Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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


No Excuses: US FDA Wants Early, Thorough Dosage Optimization For Cancer Drugs

Executive Summary

Breakthrough therapy designation is not a sufficient reason to avoid identifying an optimal dosage prior to submitting a marketing application, agency says in draft guidance under Project Optimus. Guidelines also emphasize randomized comparisons of multiple dosages and exploration of tolerability issues.

You may also be interested in...

US FDA Cancer Drug Dosage Optimization Guidance Will Increase Exposure To Ineffective Agents, NCI Says

Draft recommendations would lead to enrolling additional patients on multiple dose levels to assess activity before clinical effectiveness has been established, National Cancer Institute says; industry cites need for flexibility in recommending randomized studies of multiple dosages and questions utility of PRO data in early dose finding studies.

Build Patient-Reported Outcomes Into Cancer Drug Dose Optimization, US FDA Says

In exploratory FDA analyses, PRO data appeared more sensitive at detecting an exposure-toxicity relationship for an oral small molecule cancer drug than clinician-reported data; Project Optimus representative dispels industry concerns that FDA wants firms to find the 'mythical' optimal dose.

US FDA Uses PI3K Inhibitor Experience To Spell Out What It Wants In Dose Optimization Studies

Advisory committee review of safety issues with the PI3K inhibitor class of drugs for hematologic malignancies served as a platform for the Oncology Center of Excellence’s Project Optimus initiative, which emphasizes an earlier and more thorough assessment of different doses, including through randomized trials.

Related Content


Latest Headlines
See All



Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

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