Compendia for coverage of off-label uses of drugs and biologicals in an anti-cancer chemotherapeutic regimen

CMS evidence review | evidence_type=MEDCAC Meeting | record_type=medcac_meeting | document_id=33 | meeting_date=2006-03-30

Section 1861 (t)(2)(B)(ii)(I) of the Social Security Act lists three drug compendia that may be used in determining the “medically accepted indication” of drugs and biologicals used off-label in an anti-cancer chemotherapeutic regimen: American Hospital Formulary Service – Drug Information (AHFS-DI) American Medical Association Drug Evaluations (AMA-DE) United States Pharmacopeia – Drug Information (USP-DI) The AMA-DE is no longer in publication. The USP-DI has new ownership and will cease publication under that name, continuing under a new name. CMS has received requests for official recognition of successor and additional compendia under the following authorities: §1861(t)(2)(B), which allows the Secretary to revise the list of compendia in clause (ii)(I); and §1873, which allows the Secretary to recognize a successor publication if one of the statutorily designated publication changes its name The Medicare Coverage Advisory Committee will review and evaluate the evidence on the desirable characteristics of compendia used for this purpose, and the quality of currently published compendia.

Actions Taken: January 27, 2006 Posted Federal Register notice [PDF, 58KB] and Questions to Panel . March 27, 2006 Posted Agenda , Roster , Disclosure form for speakers , and Invited Compendia Speakers. April 4, 2006 Posted Voting Results [PDF, 148KB]. Agenda: Federal Register Notice Medicare Coverage Advisory Committee March 30, 2006 7:30 AM – 4:30 PM CMS Auditorium Agenda Alan M. Garber, MD, PhD, Chairperson Alex Krist, MD, Vice-Chairperson Steve Phurrough, MD, MPA, Coverage and Analysis Group Michelle Atkinson, Executive Secretary 7:30 – 8:00 AM Registration 8:00 – 8:20 AM Opening Remarks— M. Atkinson / S. Phurrough, MD, MPA/A.Garber, MD, PhD 8:20 9:20 AM Question #1 Review and Discussion 9:20 – 9:35 AM CMS Presentation – LCDR Tara Turner, PharmD, USPHS 9:35 – 9:45 AM BREAK 9:45 – 10:45 AM Presentation of the TA: Amy P. Abernethy, MD, Douglas C. McCrory, MD, Ethan Balk, MD MPH MHS 10:45 – 11:45AM Invited Guest Speakers (Refer to Invited Compendia Roster ) 11:45 – 12:00 PM Scheduled Public Comments: George Silberman, Independent Consultant Keith Logie, President, Indiana Medical Oncology Society, on behalf of ACCC Sharon Brigner, MS, RN, Sr. Dir. of Clinical Medical Policy, PhRMA Elizabeth Hslpern, Hogan & Hartson, on behalf of the California Healthcare Institute 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 products being discussed or with their competitors and who funded their travel to this meeting. 12:00 – 1:00PM LUNCH (on your own) 1:00 – 1:15 PM Open Public Comments Public Attendees who wish to address the panel will be given that opportunity 1:15 – 1:45 PM Questions to Presenters 1:45– 3:15 PM Open Panel Deliberations: Dr. Garber 3:15 – 4:15 PM Formal Remarks and Vote The Chairperson will ask each panel member to state his or her position on the voting questions 4:15 – 4:30 PM Closing Remarks / Adjournment: Dr. Phurrough, Dr. Garber 4:30 PM ADJOURN Disclosure Form For Speakers: This Statement Must Be Completed and Read For The Record By EVERYONE That Addresses The Committee. (This includes scheduled public speakers, invited guests, and members of the public who will speak during the open public comment period.) Name- Occupation and Employer- Speaking on behalf of: Financial Interests Do you own or have previously owned stock or have another formal financial interest in any drug compendium or any manufacturer of anti-cancer drugs or biologics ? yes/no Have you ever received financial support from any drug compendium or any manufacturer of anti-cancer drugs or biologics yes/no (This includes speaking fees, salaries, grants and other support)? If so, please disclose details. Who paid for your transportation and/or related expenses for today’s meeting? Other Conflicts Do you currently serve on, or previously served on, any other advisory committees or panels that considered the topic before the MCAC today? yes/no (This includes government panels) Were you contacted by any party prior to this meeting to discuss today’s topic? If so, please disclose details. yes/no I certify that the above statements are accurate and