360-Degree Perspective from the ACCC Institute for Clinical Immuno-Oncology Policy Summit

On August 30, the U.S. Food and Drug Administration (FDA) approved the first CAR Chimeric Antigen Receptor (CAR) T-cell therapy, tisagenlecleucel (Kymriah). With this approval of the first cancer gene therapy in the U.S., immuno-oncology took a historic step forward.

Later that same day, in a press release titled, “Innovative Treatments Call for Innovative Payment Models and Arrangement,” the Centers for Medicare & Medicaid Services (CMS) congratulated the scientists and researchers involved in the development of the new treatment, while emphasizing the agency’s  on-going commitment to working with stakeholders on “innovative payment arrangements.”

In hindsight, opening remarks at the ACCC Institute for Clinical Immuno-Oncology (ICLIO) Policy Summit held less than two weeks earlier, seem prophetic:  “Immunotherapy is a hallmark of what’s going on in oncology. . . wonderful, but expensive new therapies, how are we going to make this work? ” said Lee Schwartzberg, MD, FACP, ICLIO Advisory Committee Chair.  Dr. Schwartzberg is Chief, Division of Hematology/Oncology, The University of Tennessee;  Medical Director, The West Clinic.

“Immuno-oncology presents remarkable opportunities and challenges at the same time,” said ACCC President Mark Soberman, MD, MBA, FACS, welcoming participants to the Summit. “We have to figure out how to leverage immuno-oncology for our patients in a very sustainable way in our cancer eco-system.” Dr. Soberman is Medical Director, Oncology Service Line; Chief Physician Executive, Monocacy Health Partners, Frederick Regional Health System.

The by-invitation only, August 18, 2017, ICLIO Policy Summit brought together oncology stakeholders including representatives from patient advocacy groups, pharmacy, research, government, industry, oncology clinician leadership, oncology nursing leadership, and a payer representative to share perspectives on current real-world challenges in immuno-oncology through the lens of:

• Clinical and Policy Issues
• Alternative Payment Models
• Application and Impact of Quality Measures
• Payer Management of I-O (Immuno-Oncology)
• Future Challenges and Opportunities

Watch video for comments from ICLIO Policy Summit participants:

360-Degree Perspective

The ICLIO Policy Summit discussion by these diverse stakeholders revealed a 360-degree perspective on the current landscape for the translation of immunotherapy from bench to bedside. Top-level themes from the Summit are highlighted below:

Biomarkers. All stakeholders concur that there is a pressing need to identify biomarkers for immuno-oncology agents in order to address the issue of identifying those patients mostly likely to benefit from being treated with an I-O agent and to help mitigate cost.

Education. New agents are emerging with new mechanisms of action, and combinations and sequencing of immuno-therapy agents are on the horizon. Understanding of side effects, late effects, and long-term effects, and the nuances of immunotherapy delivery for patients in the community continues to evolve. On-going education is imperative, not just for the multidisciplinary oncology team but also for other providers who care for these patients (e.g., primary care, endocrinologists, pulmonologists, radiologists, emergency department staff) and for the patients who will receive these therapies and their caregivers.

Community Perspective. The arrival of new immuno-oncology agents has fundamentally changed the landscape of clinical practice over the past three years. In the community setting, programs need to “take a systematic approach to I-O implementation,” commented community-based provider. P&T Committees must have the capacity to address issues around appropriate use, inventory management, and cost of expensive new and emerging I-O agents to avoid financial toxicity for patients, providers, and institutions.

More Evidence Needed.  I-O is far from plateauing, participants agreed, but more evidence is needed around combination therapy and sequencing of these agents. “We don’t know which combinations are superior and which are superior to single agents,” commented a clinician participant. But that evidence “is coming very quickly,” he added.  “I think combinations are going to be important,” commented a research clinician, “rational combinations,” adding that the “PD-1 pathway is foundational.”

Access to I-O therapies. Prior authorizations continue to be a barrier to access, stakeholders agreed. Pharmacy and PBM participants, in general, indicated that they follow the lead of the NCCN Drugs & Biologics Compendium, but the high-cost of these agents leads to critical pharmacy issues of how to afford these expensive therapies and how the cancer program’s physicians will use them.

Discussion of pathways, pre-authorizations, and “totality of the evidence” for FDA approval (the summit discussion touched on expedited clinical review for I-O based on review of the ‘totality of evidence,’ as is currently the case for FDA review of biosimilar agents)—brought the conversation back to biomarkers. “We need to focus on biomarkers. . . selecting the right patients for the right agents,” emphasized a researcher participant. And he added, “We need multiple modalities because cancer is very clever.”

Clinical Trial Enrollment.  Referencing a recent New York Times article, participants cited the challenge of accruing patients to the many open immuno-oncology trials. At the same time, greater access to I-O clinical trials in the community setting may lessen access barriers to these agents for some patients, commented a researcher participant.

