Cancer Moonshot FDA Guidelines Update


In 2016, then-Vice President Joe Biden launched the Cancer Moonshot to accelerate progress against cancer. On February 2, 2022, President Biden announced plans to reignite this initiative. The revised effort aims to reduce the death rate from cancer by at least 50% over the next 25 years and improve the experience of patients and their families living with and surviving cancer; and, in doing so, the effort hopes to end cancer as we know and understand it today.

Following the President’s announcement on February 2, on March 1 the FDA issued three final guidances to the industry regarding cancer clinical trials that align with the new goals of the Cancer Moonshot effort. These guidances support the initiative's goal to facilitate continued advancement in cancer prevention, detection, research, and patient care. They are:

1. Inclusion of Older Adults in Cancer Clinical Trials

This guidance provides recommendations to sponsors and institutional review boards for including older adult patients (those aged 65 and older) in the clinical trials of drugs for treating cancer. Emphasis is placed on the importance of including adults over age 75 in clinical trials, and recommends including an adequate representation of older adults in cancer clinical trials to better evaluate the benefit-risk profile of cancer drugs in this age group.
Read final guidance | View federal register notice

2. Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics

This guidance finalizes draft guidance of the same name issued in August 2018. It provides advice on designing and conducting trials with multiple expansion cohorts that allow for concurrent accrual of patients into different cohorts to assess safety, pharmacokinetics, and antitumor activity of first-in-human cancer drugs. Pharmaceutical companies and researchers can use trials with expansion cohort design to evaluate different aspects of a drug in a single clinical trial, expediting the clinical development of the drug.
Read final guidance | View federal register notice

3. Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics

This guidance provides advice to sponsors of drugs and biologics for cancer treatment regarding the design and conduct of clinical trials, other than first-in-human trials, intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers. With traditional approaches to generating evidence becoming more expensive and challenging to execute, answers to important clinical questions are often delayed. Well-designed master protocols can provide answers more quickly and efficiently than traditional clinical trials. The guidance also advises on the information that sponsors should submit to FDA, and how sponsors can best interact with FDA to facilitate efficient review.
Read final guidance | View federal register notice

As Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence, noted in an FDA press release, “FDA is recommending important principles that involve addressing inequities, targeting the right treatments to the right patients, speeding progress against the most deadly and rare cancers, and learning from the experience of all patients.” With the renewed push from the White House, the industry should keep a close watch on the Cancer Moonshot and prepare for research and guidance to evolve as the initiative expands its scope.

The refocus on cancer research and treatments inspired by the Cancer Moonshot is a unique opportunity for the industry, though navigating these complex regulatory waters poses a definite challenge. A trusted partner like the seasoned experts at The Bracken Group can provide biopharmaceutical consulting in the development of oncology drugs, biologics, and the use of medical imaging in oncology clinical trials. Our team can also assist in preparing a regulatory strategy, interacting with regulatory authorities, and the submission processes. Learn more about our services and contact us today.


Subscribe to receive more content