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Conference Coverage: NCCN Policy Summit 2018
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Gottlieb Calls on Payers to Share Data to Aid Drug Innovation

The FDA is working to modernize the clinical trial process and answer the right questions about today’s innovative cancer drugs, but regulators need help from payers—or, more precisely, from their data, according to FDA Commissioner Scott Gottlieb, MD.

Gottlieb called for more data sharing during remarks at the National Comprehensive Cancer Network Oncology Policy Summit held June 25 in Washington, DC. The commissioner covered 4 policy areas he said were linked by “the need to modernize drug development to harness the full medical potential of this rapidly advancing science, while ensuring that innovation remains affordable for patients.”

He added that patients will not benefit from outdated regulatory frameworks that keep lifesaving drugs off the market, stretch out the process, and make drugs more expensive. Although the FDA does not have direct authority over drug costs, Gottlieb said, a different approach can take time and costs out of the approval process, as well as promote competition and encourage generics and biosimilar development. “If FDA-approved drugs are priced out of reach of patients, then the full benefits of innovation won’t be realized,” he said.

He discussed:
  • Steps the FDA is taking to modernize the clinical trial process in light of the rise of targeted therapies, which are given to select patients based on biomarkers and often bring more robust responses
  • How to harness real-world data in regulatory approaches to answer questions about how patients respond to drugs over time
  • How the FDA is working with payers and providers to “leverage” real-world evidence to answer payers’ questions, such as whether a therapy keeps more patients out of the hospital
  • Why this approach calls for payers to provide “more open access to the significant amount of data that they have”
Over the past 20 years, as both cancer therapies and survival rates have improved, more patients have been matched to drugs in trials based on “tumor biology,” Gottlieb said. This has allowed the FDA to approve drugs based on smaller trials with fewer patients and leapfrog from phase 1 studies directly to later phases. Oncology accounts for more breakthrough therapy designations than any other therapeutic area, including 50% of all designations granted since 2013.

“We had to ask ourselves some hard questions,” he said. “What should we do, for example, guided both by ethics and science, when a new drug in a heavily pretreated group of terminal cancer patients is in a phase 1 study, where it shows a dramatic response in more than half of these patients?” And, he asked, how are patients randomized in phase 2 based on that result?

Although some criticize the FDA’s approach to approvals based on smaller groups of patients, “this criticism lacks historical and scientific context,” he said.

When cancer drugs were more toxic and less effective, the fact that they worked at all was the most relevant outcome, so overall survival was the most appropriate end point. “They were standards designed for an era that no longer exists,” he said.

The FDA has consulted extensively with stakeholders, including patient groups, and is now more interested in progression-free survival, tumor shrinkage, and the severity of adverse effects. It’s not that these measures weren’t relevant in the past, Gottlieb said: “It’s just that the drugs were so toxic that these benefits generally were not seen as sufficient to justify the risks.” Today’s drugs are far less toxic, and looking at how patients fare over longer periods is highly relevant. The FDA’s changing approach “didn’t evolve overnight,” Gottlieb said. “We didn’t do it on a whim.”

The approach is working: Of 51 oncology accelerated approvals with postmarketing requirements and verified benefits, the average time from approval to verification is 3.4 years. Just 5 drugs have been pulled from the market for failing to confirm benefits.

In response to critics who say surrogate end points bring uncertainty about the eventual efficacy, Gottlieb said, “That’s, in fact, the whole point.”

In some cases, trials would take far too long; without crossover trials, some patients would miss the opportunity for lifesaving treatment. “We would literally be asking patients to die so that we could achieve a lower P value,” he said.

To promote cost-saving solutions such as value-based contracting, Gottlieb called on payers to aid the process by sharing data that will guide FDA on how drugs work in different populations and over the long haul: “It’s not enough to point fingers. We all need to work together.”

Payers who want to help the FDA ultimately drive down prices or show how therapies affect hospitalization rates must “put your data where your arguments are,” instead of charging huge sums for this information, Gottlieb said.

Real-world data do not replace the gold standard of randomized clinical trials but can fill in knowledge gaps. As a result, Gottlieb said, for the 2019 budget, the FDA seeks $23 million to invest in advanced analytics and data analysis, which he said will give the agency the ability to do “near real-time” analysis of evidence from electronic health records for 10 million individuals.

During a question-and-answer period, Gottlieb said the FDA is working to help overcome impediments to using real-world data for value-based contracting by issuing a recent guidance on this topic. When asked if he’s confident that the FDA will get the real-world evidence it needs to shift its approach, Gottlieb said that’s the role of enforcement.

