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MEDCAC Meeting 9/15/1999 - Autologous Stem Cell Transplantation (AuSCT) for Multiple Myeloma (Drugs, Biologics, and Therapeutics Panel)

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AuSCT is a process used to treat various malignancies in which stem cells are harvested from a patient's bone marrow or peripheral blood, stored, and then given back to the patient following severely myelotoxic doses of chemotherapy and/or radiotherapy. Section 35-30.1 of the Coverage Issues Manual (CIM) states that AuSCT is a non-covered condition in the treatment for multiple myeloma (MM). This decision was based on the insufficiency of data to establish efficacy. The Health Care Financing Administration (HCFA) must evaluate whether new scientific data on AuSCT supports reconsideration for national coverage in the MM population.

Federal Register Notice

Agenda for September 15-16, 1999 Meeting

Baltimore Convention Center
Room 327-9 One West Pratt Street
Baltimore, MD 21201

Panel Chairperson: Thomas Holohan, M.D.
Executive Secretary: Lauren K. Geyer, MHS

Wednesday, September 15, 1999

1:00 - 1:30 p.m.

Opening Remarks - Introduction

Agenda Item: The Committee will discuss and make recommendations regarding high dose chemotherapy and stem cell transplantation for the treatment of multiple myeloma.

1:30 - 1:45 p.m.

Overview of Stem Cell Transplantation
Michael Bishop, MD
Head, Bone Marrow Transplantation Department
NIH

1:45 - 3:15 p.m.

Open Public Session

Public Attendees, Who Have Contacted the Executive Secretary Prior to the Meeting, Will Address the Panel and Present Information Relevant to the Agenda. Speakers Are Asked to State Whether or Not They Have Any Financial Involvement with Manufacturers of Any Items and Services being discussed, or with Their Competitors.

3:15 - 3:30 p.m.

BREAK

3:30 - 4:00 p.m.

Scheduled Commentaries

4:00 p.m.

ADJOURN

Thursday, September 16, 1999

8:00 - 8:30 a.m.

Opening Remarks - Introduction

Agenda Item: The Committee will discuss and make coverage recommendations regarding high dose chemotherapy and stem cell transplantation for the treatment of multiple myeloma.

8:30 - 10:00 a.m.

Scheduled Commentaries

10:00 - 10:15 a.m.

BREAK

10:15 - 11:30 a.m.

HCFA Presentation

11:30 - 12:30 p.m.

LUNCH

12:30 - 2:00 p.m.

Open Committee Discussion

This Portion of the Meeting Is Open to Public Observers. Public Observers May not participate except at the Specific Request of the Chairperson.

2:00 - 2:15 p.m.

BREAK

2:15 - 3:00 p.m.

Open Committee Discussion - Continued

This Portion of the Meeting Is Open to Public Observers. Public Observers May Not Participate Except at the Specific Request of the Chairperson.

3:00 - 3:30 p.m.

Open Public Session

Public Attendees Who Have Contacted the Executive Secretary Prior to the Meeting, Will Address the Panel and Present Information Relevant to the Agenda. Speakers Are Asked to State Whether or Not They Have Any Financial Involvement with Manufacturers of Any Items and Services being discussed, or with Their Competitors.

3:30 - 4:00 p.m.

Committee Recommendations

4:00 p.m.

ADJOURN

Minutes from September 15-16, 1999 Meeting

OPEN SESSION

The Baltimore Convention Center
One West Pratt Street, Meeting Room 327-329
Baltimore, MD 21201

Attendees

Thomas Holohan, M.D. (FACP)
Chairperson

Lauren Geyer, M.H.S.
Executive Secretary

Voting Members
Leslie Francis, J.D., Ph.D.
Kathy Helzlsouer, M.H.S.
Robert Johnson, M.S.
Ronald Jordan, R.Ph.

Temporary Voting Members
Jeffrey Lerner, Ph.D.
Paul Mintz, M.D.

Guest Carrier Medical Director
James Adamson, M.D.

Industry Representative
Cathleen Dooley, M.P.A.

Consumer Representative
Linda Bergthold, Ph.D.

HCFA Representatives
Grant Bagley, M.D.
Vilis Kilpe, Ph.D.
Andrea Argabrite, M.S., M.P.H.

NIH Guest Speaker
Michael Bishop, M.D.

Wednesday, September 15, 1999, 1:00 p.m.

