Cancer Moonshot Effort Unveils Dozens of Initiatives to Speed Research

Alicia Ault

June 30, 2016

WASHINGTON, DC — US federal officials have unveiled a dozen new initiatives designed to accelerate cancer research, speed new therapies to patients, foster data sharing, and simplify participation in clinical trials, all part of the formal liftoff of the Cancer Moonshot program.

The Moonshot program, headed by Vice President Joe Biden, aims to "end cancer once and for all."

The new initiatives were announced in conjunction with Moonshot summits being held on June 29 in Washington, DC, and at 260 locations in all 50 states, Puerto Rico, and Guam.

Among the programs being established, though not entirely funded yet, are a "formulary" that will make drugs available to researchers through a National Cancer Institute (NCI) clearinghouse; a widening of the NCI's Genomic Data Commons, which will allow access to data from 32,000 patients; creation of an Oncology Center of Excellence at the Food and Drug Administration; a new partnership between the National Institutes of Health, philanthropies, and at least a dozen biotech and pharmaceutical companies to share data and to expedite trials of combination drug therapies; a big data collaboration between the Department of Veterans Affairs (VA) and the Department of Energy; a Department of Defense, VA. and NCI consortium in proteogenomics; and the start of a new Oncology Care Model that will use Medicare payment to steer physician practices to provide high-value care.

I firmly believe we can do in the next 5 years what would ordinarily take 10 Dr Vice President Joe Biden

Private industry and foundations stepped in with promises as well. The American Cancer Society (ACS) said it would double its research budget by 2021 to $240 million annually. The Breast Cancer Research Foundation will be doubling its research spending to a cumulative $1 billion by 2021. The Prostate Cancer Foundation will donate at least $50 million within the next 5 years and will partner with the VA to build a precision medicine program for veterans with prostate cancer. The American Society of Hematology will develop a Multiple Myeloma Data Sharing Platform, and "Coding for Cancer" efforts — coordinated by Harvard Medical and Business Schools and Sage Bionetworks — will issue multiple prizes for solving various cancer informatics puzzles.

"I firmly believe we can do in the next 5 years what would ordinarily take 10," Vice President Biden said at the Washington, DC, summit.

To patients, "Time matters. Days matter. Minutes matter," he said.

The meeting was attended by several hundred physicians, scientists, patients, survivors, advocates, academic institutions, federal officials and staff, advocates, philanthropies, social workers, and nurses, among others. The Administration is "trying to build a momentum that we think is long overdue," Nancy Davidson, MD, president of the American Association for Cancer Research, told Medscape Medical News. The goal of doubling progress "ought to speak to every oncologist," said Dr Davidson, who is director the University of Pittsburgh Cancer Institute in Pennsylvania.

And it's not just all talk, said NCI Acting Director Douglas R. Lowy, MD. "The vice president is really having an impact," he told Medscape Medical News.

The NIH received a $195 million investment in fiscal 2016 for Moonshot, and the Obama administration has asked Congress for another $755 million for the NIH and the Food and Drug Administration in 2017. The ACS is seeking $680 million for the NIH and NCI. But Congress has not acted on any of the requests.

"Congress can't let this moment pass," said Chris Hansen, president of the ACS Cancer Action Network. "We're on the cusp of so many new developments in diagnostic tests and treatments thanks to past investment that has allowed us to understand cancer at the molecular level," he said in a statement. "Congress must continue the funding momentum if we are going to conquer cancer."

NIH and NCI Bring Industry Under Tent

The money that the NIH has received so far for Moonshot should help get some of the proposed programs off the ground, NIH Director Francis Collins, MD, told reporters.

Dr Collins is spearheading the new Partnership for Accelerating Cancer Therapies (PACT), an idea that he says started with conversations he had with pharmaceutical and biotech executives at the World Economic Forum meeting in Davos, Switzerland, in January 2016. A dozen companies (which aren't being named yet) have signaled interest in sharing data with each other and with the academic and government researchers that would participate.

"NIH is willing to cover half the costs," said Dr Collins, but he added that the agency is counting on private industry to foot the rest of the bill. "This is not a hand-off or a giveaway to the private sector," Dr Collins said in response to a question from Medscape Medical News. But he said the NIH would likely continue to look to industry when there was a shared need. "Money is tight — we're all in a hurry to make progress."