At the time of Gottlieb’s speech, the FDA had just sent a letter to a manufacturer that has not fulfilled its postmarketing requirements. “The agency feels far more confident it will get the data,” he said. “The system is dependent on that.”

 

Paying for Innovation in Cancer Care Means No Easy Answers

As Ron Kline, MD, put it: If figuring out how to pay for innovation in cancer care was easy, someone would have already done it.

“We know it’s hard. We know it’s going to take a while,” said Kline, medical officer in the Patient Care Models Group for the Center for Medicare and Medicaid Innovation (CMMI) at CMS.

Kline was on hand to discuss the Oncology Care Model (OCM), the 5-year effort to rethink cancer care delivery and cut costs in Medicare, during a discussion by the National Comprehensive Cancer Network (NCCN) on how to balance the revolution in cancer therapy with the need to pay for it.

During the session “Paying for Innovation,” part of NCCN’s June 25, 2018, Policy Summit in Washington, DC, Kline explained that the OCM shouldn’t be easy from the get-go—if it was, then CMMI would know it hadn’t truly pushed the envelope. But CMMI wants practices to succeed, and early results show that 25% of practices cut costs 7%. “Sixty percent of practices decreased costs, compared with the baseline,” he said.

Guided by questions from Moderator Cliff Goodman, PhD, of The Lewin Group, panelists discussed today’s challenges in cancer care: the need to simultaneously overhaul delivery systems and learn to use—and bill for—some of most complex therapies and technologies ever conceived. Heading that list: chimeric antigen receptor (CAR) T-cell treatments, gene therapies that can take 2 to 4 weeks to manufacture for a specific patient, at a cost of up to $475,000 just for the drugs.

But paying for innovation is more complicated than figuring out how to pay for CAR T-cell treatments or targeted therapies. It’s also about helping patients who will live with cancer as a chronic condition, such as those with certain blood disorders.

“The advance of novel, innovative therapies has transformed treatment,” said Meghan Gutierrez, chief executive officer of the Lymphoma Research Foundation. The combination of new treatments, diagnostic tests, and sequencing has produced more information about the disease. Patients have more opportunities than ever for better care, but for many, treatment will not be a one-time event. “Patients recognize they are likely to be in and out of care for the rest of their lives,” she said.

Innovation isn’t just about approving new therapies, said Pavan Reddy, MD, FACP, of the Cancer Center of Kansas. Precision medicine means identifying treatments that are “tumor agnostic,” based on biomarkers, as well as research that tells clinicians when they can de-escalate or stop treatment, he said.

Reimbursement drives decisions

Patients are citing treatment costs as a factor in their medical decisions, according to Gutierrez. “Increasingly, patients are speaking to the economic burden across their lifetime,” she said. Therapies can extend life well beyond what was once imaginable, but with these advances come rising out-of-pocket costs.

Stephanie Farnia, MPH, director of health policy and strategic relations for the American Society for Blood and Marrow Transplantation, is a veteran of dealing with reimbursement challenges. Things such as delays in getting treatment codes, which allow physicians to bill properly, can be a barrier to lifesaving advances.

Panelist Stefanie Joho, a cancer survivor, lives with fatigue from the immunotherapy that saved her life, but, as Goodman noted, “some long-term impacts are unknown.” In Joho’s case, that’s better than what she was facing when her sister, refusing to accept the grim prognosis that nothing could be done for Joho’s colon cancer, scoured the internet until she found an appropriate clinical trial. Other panelists conceded that some oncologists, especially in rural areas, don’t always refer patients for clinical trials. At this point, CAR T-cell therapies are unlikely to be delivered outside of a major academic medical center with expertise in transplants, said Caron A. Jacobson, MD, of the Dana Farber Cancer Institute/Brigham and Women’s Hospital in Boston, Massachusetts.

Kline, however, called this a copout. As a pediatric oncologist, he said, he finds that clinical trials for young patients “are the standard of care.”

Joho and others said they’d like to see more patients enroll in clinical trials, although they recognize barriers such as travel. Michael Ybarra, MD, representing the Pharmaceutical Research and Manufacturers of America, said that drugmakers are concerned about the ability of rural patients to gain access to trials, but factors such as hospital consolidations and benefit design also create access barriers. A major driver of consolidation in the health care industry has been the 340B program, which groups such as the Community Oncology Alliance say has been exploited beyond its original mission, to the detriment of community providers.

The arrival of CAR T-cell therapy

 
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