The Drugs, Biologics, and Therapeutics Advisory Panel met on September 15-16, 1999, to discuss combination high dose chemotherapy and stem cell transplantation in the treatment of multiple myeloma. This was the first meeting for this medical specialty panel of the Medicare Coverage Advisory Committee (MCAC). The meeting began with the introduction of the panelists, a reading of the conflict of interest statement, and the call to order.

HCFA Presentation. HCFA provided an introduction and overview of the Social Security Act and the new Medicare coverage process. The statute states that Medicare may pay for those items and services that are reasonable and necessary for the diagnosis and treatment of an illness or injury.

HCFA representatives then discussed the agenda topic and gave a brief history of the issues surrounding autologous bone marrow transplantation (e.g., stem cell transplantation, etc.) in combination with high dose chemotherapy in the treatment of multiple myeloma. They concluded their presentation by asking the panelists to consider the following questions during their deliberations:

  1. Is there sufficient evidence to support autologous stem cell transplantation for the treatment of multiple myeloma in the Medicare population (individuals who are either: 65 or older, disabled, or who have end-stage renal disease?
  2. What factors should be considered when developing a Medicare coverage policy for autologous stem cell transplantation for multiple myeloma (e.g., age, prior treatment, co-morbidity, response to treatment, survival, etc.)?
  3. What is the most appropriate measure of successful outcome with autologous stem cell transplantation?
  4. What evidence supports the efficacy of more than one autologous stem cell transplantation per patient?
  5. What qualifications should apply for providers and centers performing the autologous stem cell transplantation procedure?
  6. Should there be specific protocols for performing the procedure?
  7. Based upon current evidence, how does the source of stem cells (bone marrow vs. peripheral blood) effect successful outcome?
  8. Are there any other questions or concerns that the panel would like to address?

Chairperson’s Opening Remarks. The Chairperson acknowledged that this was the first meeting of the Medicare Coverage Advisory Committee and remarked on its significance. He informed the panel of its charge to weigh available evidence, to provide independent expert scientific advice, and to help HCFA make sound decisions based upon the reasoned application of good science. He advised the panel to consider objective medical evidence as the paramount factor in its deliberation, to carefully evaluate the quality of the evidence, and to judge whether or not the evidence supports the use of the autologous stem cell transplantation for the treatment of multiple myeloma.

NIH Presentation. A representative from the National Institutes of Health, Bone Marrow Transplantation Department presented an overview of multiple myeloma. Panelists heard statistics on the numbers of cases diagnosed annually, incidence, patient demographics, and the morbidity and mortality associated with multiple myeloma. A discussion of various treatment options followed and the fact that most oncologists approach treatment in three phases: induction, consolidation, and maintenance. In regards to stem cell transplantation, information as to how cells are obtained, the differences between allogeneic vs. autologous transplants, and the complications that may present to patients was provided. Finally, the NIH spokesperson concluded with an overview of the results of phase 2 and phase 3 clinical investigations.

Open Public Session. Panelists heard from 11 speakers, all of whom presented testimony regarding their experiences with the disease, as well as their support for coverage of this treatment. Most of the speakers either had multiple myeloma themselves, or had family members with the disease. The speakers related real-life situations, their opinions and their hope that stem cell transplantation would be reimbursed by Medicare. A few of the speakers questioned the panel as to why Medicare does not cover stem cell transplantation for multiple myeloma, despite the fact that the Veteran’s Administration (VA) will provide the service. The Chairperson clarified the confusion by stating that the VA provides 4 services: clinical care, education & research for medical professionals, research, and a back-up system for the Department of Defense. The VA conducts stem cell transplantation for multiple myeloma as part of its research function. Other speakers questioned why Medicaid reimburses stem cell transplantation, but Medicare does not.

Scheduled Commentaries. On the first day, panelists heard from two speakers who provided testimony in support of coverage for the combination of high dose chemotherapy and stem cell transplantation in the treatment of multiple myeloma. The first speaker was Mr. Greg Dean, a financial coordinator at Northwestern Memorial Hospital. Mr. Dean spoke about the inconsistencies between Medicare and Medicaid in regards to stem cell transplantation and requested that Medicare provide coverage for stem cell transplantation in multiple myeloma.

