Cancer Moonshot Launches in 2017

Cancer Moonshot Launches in 2017

This blog post covers recent developments in U.S. federal policy regarding cancer diagnostics and therapies. For more information regarding the U.S. molecular cancer diagnostics market, please consultThe World Market for Molecular Diagnostics, 7th Ed.published this week by Kalorama Information.

Rare bipartisanship at the end of the Obama administration led to the passage of the 21st Century Cures Act championed by former Vice President Biden. The Act’s leading provision authorized $1.8 billion over 7 years for National Institutes of Health (NIH)-administered research into cancer. The Cures Act also authorized funding of $1.5 billion for related research under former President Obama’s Precision Medicine Initiative (PMI). Also relevant to cancer therapeutics and diagnostics market development were the Act’s provisions for easing requirements and accelerating review of FDA submissions. Regulatory reform for advanced medical technologies will remain a key priority in healthcare for the current White House administration.

The full impact of research funding under the Act is uncertain. Only $300 million and $40 million were appropriated for the Moonshot and Precision Medicine Intiatives in fiscal year 2017; remaining authorized funding must be appropriated every year hereafter. Discretionary federal spending will likely come under significant budgetary restrictions in the coming years, possibly limiting the amount of future funding made available to the initiatives. Ongoing data sharing collaborations supported by NIH’s National Cancer Institute (NCI) with private sector researchers are ongoing and will be less vulnerable to funding shortfalls. The Genomic Data Commons (GDC) and Cancer Genomic Clout (CGC) Pilots were launched by the NCI in early 2016 as part of the former President’s PMI. The GDC unifies and genomic data from NCI-supported programs like The Cancer Genome Atlas (TCGA) and Therapeutically Applicable Research to Generate Effective Treatments (TARGET). The Moonshot Initiative also launched the Blood Profiling Atlas pilot or an open database of liquid biopsies useful for blood-based cancer diagnostics development.

Appropriated funds for the Moonshot Initiative will also be allocated to the FDA Oncology Center of Excellence. In June 2016, Richard Pazdur was appointed as the acting director of the FDA’s virtual Oncology Center of Excellence. The center brings together regulatory scientists and other reviewers with expertise in cancer diagnostics and cancer therapeutics. The FDA’s history of reorganization has been responsive to the development and specialization of cancer products – the Office of Hematology and Oncology Products (OHOP) was created in 2005 to consolidate the review of cancer therapeutics; OHOP then organized disease-specific teams in 2011; and most recently in 2016 integrated the review of biologics and devices alongside targeted therapies. With its Center of Excellence, the FDA plans to keep pace with innovation in cancer products and improve upon its review rate that included 61 approvals for cancer therapeutics under OHOP between January 2010 and July 2016.

Title III of the 21st Century Cures Act provides $500 million to the FDA over ten years to enact regulatory reform in pharmaceutical and medical device approval. Funding will be used to staff the FDA Oncology Center of Excellence and other departments with additional scientific experts. The provision also directs the FDA to include “real world evidence” or evidence collected outside of clinical trials in the review process. Title III also promotes the modernization of clinical trial design: the FDA will evaluate alternative approaches such as common control trials. The legislation singled out regenerative medicine products for streamlined development using enhanced information sharing between the FDA, researchers and industry developers regarding the highly innovative field.

The FDA approval process is expected to become more product-friendly with paired review of cancer therapeutics and companion diagnostics; alternative clinical trial designs; submission of real world evidence; and expedited reviews for priority products. Less strenuous and more flexible requirements for device submission to the FDA would presumably encourage more laboratory service companies to submit their lab-developed test (LDT) services for approval as in vitro diagnostic (IVD) devices. However, the impetus to introduce advanced and esoteric tests as IVD kits will likely be moderated by deregulation trends across review processes. As recent as last year, the FDA intended to finalize LDT guidance implementing FDA review of many LDTs, including most cancer tests, with requirements on par with IVD product submissions. By the end of 2016, the FDA announced its LDT oversight regulation would be put on hold. Industry opposition likely hindered oversight efforts as did the political realities of an incoming regulation-averse White House administration. In short, the U.S. test service industry is here to stay. Without FDA regulatory oversight, many labs and biotechnology companies will continue to utilize the LDT route to commercialization in order to bypass clinical trials and added millions in development expenses.