers More Advice On Mid-Trial Changes For Pandemic-Impacted Studies

A protocol amendment is not needed to pause enrollment to decrease potential exposure to COVID-19, and virtual visits can be immediately implemented to ensure safety; experts say pandemic is forcing a rethinking in how companies carry out trials and in how regulators ensure patient safety and trial integrity.

Topics

UsernamePublicRestriction

Register

PS055942

Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel