The Agency is proposing to amend Rule 59B-12.001, F.A.C., to update procedures based on recommendations from the Bone Marrow Transplant Advisory Panel.

AGENCY FOR HEALTH CARE ADMINISTRATION
Cost Management and Control

RULE NO.: RULE TITLE:
59B-12.001: Bone Marrow Transplantation
PURPOSE AND EFFECT: The Agency is proposing to amend Rule 59B-12.001, F.A.C., to update procedures based on recommendations from the Bone Marrow Transplant Advisory Panel.
SUMMARY: The proposed amendments include coverage for cellular therapies which include cellular immunotherapies, chimeric antigen receptor (CAR) T cells, cancer vaccines, and other types of autologous and allogeneic cells for certain therapeutic indications. The language mirrors that of the Food and Drug Administration’s language for approved cellular therapy.
New language adds to the specific conditions for which the use of cellular therapies is necessary. The amendments also adjust the conditions in sections one through three to add or move conditions to the proper section.
SUMMARY OF STATEMENT OF ESTIMATED REGULATORY COSTS AND LEGISLATIVE RATIFICATION:
The Agency has determined that this will not have an adverse impact on small business or likely increase directly or indirectly regulatory costs in excess of $200,000 in the aggregate within one year after the implementation of the rule. A SERC has not been prepared by the Agency.
Any person who wishes to provide information regarding a statement of estimated regulatory costs, or provide a proposal for a lower cost regulatory alternative must do so in writing within 21 days of this notice.
RULEMAKING AUTHORITY: 627.4236 F.S.
LAW IMPLEMENTED: 627.4236 F.S.
IF REQUESTED WITHIN 21 DAYS OF THE DATE OF THIS NOTICE, A HEARING WILL BE HELD AT THE DATE,TIME AND PLACE SHOWN BELOW(IF NOT REQUESTED, THIS HEARING WILL NOT BE HELD):
DATE AND TIME: Tuesday, October 16, 2018, 9:00 A.M.
PLACE: Agency for Health Care Administration, Building #3, Florida Center Conference Room, 2727 Mahan Dr., Tallahassee, FL 32308
Pursuant to the provisions of the Americans with Disabilities Act, any person requiring special accommodations to participate in this workshop/meeting is asked to advise the agency at least 3 days before the workshop/meeting by contacting: Dana Watson, [phone], email:[e-mail].. If you are hearing or speech impaired, please contact the agency using the Florida Relay Service, 1(800)955-8771 (TDD) or 1(800)955-8770 (Voice).
THE PERSON TO BE CONTACTED REGARDING THE PROPOSED RULE IS: Dana Watson, [phone], email:[e-mail].

THE FULL TEXT OF THE PROPOSED RULE IS:

        59B-12.001 Bone Marrow Transplantation

Bone marrow transplant refers collectively to hematopoietic stem cell transplantation using stem cells that are collected from peripheral blood and cord blood as well as bone marrow following a conditioning regimen. As used in this rule, the term “appropriate oncological specialty” means that where a particular kind of tumor or disease is usually treated by a subspecialty group within the general discipline of oncology, those who practice within that subspecialty have had specific input into the decision making process.  Cellular therapies therapy products include cellular immunotherapies, chimeric antigen receptor (CAR) T cells, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications. Human gene therapy refers to products that introduce genetic material into a person’s DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.

(1) Upon the recommendation of the Bone Marrow Transplant Panel, each of the following procedures meets a minimum level of evidence based on high quality systematic reviews of case control or cohort studies, high quality case-control or cohort studies with a very low risk of confounding bias, or chance, and a high probability that the relationship is causal, and is considered accepted within the appropriate oncological specialty and not experimental for the purposes of Section 627.4236, F.S.

(a) Autologous bone marrow transplant for acute myelogenous leukemia (stem cells collected in remission);

(b) Allogeneic bone marrow transplant for acute myelogenous leukemia and myeloid sarcoma;

(c) Allogeneic bone marrow transplant for acute lymphoblastic leukemia;

(d) Allogeneic bone marrow transplant for chronic myelogenous leukemia;

(e) Autologous bone marrow transplant for Hodgkin lymphoma;

(f) Allogeneic bone marrow transplant for Hodgkin lymphoma after autologous stem cell collection failure or relapsed after autologous transplant but not progressing on salvage chemotherapy;

(g) Autologous bone marrow transplant for non-Hodgkin lymphoma;

(h) Allogeneic bone marrow transplant for non-Hodgkin lymphoma;

(i) Autologous bone marrow transplant for Ewing sarcoma, chemotherapy sensitive after first relapse;

(j) Autologous bone marrow transplant for neuroblastoma;

(k) Autologous bone marrow transplant for germ cell tumor, after failure of first therapy but not progressing on salvage therapy;

(l) Autologous bone marrow transplant for multiple myeloma (including double bone marrow transplant), Waldenstrom macroglobulinemia and primary amyloidosis;

(m) Allogeneic bone marrow transplant for myelodysplastic syndrome;

(n) Autologous bone marrow transplant for primitive neuroectodermal tumor (PNET), (including medulloblastoma and pinealoblastoma), chemotherapy sensitive after first relapse;

(o) Autologous bone marrow for medulloblastoma and other PNET tumors, metastatic, at diagnosis;

(p) Allogeneic bone marrow transplant for chronic lymphocytic leukemia;

(q) Allogeneic bone marrow transplant for severe or very severe aplastic anemia from HLA compatible siblings  and any type of bone marrow transplant for acquired or genetic severe aplastic anemia unresponsive to immunosuppression;

(r) Allogeneic bone marrow transplant for sickle cell anemia, thalassemia, and other severe red cell disorders; and,

(s) Allogeneic bone marrow transplant for severe combined immune deficiency disorder  and other severe immune deficiency disorders.

