FEATURE
ed Outcomes (PRO) should be considered by CMS as it develops its
National Coverage Determinations (NCDs) that govern Medicare
coverage of a medical service, procedure or pharmaceutical. CMS
was all set to provide coverage for CAR-T using its normal scientific
evidence only. CMS had even developed a payment that was well
under the list price for Kymriah (tisagenlecleucel) or of Yescar-
ta (Axicabtagene Ciloleucel) which are just under a half-million
dollars. United Health Care’s chief medical officer had written an
appeal to CMS asking that there be a new review of CAR-T before
it became widely used, not just for B-cell non-Hodgkin Lymphoma
and acute lymphoblastic leukemia refractory to standard therapies.
Because of the additional costs for apheresis, B-cell ablative therapy,
and treatment of complications, the costs for CAR-T therapy may
approach a million dollars. United Health Care argued that for
both Medicare and for Medicare Advantage plans, access to CAR-T
should be more closely reviewed. those same chemotherapeutic agents. His study showed that, while
manufacturers and researchers report a given level of symptomatic
toxicity upon their understanding of what patients say, it turns out
to be only half of what patients actually report. What may seem as
a less important side effect to the medical staff may be a much more
significant problem for the patient. (3.)
For this reason, CMS determined that it would stop the devel-
opment of the NCD and hold the panel that I found myself on.
Our focus was not on CAR-T itself but whether the role of Patient
Reported Outcomes should be included as parts of the package of
evidence that CMS considers. This is a big deal for Medicare. There
are a number of PRO instruments out there. CMS felt that only a
few of these should be in general use. Too many instruments being
used could cause more statistical confusion in trying to understand
the impacts across multiple submissions. Additionally, the style
of presentation of data to CMS, insurers, physicians and patients
needed some clarification. Pie charts, bar charts, or line charts with
or without confidence intervals: all became subject in our panel’s
recommendations to CMS. a series of votes on questions that collectively formed the opinion.
Our consensus was that not only should Patient Reported Outcomes
be included in the National Coverage Determination on CAR-T,
but that such information should continue to be included in NCS
related to subsequent novel therapies. In closing comments, the
majority of the panelists raised the point that the effectiveness of
treatment is not just what that therapy does to the human from a
biologic point of view. Both the patients’ values and their experience
of this care are essential in our evaluation of treatments offered
to patients. This is a major new way to include of the voice of the
patient in shared decision making.
Novartis and Kite/Gilead, the two manufacturers, presented
that morning. Their stance was that asking patients to report their
outcomes and their side effects would add costs and take time. All
of that would add to the expense of an already expensive therapy.
They were followed by physicians from Mayo Clinic and Fred
Hutchinson Cancer Center arguing against inclusion of patient
reported outcomes as they could not be sure to follow patients
long enough to have good data. However, they were followed by
physicians from Sloan Kettering Memorial Hospital and from the
University of North Carolina both indicating that they use PRO in
many of their programs and have found ways through computer
surveys to capture PRO data.
An important presentation came from Ethan Basch, MD, Pro-
fessor of Health Policy and Management, Director of the Cancer
Outcomes Research Program, and Professor of Hematology and
Oncology at the University of North Carolina. His work has in-
cluded a comparison of the adverse events profiles in FDA package
labeling compared to Patient Reported Outcomes of patients on
Before lunch was the opportunity for “public comment” which
was really the opportunity for more protagonists from both sides
to make comments, though all were limited to just a few minutes.
After lunch, we had the opportunity to ask questions of all of the
presenters. We were not to ask about the sciences behind CAR-T,
the effectiveness of the therapy or the costs of care, but only the
impact of having the Patient Reported Outcomes included in the
requested information to inform CMS in making a National Cov-
erage Determination.
By 4:30 p.m., we had made our pronouncements to CMS through
Citations:
1.) Medicare Evidence Development & Coverage Advisory Committee
https://www.cms.gov/Regulations-and-Guidance/Guidance/FACA/
MEDCAC.html Accessed Sept. 3, 2018
2.) National Coverage Analysis (NCA) Tracking Sheet for Chimeric Antigen
Receptor (CAR) T-cell Therapy for Cancers (CAG-00451N) https://
www.cms.gov/medicare-coverage-database/details/nca-tracking-sheet.
aspx?NCAId=291 Accessed Sept 3, 2018
3.) Basch E, Deal AM, Dueck AC, et al. Overall Survival Results of a Trial
Assessing Patient-Reported Outcomes for Symptom Monitoring During
Routine Cancer Treatment. JAMA. 2017;318(2):197–198.
Dr. James is the Senior Medical Director for Highmark Inc. in Pittsburgh,
PA and is an Affiliate GLMS member.
NOVEMBER 2018
9