Dr. Kyle, speaking on behalf of the American Society of Hematology, provided the second commentary. Dr. Kyle related statistics on the incidence of the disease and provided background information on its etiology. Dr. Kyle then quoted the findings of two studies and his own experiences. He believed that patients who are 70 years of age or younger should be covered for this service. However, he stated that the American Society of Hematology has not adopted a formal position on autologous stem cell transplantation. Panelists asked a number of questions of Dr. Kyle concerning the design of the clinical investigations that he quoted, whether they were randomized, and the types of treatment regimens used. Panelists also questioned Dr. Kyle’s opinion about patients who were resistant to treatment. Dr. Kyle responded by providing additional detail regarding the quoted study’s design, and by relating his treatment experience with resistant and refractory multiple myeloma patients. Dr. Kyle indicated that patients who failed to respond to chemotherapy might have a favorable outcome if stem cell transplantation was attempted. However, those patients who are refractory and have had long-term treatment with alkylating agents are not good candidates for stem cell transplantation.

Recess. Following Dr. Kyle’s testimony, the meeting was recessed at 4:00 p.m. to reconvene the following day.

Thursday, September 16, 1999, 8:00 a.m.

The Drugs, Biologics, and Therapeutics Advisory Panel meeting reconvened for the second day on September 16, 1999, to discuss combination high dose chemotherapy and stem cell transplantation in the treatment of multiple myeloma. The meeting began with the introduction of the panelists, a reading of the conflict of interest statement, and the call to order.

Chairperson’s Opening Remarks. The Chairperson spoke about to two misconceptions that were made by some of the speakers during the previous day’s open public session. The first being that Medicaid will reimburse for stem cell transplants but Medicare does not. The Chairperson clarified that Medicaid programs are run by the states, and therefore, different states may have different policies. The second issue concerned the disorders that are covered under Medicare for stem cell transplantation. He then read from the Coverage Issues Manual those conditions where autologous stem cell transplantation is considered to be reasonable and necessary. The covered conditions are: 1) for those individuals with acute leukemia in remission (lymphoid, myeloid, monocytic, acute erythremia, and erythroleukemia) who have a high probability of relapse and who have no human leukocyte antigen (HLA)-matched, 2) resistant non-Hodgkin’s lymphoma and or those presenting with poor prognostic features following an initial response, 3) recurrent or refractory neuroblastoma, and 4) advanced Hodgkin’s disease who have failed conventional therapy and have no HLA-matched donor. Non-covered conditions are for those individuals with 1) acute leukemia not in remission, 2) chronic granulocytic leukemia, 3) solid tumors (other then neuroblastoma), and 4) multiple myeloma.

Scheduled Commentaries. Panelists heard from 5 speakers who provided their comments regarding autologous stem cell transplantation and high dose chemotherapy in the treatment of multiple myeloma. The first speaker, Ms. Lori Williams, an oncology nurse with U.S. Oncology, authored Oncology Nursing Society Guidelines for stem cell transplantation. Ms. Williams urged Medicare to adopt a coverage policy similar to private payers, to ensure patient access to therapy, and to establish eligibility criteria for stem cell transplantation for multiple myeloma.

Dr. Ken Anderson presented evidence from published studies that supported the combination of high dose chemotherapy and stem cell transplantation as being reasonable and necessary in the treatment of multiple myeloma. He provided information on event-free survival and quality of life measures. Lastly, Dr. Anderson discussed an analysis on time without symptoms of toxicity (TWST) that has been used at his institution. Dr. Barlogie gave an overview of past and current treatment regimens for multiple myeloma. He also presented evidence from published studies and data from his own investigations in support of stem cell transplantation in multiple myeloma patients. Dr. Barlogie asserted that age is not a prognostic factor and that high dose chemotherapy promotes a much higher incidence of complete remission that results in an extension of event-free and overall survival. Dr. Anne Traynor presented a summary of the available evidence to support stem cell transplantation in the treatment of multiple myeloma. She concluded that based upon the evidence that was presented, stem cell transplantation in combination with high dose chemotherapy is a proven therapy that can benefit patients with multiple myeloma. Mr. Pearlman, a legislative assistant for Congressman John Porter, was the last speaker. Mr. Pearlman testified on behalf of Kathy Hill, a constituent of Congressman Porter. He stressed the importance of ensuring that medical advancements are made available to the public and he urged the panel to consider the data and evidence that had been presented to provide coverage for what appears to be a life-saving treatment.

