The Agency is proposing to amend Rule 59B-12.001 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-12.001Bone Marrow Transplantation

PURPOSE AND EFFECT: The Agency is proposing to amend Rule 59B-12.001 to update procedures based on recommendations from the Bone Marrow Transplant Advisory Panel.

SUMMARY: Rule 59B-12.001 specifies bone marrow transplant procedures that are accepted within the appropriate oncological specialty. The Agency is proposing to amend Rule 59B-12.001 to update procedures based on recommendations from the Bone Marrow Transplant Advisory Panel.

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 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: A SERC has not been prepared by the agency. For rules listed where no SERC was prepared, the Agency prepared a checklist for each rule to determine the necessity for a SERC. Based on this information at the time of the analysis and pursuant to section 120.541, Florida Statutes, the rule will not require legislative ratification.

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 FS

LAW IMPLEMENTED: 627.4236 FS

A HEARING WILL BE HELD AT THE DATE, TIME AND PLACE SHOWN BELOW:

DATE AND TIME: June 13, 2023, from 11:00 a.m. to 12:00 p.m.

PLACE: Agency for Health Care Administration, 2727 Mahan Drive, Tallahassee, Florida, 32308, Building 3, Conference Room B. You may also participate by dialing the Open Voice conference line, 1(888)585-9008, then enter the conference room number followed by the pound sign, 998-518-088#. The agenda and related materials can be found on the web at:

https://ahca.myflorida.com/MCHQ/Health_Facility_Regulation/Rulemaking.shtml

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, Florida Center, 2727 Mahan Drive, Tallahassee, Florida, 32308, [phone]. 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 at [phone] or email at: [e-mail].

 

THE FULL TEXT OF THE PROPOSED RULE IS:

 

59B-12.001 Bone Marrow Transplantation.

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 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) Allogeneic bone marrow transplant for the following:

1. Acute myelogenous leukemia and myeloid sarcoma;

2. Acute lymphoblastic leukemia;

3. Chronic myelogenous leukemia;

4. Myelodysplastic syndromes;

5. Chronic myelomonocytic leukemia;

6. Myelofibrosis;

7. Non-Hodgkin lymphoma;

8. Hodgkin lymphoma after autologous stem cell collection failure or relapsed after autologous transplant but not progressing on salvage chemotherapy;

9. Chronic lymphocytic leukemia;

10. Severe or very severe aplastic anemia from HLA compatible siblings and any type of bone marrow transplanat for acquired severe aplastic anemia unresponsive to immunosuppression or bone marrow failure syndromes;

11. Severe aplastic anemia and other bone marrow failure syndroms;

12. Thalassemia;

13. Inborn errors of immune system including severe combined immune deficiencies, primary immune deficiencies, and primary immune regulatory disorders;

14.Sickle cell disease.

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

(b) Autologous bone marrow transplant for the following:

1. Multiple myeloma (including double bone marrow transplant), Waldenstrom macroglobulinemia and primary amyloidosis;

2. Non-Hodgkin lymphoma;

3. Hodgkin lymphoma;

4. Acute myelogenous leukemia (stem cells collected in remission);

5. Neuroblastoma;

6. Germ cell tumor, after failure of first therapy but not progressing on salvage therapy;

7. Primitive neuroectodermal tumor (PNET), (including medulloblastoma and pinealoblastoma), chemotherapy sensitive after first relapse;

8. Medulloblastoma and other PNET tumors, metastatic, at diagnosis;

9. Ewing sarcoma, chemotherapy sensitive after first relapse.

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

(c) Gene and cellular therapy.  Tisagenlecleucel, a CD19-directed, genetically modified autologous T cell immunotherapy is medically necessary for the treatment of:

1. Adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy including:

a. Diffuse large B-cell lymphoma not otherwise specified;

b. high grade B-cell lymphoma and diffuse large B-cell lymphoma arising from follicular lymphoma.

2. Axicabtagene ciloleucel, a CD19-directed, genetically modified autologous T cell immunotherapy is medically necessary for adult patients with:

a. Adult patients with diffuse large B-cell lymphoma that is refractory to first line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.

b. Relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including:

(I) Diffuse large B-cell lymphoma not otherwise specified,

(II) Primary mediastinal large B-cell lymphoma,

(III) High grade B-cell lymphoma, and

(IV) Diffuse large B-cell lymphoma arising from follicular lymphoma.

c. Relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

d. Axicabtagene ciloleucel is NOT INDICATED for the treatment of patients with primary central nervous system lymphoma.

3. Brexucabtagene autoleucel, a CD19-directed, genetically modified autologous T cell immunotherapy, is medically necessary for the treatment of adult patients with:

a. Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

b. Relapsed or refractory Mantle cell lymphoma.