true. SIGNATURE/DATE Issue: Section 1861 (t)(2)(B)(ii)(I) of the Social Security Act lists three drug compendia that may be used in determining the “medically accepted indication” of drugs and biologicals used off-label in an anti-cancer chemotherapeutic regimen: American Hospital Formulary Service – Drug Information (AHFS-DI) American Medical Association Drug Evaluations (AMA-DE) United States Pharmacopeia – Drug Information (USP-DI) The AMA-DE is no longer in publication. The USP-DI has new ownership and will cease publication under that name, continuing under a new name. CMS has received requests for official recognition of successor and additional compendia under the following authorities: §1861(t)(2)(B), which allows the Secretary to revise the list of compendia in clause (ii)(I); and §1873, which allows the Secretary to recognize a successor publication if one of the statutorily designated publication changes its name The Medicare Coverage Advisory Committee will review and evaluate the evidence on the desirable characteristics of compendia used for this purpose, and the quality of currently published compendia. Other Material: Transcript 1 Voting Results Panel Voting Questions: March 30, 2006 MCAC DRAFT Questions 012705 Compendia for coverage of off-label uses of drugs and biologicals in an anti-cancer chemotherapeutic regimen 1. A good compendium should be evidence-based. What additional characteristics are desirable and of high priority in a robust, evidence-based compendium? Rate each characteristic below on its desirability and on the priority of that desirability rating. This list is provided as a reference. The MCAC may amend this list. D scores Desired Equivocal Undesired P scores High Priority Intermediate Low Priority Characteristic D score P score a Extensive breadth of listings b Quick throughput from application for inclusion to listing c Detailed description of the evidence reviewed for every individual listing d Use of prespecified published criteria for weighing evidence e Use of prespecified published process for making recommendations f Publicly transparent process for evaluating therapies g Explicit “Not Recommended” listings when validated evidence is appropriate h Bias toward “Recommended” when validated evidence is equivocal i Bias toward “Not Recommended” when validated evidence is equivocal j Explicit listing of appropriate combinations of therapies k Explicit recommendations on the sequential use of a therapy or combination in relation to other therapies l Silence, i.e. no listing, when validated evidence is equivocal m Explicit “Equivocal” listing when validated evidence is equivocal n Public identification of the members of the advisory/scientific review committee o Public notification of reviewers’ and committee members’ conflict(s) of interest, including institutional funding sources p Public notification of all funding sources of the compendium and its parent and sibling organization(s), including unrestricted grants and gifts q Net benefit analysis based on potential harm and potential benefit r Explicit stratification of the risks of available therapies 2. How confident are you that the AHFS and USPDI compendia have adequately stated evidence-based criteria and processes? Very confident — Somewhat confident — Unsure — Somewhat unconfident — Very unconfident 3. How confident are you that the AHFS and USPDI compendia adhere to evidence-based criteria and processes in making recommendations? Very confident — Somewhat confident — Unsure — Somewhat unconfident — Very unconfident 4. Considering each separately, how confident are you that compendia other than the AHFS and USPDI have adequately stated evidence-based criteria? Very confident — Somewhat confident — Unsure — Somewhat unconfident — Very unconfident 5. Considering each separately, how confident are you that compendia other than the AHFS and USPDI adhere to evidence-based criteria and processes in making recommendations? Very confident — Somewhat confident — Unsure — Somewhat unconfident — Very unconfident 6. Considering each compendium separately, please rate it on each of the desired characteristics. The list below is provided for reference. The MCAC should apply the characteristics as amended by its response to Question 1. Very confident — Somewhat confident — Unsure — Somewhat unconfident — Very unconfident Characteristic R score a Extensive breadth of