PACT will focus on four areas: how to develop and test biomarkers; better disease modeling; how to address rare cancers; and how to encourage trials of combination therapies, even if they involve drugs from different manufacturers. PACT could possibly "provide a safe space for those kinds of collaborative trials," Dr Collins said.

Another partnership will bring the NCI together with 20 to 30 drug and biotech companies to create a preapproved "formulary" of compounds — investigational and approved — that NCI-funded researchers and NCI-designated cancer centers could use for new indications or in new combinations. A blanket agreement will clear use of the compound, which would cut negotiations with drug makers from a year to maybe just a few months, Dr Lowy told Medscape Medical News.

The manufacturers will also agree in principle "that drugs from more than one company will be combined with each other for testing," he said. The first compound will be available this year, said Dr Lowy.

Genomic Profile of Cancers Released

In another collaboration announced on June 29, Foundation Medicine will release 18,000 genomic profiles of adult cancers and make them available through the NCI's Genomic Data Commons portal. That will double the size of the NCI database, which is used by researchers. Dr Lowy said the Foundation profiles contain more patient-level data than the NCI's Cancer Genome Atlas, including details of how patients have "'been treated, the response, and the outcomes."

Spurring Interest in Trials

Moonshot is proposing various ways to boost knowledge of and participation in clinical trials as a way to keep research moving. At present, a very small proportion of the total cancer patient population is enrolled in clinical trials.

"In adult solid tumors, we put 3% to 5% of adults on study," Clifford A. Hudis, MD, chief executive officer of the American Society of Clinical Oncology, told Medscape Medical News. "That can only hold us back."

Dr Hudis suggested that new trial models — in settings outside rigorously controlled randomized trials, and that use more real-world data — are needed, along with new incentives for clinicians and patients to participate.

He applauded the NCI's plan to redesign its portal. This portal is currently more focused on clinicians than patients, Warren Kibbe, PhD, NCI's acting deputy director, told Medscape Medical News. "Clinical trials are a statement of what do we currently know about that disease and what do we think are the best possible treatments," he said. That knowledge base should be made available to patients and to more oncologists, said Dr Kibbe.

The NCI is allowing advocacy groups to repackage the trial data and feature them on their own websites. Patients will be searching, but through the advocacy group's site. Advocates can prioritize the search result to highlight trials they think will be of greater interest to their particular community, said Dr Kibbe.

And, in conjunction with Moonshot, the Cancer Support Community launched "Frankly Speaking About Cancer Clinical Trials," an education program that aims to dispel the myths and raise awareness about trials.

FDA to Combine Cancer Expertise

As oncology has become more complex, the FDA has been contemplating how to address therapies that are coupled with diagnostics or that combine traditional drugs with biologics or devices, Richard Pazdur, MD, told reporters at the Moonshot summit. Dr Pazdur, the former director of the agency's Office of Hematology and Oncology Products, has just been named the acting director of the new FDA Oncology Center of Excellence.

The Center is being established to bring together the more than 70 medical oncologists at the FDA with staff from the agency's diagnostics, drugs, and biotech divisions to collaborate on product reviews. "The whole purpose is really to look forward," said Dr Pazdur. Part of that effort will be to work more closely with companies when they are first starting their development process, he said. "We're interested in more of the upfront work — how the drug is going to be developed," said Dr Pazdur.

The Center "will expedite the development of novel combination products and support an integrated approach to tackle this devastating disease that touches so many American families," said FDA Commissioner Robert Califf, MD, in a statement.

Dr Pazdur said the agency has a track record of reviewing products quickly. "It doesn't mean you can't be quicker," he said, noting that to patients, speed was of the essence. "There is a sense of urgency and we can't ignore that," he said, but added that "urgency does not mean a suboptimal job, a sloppy job."

ASCO President Daniel F. Hayes, MD, praised the establishment of the Center and Dr Pazdur's selection. "We are confident that this initiative will build on past success excellence and create a larger community of scientists and clinicians within the agency," he said in a statement.

"The oncology center is coming at a crucial time in response to the need to coordinate review of drugs, diagnostic tests, and other types of devices," said Dr Hayes, adding, "There is no better person to lead this effort than Dr. Pazdur."

The FDA also announced that it will accelerate efforts to help physicians and patients pursue requests for investigational new drugs and that it will establish a "Navigator" program to help facilitate those requests.

Meanwhile, the US Patent and Trademark Office said it was starting a free pilot program that will fast-track certain cancer therapy patent applications. Reviews will be completed in less than a year — half the normal review time.

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