4. Lisocabtagene maraleucel, a CD19-directed, genetically modified autologous T cell immunotherapy, is medically necessary for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including:

a. Diffuse large B-cell lymphoma not otherwise specified;

b. High-grade B-cell lymphoma;

c. Primary mediastinal large B-cell lymphoma;

d. Diffuse large B-cell lymphoma arising from indolent lymphoma; and

e. Follicular lymphoma grade 3B.

5. Idecabtagene vicleucel, a genetically modified autologous T cell immunotherapy directed against the B-cell maturation antigen called BCMA, is medically necessary for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

6. Ciltacabtagene autoleucel, a genetically modified autologous T cell immunotherapy directed against the B-cell maturation antigen BCMA, is medically necessary for adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

7. Betibeglogene autotemcel, patient’s own bone marrow stem cells that are genetically modified to produce functional beta-globin, is medically necessary for adult and pediatric patients with ß-thalassemia who require regular red blood cell transfusions.

8. Elivaldogene autotemcel, patient’s own bone marrow stem cells that are genetically modified, is medically necessary to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy.

9. Allogeneic processed thymus tissue–agdc is medically necessary for immune reconstitution in pediatric patients with congenital athymia.

10. Sipuleucel-T, an autologous T cell immunotherapy, is medically necessary for patients with asymptomatic or minimally symptomatic metastatic castrate resistant, hormone refractory, prostate cancer.

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

(d) 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. are covered.  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.

(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) Allogeniec bone marrow transplant for multiple myeloma and other plasma cell dyscrasias; (e.g., Waldenstrom, amyloid). Autologous bone marrow transplant for chronic lymphocytic leukemia;

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

1. Chronic lymphocytic leukemia;

2. Germ cell tumor, high risk, at diagnosis;

3. Ewing sarcoma, after relapse;

4. Wilms tumor, at relapse;

5. Soft tissue sarcoma, pediatric, after failure of first therapy;

6. Multiple autologous bone marrow transplants for pediatric solid tumors;

7. Autoimmune disorders.

(c) Autologous bone marrow transplant for breast carcinoma;

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

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

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

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

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

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

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

(k) 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) 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) Prolymphocytic leukemia Myelofibrosis;

(b) Blastic plasmacytoid dendritic cell neoplasm; Chronic myelomonocytic leukemia (CMML);

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

(d) POEMS syndrome;

(e) Atypical chronic myeloid leukemia;

(f) Chronic neutrophilic leukemia;

(g) Juvenile myelomonocytic leukaemia;

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

(i) Cerebral form of adrenoleukodystoryphy and metachromatic leukodystrophy;

(j) Mucopolysaccharidoses.

(k) Treatment of other rare conditions for which there is no definitive data documenting the indication for BMT and cannot realistically be expected to generate such data, can be covered. There must be reasonable evidence of benefit from reputable sources and prior practice must have demonstrated a benefit.

(4) no change.

(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 (6) (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) No Change

(7) Phases of the BMT Episode:

(a) Evaluation:  The evaluation phase includes services required to assess and evaluate whether a patient and, in the case of allogeneic BMT, the donor, are suitable for the transplantation procedure. It may also include evaluations to assess whether a transplantation is an appropriate treatment option for the patient.

(b) Pretransplantation Care:  The pretransplantation care phase involves care provided from the time a patient is identified as a candidate for BMT and includes all related care until the initiation of conditioning regimen.

(c) Transplantation Event:  The transplantation event phase usually starts from the day of starting conditioning regimen and it can last from 30 to 120 days after transplantation. This phase covers the hematopoietic stem cell infusion and the transplantation hospitalization, and it also typically includes graft procurement, stem cell mobilization, and processing. In some situations, this phase can extend for a longer period of time (eg, tandem transplantation for germ cell tumors). This phase also includes any clinic visits associated with providing care to patients receiving an outpatient transplantation.

(d) Follow-Up Care:  The follow-up care phase of the BMT episode of care starts on completion of the transplantation event phase that may extend up to one year.  However, transplant physicians will continue caring for patients life long, to deal with late effects of therapy including chronic GVHD from allogeneic transplant.  Therefore, patients are never truly discharged from transplantation center follow-up.

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, 2-4-19,_________.

 

NAME OF PERSON ORIGINATING PROPOSED RULE: Dana Watson

NAME OF AGENCY HEAD WHO APPROVED THE PROPOSED RULE: Jason Weida

DATE PROPOSED RULE APPROVED BY AGENCY HEAD: 4/21/2023

DATE NOTICE OF PROPOSED RULE DEVELOPMENT PUBLISHED IN FAR: 3/1/2023