The Agency is proposing to amend 59B-12.001 to update accepted procedures, and adds provisions for transplants from living related donors, based on recommendations by the Bone Marrow Advisory Panel.

 

AGENCY FOR HEALTH CARE ADMINISTRATION

Cost Management and Control

RULE NO.:RULE TITLE:

59B-12.001Bone Marrow Transplantation

PURPOSE AND EFFECT: The Agency is proposing to amend Rule 59B-12.001, F.A.C. to update accepted procedures, and adds provisions for transplants from living related donors, based on recommendations by the Bone Marrow Advisory Panel.

SUMMARY: The proposed amendments to this rule include: updates to accepted procedures in bone marrow transplantation and adds provisions for transplants from living related donors.

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.

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.

The Agency has determined that the proposed rule is not expected to require legislative ratification based on the statement of estimated regulatory costs or if no SERC is required, the information expressly relied upon and described herein: The Agency has determined that the proposed rule is not expected to require legislative ratification pursuant to Section 120.541(3), F.S., based on the statement of estimated regulatory costs or if no SERC is required, the information expressly relied upon and described herein: A checklist was prepared by the Agency to determine the need for a SERC. As there will be no impact on economic growth, job creation or employment, private-sector investment, or business competitiveness and no increase in regulatory costs—no adverse impact is likely. Any person who wishes to provide information regarding a statement of estimated regulatory cost, or provide a proposal for a lower cost regulatory alternative must do so in writing within 21 days of this notice.

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 (3)(a) FS.

LAW IMPLEMENTED: 627.4236 FS.

IF REQUESTED WITHIN 21 DAYS OF THE DATE OF THIS NOTICE, A HEARING WILL BE HELD AT THE DATE, TIME AND PLACE SHOWN BELOW:

DATE AND TIME: April 17, 2015, 9:00 a.m. - 10:00 a.m.

PLACE: Agency for Health Care Administration, Building 3, Conference Room C, 2727 Mahan Drive, Tallahassee, FL 32308

THE PERSON TO BE CONTACTED REGARDING THE PROPOSED RULE IS: Dana Watson, Florida Center for Health Information and Policy Analysis, 2727 Mahan Drive, Mail Stop #16, Building 3, Tallahassee, Florida. Email [e-mail] or call [phone].

 

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.

(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 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) 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; and

(q) Allogeneic bone marrow transplant for severe or very severe aplastic anemia animia from HLA compatible siblings for patients below 40 years of age, and any type of bone marrow transplant for severe aplastic anemia unresponsive to immunosuppression.

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

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

In cases where treatment for any of the above conditions includes a clinical trial that conforms to subsection (6)(5), 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 and conducted Phase II or Phase III clinical treatment trial as described in subsection (6)(5).

(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);

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

(d) Autologous bone marrow transplant for breast carcinoma;

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

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

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

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

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

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

(k) Allogeneic bone marrow transplant for Hodgkin lymphoma;

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

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

(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)(4) 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)(5) 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, _________.

 

NAME OF PERSON ORIGINATING PROPOSED RULE: Dana Watson

NAME OF AGENCY HEAD WHO APPROVED THE PROPOSED RULE: Secretary Elizabeth Dudek

DATE PROPOSED RULE APPROVED BY AGENCY HEAD: January 5, 2015

DATE NOTICE OF PROPOSED RULE DEVELOPMENT PUBLISHED IN FAR: September 24, 2014