HCFA Presentation. HCFA’s presenter stated the meeting objectives and reviewed the history of stem cell transplantation as a formal request for coverage. The speaker described the clinical characteristics of multiple myeloma, discussed the course of the disease, the available treatment options, and reviewed the scientific literature. HCFA reminded the panel that there was only one randomized study to demonstrate autologous stem cell transplantation improved the outcome of newly diagnosed multiple myeloma patients when compared to conventional chemotherapy. HCFA concluded their presentation by stating that the outcome of stem cell transplantation depends on the phase, stage, prognostic markers, and the resistance or sensitivity of the disease. The speaker provided the panel with an estimate of what can be achieved with stem cell transplantation, stating that complete remissions are claimed to increase from 5 to 50 percent. Regarding event-free survival, a single randomized clinical trial demonstrated an improved event-free survival from 1 to 2.3 years and an improved overall survival from 3 to 4.8 years. HCFA also presented mortality statistics for this procedure that ranged from 1, 2, 7, and 10 percent. Lastly, the presentation concluded with the observation that the ultimate fatal outcome of multiple myeloma has not changed.

Open Committee Discussion. The Carrier Medical Director gave an overview of a local medical review policy that currently does not cover the combination of high dose chemotherapy and stem cell transplantation for multiple myeloma. He indicated that claims for this treatment have been investigated, and he presented data on his findings. He felt that based on this information, there should be limitations imposed on the type of patient for whom this therapy should be offered. Panelists asked a number of questions and requested clarification concerning the data sets that were discussed by the speakers. Overall, the panelists felt that the evidence supported a conclusion that the procedure was reasonable, however they were unsure as whether it was medically necessary. One questioned whether the evidence from one randomized trial could be generalized to the Medicare population. Others questioned the data collection, the types of patients who were enrolled and enrollment criteria of the cited clinical trials. Panelists also discussed quality of life issues and how these could be measured. Other panel members felt that there was not enough data to define subgroups or those patients who would respond to stem cell therapy.

Open Public Session. Prior to the committee’s recommendation and vote, the speakers were granted time to make their final remarks. The speakers recognized that only one randomized clinical investigation had been conducted; however, they all felt that it demonstrated a benefit that should be made available to patients.

Panel Recommendation. The panel was asked to vote on the questions that were posed. The panel began with the third question.

On the third question, the panel voted unanimously in favor of the motion that appropriate measures of successful outcome for stem cell transplantation included overall survival and quality of life measures, e.g., event-free survival, time without symptoms of toxicity (TWST), etc.

On the first question, the panel voted 5 in favor of and 1 abstained from the motion that sufficient evidence existed in support of autologous stem cell transplantation for the treatment of multiple myeloma in the Medicare population. The abstaining panelist felt that additional data needed to be provided and that current evidence needed to be reanalyzed to indicate whether or not age was a prognostic variable.

Regarding the second question, the panel was unanimously in favor of the motion that age should not be a limiting factor when considering treatment. However, the panel was reluctant to determine which groups of patients should be eligible and directed HCFA to consider whether or not patients with resistant relapse should be included in establishing its coverage decision.

On the fourth question, the panel voted unanimously in favor of the motion that there was insufficient evidence presented to the panel in support of the efficacy of multiple (more than one) stem cell transplantations.

On the fifth question, the panel voted unanimously in favor the of the motion that centers performing stem cell transplantation should be certified by an appropriate accreditation organization.

Regarding the sixth question, the panel voted unanimously in favor of the motion that Medicare should not consider treatment protocols with respect to its coverage decisions.

On the seventh question, the panel voted unanimously in favor of the motion that coverage should not be related to the source of the hematopoetic stem cells.

And lastly, the panelists made an additional recommendation that detailed information regarding the risks and benefits of autologous stem cell transplantation should be developed and discussed with patients so that they may make an informed decision in collaboration with their physician.

ADJOURNMENT

The meeting was adjourned at 4:00 p.m.

I certify that I attended the meeting
of the Drugs, Biologics, and Therapeutics Panel
on September 15 and 16, 1999, and that
these minutes accurately reflect what
transpired.


Lauren Geyer, M.H.S.
Executive Secretary, HCFA

I approve the minutes of this meeting
as recorded in this summary.


Thomas Holohan, M.D. (FACP)
Chairperson
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