listings b Quick throughput from application for inclusion to listing c Detailed description of the evidence reviewed for every individual listing d Use of prespecified published criteria for weighing evidence e Use of prespecified published process for making recommendations f Publicly transparent process for evaluating therapies g Explicit “Not Recommended” listings when validated evidence is appropriate h Bias toward “Recommended” when validated evidence is equivocal i Bias toward “Not Recommended” when validated evidence is equivocal j Explicit listing of appropriate combinations of therapies k Explicit recommendations on the sequential use of a therapy or combination in relation to other therapies l Silence, i.e. no listing, when validated evidence is equivocal m Explicit “Equivocal” listing when validated evidence is equivocal n Public identification of the members of the advisory/scientific review committee o Public notification of reviewers’ and committee members’ conflict(s) of interest, including institutional funding sources p Public notification of all funding sources of the compendium and its parent and sibling organization(s), including unrestricted grants and gifts q Net benefit analysis based on potential harm and potential benefit r Explicit stratification of the risks of available therapies 7. Do you believe that the interests of the Medicare program and its beneficiaries are best served by having a particular number or type of available published compendia on the off-label use of anti-cancer drugs and biologicals for cancer treatment? 8. If the answer to question 7 is yes, what is the minimum and/or maximum number or type of compendia that should be available? 9. How confident are you that prescribers can rely on currently available published compendia to determine appropriate off-label uses of drugs and biologicals for anti-cancer chemotherapy? Very confident — Somewhat confident — Unsure — Somewhat unconfident — Very unconfident Validity - CMS uses “validity” or related terms here as defined by Meinert, “Validity, in the context of a treatment difference, refers to the extent to which that difference can be reasonably attributed to a treatment assignment.” (Meinert CL. Clinical Trials, Overview. In: Redmond CK, Colton T, eds. Biostatistics in clinical trials. Wiley and Sons, 2001. pp. 37-51). This encompasses all issues of methodologic framework, study design, observed results, biological rationale, etc. Net health benefit - Balance between risks and benefits including complications of treatment Roster: Chair: Alan M. Garber, M.D, Ph.D. Director, Center for Primary Care and Outcomes Research Stanford University Stanford, CA Vice-Chair: Alexander H. Krist, MD Family Physician Fairfax Family Practice Center Fairfax, VA Voting Members: Deborah S. Cummins, PhD Director of Research and Evidence Analysis American Dietetic Association Chicago, IL Deborah Schrag, MD, MPH Memorial Sloan-Kettering Cancer Center New York, NY Robert S. McDonough, MD Aetna Hartford, CT Clifford Goodman, PhD Vice President The Lewin Group Falls Church, VA Daniel D. Foley, MD Vice President, Medical Affairs and Medical Director United Hospital of St. Paul Allina Health System St. Paul, MN Norman S. Kato MD, FACC, FCCP, FACS, FAHA Cardiac Care Medical Group, Inc. Encino, CA Alexander Emyr Ommaya, ScD, MA Director, Clinical Research Roundtable Institute of Medicine, The National Academies Washington, DC Nora A. Janjan, MD Professor, Dept of Radiation Oncology University of Texas M.D. Anderson Cancer Center Houston, TX Catherine A. Glennon, RN Private Diagnostic at Duke Oncology Services Health Center Administrator, Hematology/Oncology Durham, NC Mark Fendrick, MD Professor, Internal Medicine and Health Management & Policy Co-Editor in Chief, American Journal of Managed Care Ann Arbor, MI Nancy Davenport-Ennis, BA CEO Patient Advocate Foundation Newport News, VA Industry Rep: Kim K. Kuebler, MN, RN Palliative Care Nurse Practitioner Savannah, GA Consumer Rep: Linda A. Bergthold, PhD WatsonWyatt Santa Cruz, CA Guest Expert Panelists: Richard W. Whitten, MD, FACP Contractor Medical Director, Medicare B for AK, HI & WA Mark Clanton, MD, MPH Deputy Director, National Cancer Institute and Deputy Director for Cancer Care Delivery Systems Bethesda, Maryland CMS Liaison Steve Phurrough, MD, MPA Director, Coverage and Analysis Group Executive Secretary Michelle Atkinson