Risky Business: Alternative Payment Models. Discussion of alternative payment models (APMs) focused primarily on the CMS Oncology Care Model (OCM).  Summit attendees participating in the OCM agreed that during the first year of the model, efforts centered largely on “getting all the mechanisms in place”—readying practice infrastructure for OCM requirements. With that accomplished, priorities for OCM practices include reducing inpatient admissions and ER visits, and avoiding adverse events.  However, participants agreed that the need to address issues around high-cost anticancer agents is nearing.  In a risk-sharing payment model, it will be critical to find methods to sustain small and large practices, commented a physician leader.

Stakeholders agreed that a challenge with OCM design is that the episode being measured is too brief; it does not follow the patient’s entire cancer journey. Outcomes such as cure or disease-free survival, for example, are not included in the OCM.  “The model looks at cost, not value,” noted a participant.

Still, the OCM provides a path toward demonstrating attributes of patient-centered care that are components of the new value-based payment models, participants said.

Quality Measures & I-O. Coming to consensus on quality measures in oncology remains a challenge. Patient advocate stakeholders pointed to the study by Basch and colleagues presented at ASCO 2017 showing that just by tracking patient-reported outcomes (PROs), patients lived longer. From the patient advocate perspective quality measure concerns are multifold, including:

• Tension between the driving trend in oncology toward standardized measure sets (pathways, etc.) and precision medicine, i.e., the need to support appropriate variation in order to individualize patient care.
• Current patient satisfaction measurement tools that do not assess what really matters to patients (e.g., quality of life and outcomes).
• Quality measures that assess process (much of which is already being done), rather than outcomes measures that would be tangible to patients (e.g., staying out of the hospital).

In response, patient advocacy groups are developing their own quality measures based on what patients’ say is important to them, including not just clinical measures but quality of life measures such as disruption to work, childcare, and transportation to treatment.

Payer Management of I-O. As the current healthcare reimbursement landscape continues to evolve, key concerns identified during the ICLIO Summit were:

• The need for biomarkers for patient selection to ensure those most likely to benefit from the I-O therapy will receive it and those who won’t, don’t.
• The need for the healthcare system to be more nimble and adaptable in “looking at good data.”
• One of the biggest challenges for clinicians is variation in coverage under different health plans. As an example, a provider sees five different patients with the same cancer type, each with a different health plan, each with its own coverage options and requirements. The end result: guidelines to reduce variation are not working, commented a health system executive.
• Prior authorizations creating barriers to access and uncertainty for patients, providers, and practices. “We’re taking on risk with . . .value-based payment, but we’re still saddled with prior authorization. Maybe it should be one or the other,” said a clinician leader.
• Managed care organizations and others are looking at how to bundle oncology products into trend management pools.

Looking to the Future

The final discussion block looked to the future. In a lightening round, Summit participants were asked to share their perspectives one key challenge or opportunity for immuno-oncology in the near future—summed up in a sentence or two.  Their responses offer a final 360-degree look ahead at real-world issues facing immuno-oncology:

• We need to develop a quality measure that is “patient returns to functional status.”

• In the value discussion, there is realistic, and then there is reality. A lot of what becomes value is tied to your resources. [Many times] in medical situations, that’s not taken into consideration.

• Future treatment decisions informed by biomarkers and life circumstances.

• View all navigators as integral parts of the cancer care system.

• Adapt [the] delivery system to be more nimble to adopt major advances.

• We need to look at real-world evidence for comparative effectiveness. We have to go beyond the regulatory system to really understand the value in the community that each agent brings.

• Will we have enough doctors, nurses, social workers, and navigators to treat patients with these complex therapies?

• We need to develop and implement patient-reported outcomes, and we need to understand the real cost of care.

• Between academic programs, the federal government, [industry], and the community, we need to get more serious around biomarker development and who is most likely to benefit from expensive therapies and those not likely to benefit.

• Clinical trials. . . how can we bring clinical trials to community hospitals? We need a process to open clinical trials [so that the community can] benefit from access to these drugs early on.

• Evidence generation. . . stakeholders need to generate evidence.

• Preparing the nursing workforce [to move] from a disease-state specific [care model] to a more biomarker-driven model.

• Form follows function; if the future of medicine is biomarker driven, then resources have to be organized along that line.

• Greater investment in analytics so that we can get more nimble feedback; greater degree of analytic support.

• Establish and maintain a national registry to capture and analyze data from real-world care.

In closing the ICLIO Policy Summit, Advisory Committee Chair Lee Schwartzberg, MD, FACP, thanked participants: “We come from different points of view, but we have common ground. . . . Communication is the way we’re going to go forward with new therapies in [our] complex [healthcare] system.

The ACCC Institute for Clinical Immuno-Oncology is the only comprehensive initiative to prepare multidisciplinary cancer care providers for the complex implementation of immuno-oncology in the community setting.  View ICLIO’s robust resources, webinars, education offerings, and more, on the ICLIO website accc-iclio.org.