FEATURE
Timeline of the National Coverage Determination for AlloHCT in Multiple Myeloma , Myelofibrosis , and Sickle Cell Disease
The changes to the National Coverage Determination ( NCD ) for allogeneic hematopoietic cell transplantation ( alloHCT ) have been a long time coming . Here is a timeline of the actions – from CMS ’ “ silence ” on alloHCT for the treatment of sickle cell disease and myelofibrosis to the most recent changes in coverage .
April 30 , 2015 : Formal request for coverage determination initiated by Sergio Giralt , MD , president of the American Society for Blood and Marrow Transplantation , and Michael Boo , JD , chief strategy officer of the National Marrow Donor Program ( ASH supported the request .)
April 30 , 2015 through May 30 , 2015 : Public comment period
October 29 , 2015 : Proposed NCD memo released
October 29 , 2015 through November 28 , 2015 : Proposed NCD public comment period ( ASH submitted comments on November 27 .)
January 27 , 2016 : NCD memo released
protocol meets CMS requirements for study quality , Dr . Horowitz noted .
The U . S . Stem Cell Therapeutic Outcomes Database , a component of the C . W . Bill Young Cell Transplantation Program , is another organization charged with collecting and assessing outcomes data for all U . S . allogeneic transplants . Dr . Horowitz is the research and project director of the database .
In addition , many individual transplant centers often keep their own registries on alloHCT patient outcomes , Ms . Farnia pointed out . She encourages centers to continue that practice . The trials under the NCD CED “ are intended to be national studies that can be opened at all transplant centers ,” she said , “ but there are other studies that individual transplant centers can submit to CMS . We don ’ t see a conflict with that right now .”
Overall , the doctors who spoke with ASH Clinical News are optimistic that protocols and registries that meet the CED requirements will be in place by the end of 2016 , despite that even the advocates who worked closely with CMS on this NCD did not necessarily have information beforehand about its specific details .
To further complicate matters , it is not clear how much data and how long of a delivery timeframe will be enough . Ms . Farnia noted that the NCD does not address either of these points .
“ We don ’ t know how long these studies are going to last ,” she explained . “ For instance , the MDS CED is a study that ’ s been open since 2010 , and Medicare doesn ’ t give a timeline or minimum number of patients required to satisfy it . Do they want to see data for three years ? For 300 patients ? We don ’ t know how long the additional data gathering is going to be required .”
The stringent criteria laid out in the CED prompted the American Society of Hematology ( ASH ) to caution , during the NCD review process , that the CED “ will yield small numbers of patients and insufficient power to add meaningful clinical questions about prognostic factors for patients , policymakers , and physicians .” 4
Nonetheless , the CIBMTR has already laid out target dates for submitting trial protocols to CMS , most of which fall before summer 2016 . In myelofibrosis , the hope is that an existing CIBMTR study can be adapted to be compliant , while in SCD , the SCURT trial ( Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease ; BMT CTN 0601 ) is active . 5 Finally , in MM , the hope is to use patients who have received autologous hematopoietic cell transplantations ( AHCT ) as controls .
One of the advantages that the transplant community has over other areas of medicine is that it has been engaged in data-tracking on patient outcomes for some time .
“ The transplant community is really well organized ,” Dr . Komanduri said . “ There ’ s excellent cooperation between NMDP , ASBMT , and CIBMTR . We have a really cohesive group of organizations in hematology and transplantation that are used to taking on these kinds of [ data collection ] challenges .”
Dr . Horowitz noted that autoHCT is an accepted Medicare-covered standard therapy for many of these disease states . Because of the work CIBMTR and the transplant community as a whole has done for MDS , she is confident that CED-ready studies can be designed and launched fairly quickly for the new NCD .
Now What ?
With clinicians and their patients in a holding pattern as the logistics of the CED are sorted out and implemented , what are the next steps ?
Ms . Farnia recommended that clinicians continue to work with their transplant centers and local Medicare contractors on a patient-by-patient basis .
“ We would say [ to ] always refer a patient who might be eligible for transplant to your transplant center ,” she explained . “ Then we are suggesting that transplant centers have conversations with their local Medicare contractors to see if they can find a workaround on a local level . We are continuing to have conversations with Medicare about what we can do in the interim at a national level .”
Dr . Burns recommended that clinicians continue to follow various published treatment guidelines – developed by NMDP , ASBMT , and the National Comprehensive Cancer Network – to determine which patients should be referred to transplant centers . “ These are based on the best evidence available and regularly updated ,” she said .
Dr . Komanduri suggested that interested parties continue to check in regularly at the ASBMT and NMDP websites for updates on the situation – and to have some patience . “ I ’ ve already gotten emails on the local level from physicians saying , ‘ I understand that we can now refer Medicare patients to you for allogenic transplant for SCD .’ I have to walk them back , and say , ‘ Yes , that
“ This decision is a significant milestone that reflects our advances in the art and science of stem cell transplantation ... but there are some caveats .”
will be possible , but there are some criteria that have to be met first .’”
Dr . Komanduri also emphasized the importance of early referral of patients to experienced hematologists and transplant centers to discuss all available treatment options – alloHCT or otherwise .
“ For MM , the primary treatment approach has been autologous transplant , so allogeneic transplant is not typically used as an upfront option . It ’ s generally used in the salvage setting and , even then , in the context of clinical trials ,” he explained . “ For SCD and myelofibrosis , these diseases may have an indolent course , and there are certain subsets of patients who should not be considered for allogeneic transplant . There are multiple treatment options that should be considered .”
For Dr . Horowitz , there is still some concern that the NCD will put a stopper on alloHCT for Medicareeligible patients who were covered during the “ silent ” era , simply because local Medicare contractors will want to wait to see how the CED requirements play out .
“ There are a number of places in the country where patients were being appropriately covered , in my opinion . Those patients are now in limbo ; it ’ s uncertain as to how that ’ s going to be handled ,” she said . “ We are in a situation where , in the short term , access to alloHCT may be hindered rather than helped . Of course , there are also many areas where local contractors did not approve allogenic transplantation , so in the long run , this [ NCD ] will benefit everyone .” — By Shalmali Pal ●
References
— KRISHNA KOMANDURI , MD
1 . Centers for Medicare & Medicaid Services . Decision Memo for Stem Cell Transplantation ( Multiple Myeloma , Myelofibrosis , and Sickle Cell Disease ) ( CAG-00444R ). Accessed March 12 , 2016 from https :// www . cms . gov / medicare-coverage-database / details / ncadecision-memo . aspx ? NCAId = 280 .
2 . Moulard O , Mehta J , Fryzek J , et al . Epidemiology of myelofibrosis , essential thrombocythemia , and polycythemia vera in the European Union . Eur J Haematol . 2014 ; 92:289-97 .
3 . Ballen KK , Shrestha S , Sobocinski KA , et al . Outcome of transplantation for myelofibrosis . Biol Blood Marrow Transplant . 2010 ; 16:358-67 .
4 . “ American Society of Hematology ’ s Comments on Proposed National Coverage Determination on Stem Cell Transplantation ( letter to the Centers for Medicare & Medicaid Services ).” November 27 , 2015 .
5 . ClinicalTrials . gov . Evaluating the safety and effectiveness of bone marrow transplants in children with sickle cell disease ( BMT CTN 0601 ) ( SCURT ). Accessed March 12 , 2016 from https :// clinicaltrials . gov / ct2 / show / NCT00745420 ? term = 00745420 & rank = 1 .
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