(t) Tisagenlecleucel, a CD19-directed, genetically modified autologous T cell immunotherapy is medically necessary for patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse.

(u) Axicabtagene ciloleucel, a CD19-directed, genetically modified autologous T cell immunotherapy is medically necessary for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and diffuse large B-cell lymphoma arising from follicular lymphoma.

(v) Cellular therapies that are Food and Drug Administration (FDA)-approved for a specific indication and are medically necessary,  accepted within the appropriate oncological specialty and not experimental for the purposes of Section 627.4236, F.S.

In cases where treatment for any of the above conditions includes a clinical trial that conforms to

subsection (6) routine care costs associated with the bone marrow transplant will be covered.

(2) Each of the following procedures is considered accepted within the appropriate oncological specialty and not experimental for the purposes of Section 627.4236, F.S., provided that the bone marrow transplantation procedure is performed in the context of a well-designed clinical treatment trial as described in subsection (6). 

Routine care costs associated with the bone marrow transplant will be covered for the following procedures:

(a) Autologous bone marrow transplant for chronic lymphocytic leukemia;

(b) Autologous bone marrow transplant for plasma cell dyscrasias other than multiple myeloma (e.g., Waldenstrom);

(b) (c) Allogeneic bone marrow transplant for multiple myeloma and other plasma cell dyscrasias (e.g., Waldenstrom, amyloid);

(c) (d) Autologous bone marrow transplant for breast carcinoma;

(d) (e) Autologous bone marrow transplant for Ewing sarcoma, localized, greater than eight cm or metastatic at presentation;

(e) (f) Autologous bone marrow transplant for soft tissue sarcoma, pediatric, after failure of first therapy;

(f) (g) Autologous bone marrow transplant for Wilms tumor, at relapse;

(g) (h)Autologous bone marrow transplant for germ cell tumor, high risk, at diagnosis;

(h) (i) Allogeneic bone marrow transplant for renal cell carcinoma;

(i) (j)Multiple autologous bone marrow transplants for pediatric solid tumors;

(k) Allogeneic bone marrow transplant for Hodgkin lymphoma;

(j) (l)Autologous bone marrow transplant for metastatic malignant melanoma;

(k) (m) Allogeneic bone marrow transplant for sickle cell anemia, thalassemia, and other severe red cell disorders; and,

(l) Cellular therapies and human gene therapies that are provided on clinical trial at a Blood and Marrow Transplant Clinical Trials Network (BMT CTN) core or non-core center, and are accepted within the appropriate oncological specialty and not experimental for the purposes of Section 627.4236 F.S. 

(m) (n) Autologous bone marrow transplant for autoimmune disorders.

(3) The following rare diseases, where there are no existing clinical trials available, are covered for bone marrow transplant at the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) core or non-core facilities when deemed medically necessary:

(a) Myelofibrosis;

(b) Chronic myelomonocytic leukemia (CMML);

(c) Paroxysmal nocturnal hemoglobinuria (PNH); and,

(d) POEMS syndrome.

(4) Transplants from living related donors incompatible for one HLA-A, -B, and -DRB1 loci are covered for bone marrow transplant at BMT CTN core or non-core medical facilities.

(5) Any bone marrow transplant performed outside of a clinical trial will be covered when all the following criteria are met:

(a) The plan of care follows a clinical trial protocol that meets the requirements of subsection (5);

(b) Patient cannot be enrolled in the proposed clinical trial;

(c) Bone marrow transplant treatment is medically necessary;

(d) Patient is an appropriate candidate for bone marrow transplant; and,

(e) Treatment center is part of the BMT CTN at a core or non-core center.

(6) A well-designed and conducted clinical treatment trial is one which includes an IRB-approved written protocol. At a minimum, such protocol shall have specific criteria for evaluating the effect of treatment with defined endpoints that are precise, meaningful, and reliable and which allow valid conclusions to be drawn about therapeutic efficacy and safety. Protocols should include an adequate statistical section describing the method of randomization and stratification, if any, expected outcome parameters relating to response rates, time to progression, survival times and other relevant information. Such clinical treatment trials shall be consistent with protocols reviewed and approved by the National Cancer Institute for scientific merit.

Rulemaking Authority 627.4236 FS. Law Implemented 627.4236 FS. History–New 11-9-95, Formerly 10D-127.001, Amended 9-26-00, 8-10-05, 7-7-13, 7-12-15,                   .

 

NAME OF PERSON ORIGINATING PROPOSED RULE: Dana Watson, [phone], email: [e-mail].
NAME OF AGENCY HEAD WHO APPROVED THE PROPOSED RULE: Secretary Justin Senior
DATE PROPOSED RULE APPROVED BY AGENCY HEAD: August 23, 2018
DATE NOTICE OF PROPOSED RULE DEVELOPMENT PUBLISHED IN FAR: 6/21/2018