Wednesday, May 20, 2015

Stem Cell Transplant: CMS Opens NCD Revision; LA Times Criticizes Wild Hype

An  interesting collision of events in the past month.  Medicare has a longstanding policy for limited coverage of stem cell (bone marrow) transplants.  On April 30, 2015, it opened the NCD for comment and expansion, based on a request from the National Marrow Donor Program and the American Society for Blood and Bone Marrow Transplantation (webpage here; request here).  CMS will review future coverage for stem cell transplantation in patients with myelofibrosis and patients with sickle cell disease.

Just a couple weeks later, the Los Angeles Times publishes an expose on the field (at least under the same name) being "infected with hype" and populated even with doctors with revoked licenses (here, here).   Discussion of these puzzle pieces after the break.

FDA is holding a workshop on stem cell research & therapy & FDA regulation, according to an October 29, 2015 article in Pink Sheet (here).  The workshop will be April 13, 2016.  Pink Sheet writes, "[FDA] is planning an April public meeting to discuss the product category and four recently issued draft guidance documents addressing the regulation of human cells, tissues, or cellular or tissue-based products (HCT/Ps)."

Reopening the National Coverage Decision

The current NCD for stem cell transplantation is version 5 and was last revised in August, 2010 (here) as NCD 110.8.1.  Stem cell transplantation is defined as autologous or allogenic, writing that autologous SCT must be used after myelotoxic chemotherapy, while allogenic SCT may restore function for an inherited or acquired defect.  CMS defined the process as including all steps, when covered, and excluding all steps, when non-covered.  I believe this was related to the fact that normally, and otherwise, CMS covers treatments necessary in the wake of a non-covered service.  For example, there would be full coverage for a heart attack that occured during cosmetic surgery.  Here, CMS did not want to cover the extensive and prolonged expenses that would necessarily follow just as much from a chemoablation in a non-covered illness as chemoablation for a covered illness.

The conditions of coverage are extensive, but in a nutshell, allogenic SCT is covered for leukemia, leukemia in remission, or aplastic anemia; for severe combined immunodeficiency disease or Wiskott-Aldrich syndrome; and under CED in an approved study, for treatment of myelodysplastic syndromes.  Autologous transplant is covered for leukemia in remission; resistant non-Hodgkins lymphoma; neuroblastoma; advanced Hodgkin's disease; multiple myeloma; severe amyloidosis. A few additional conditions are noted explicitly as non-covered, while all possible conditions not stated as covered are stated as falling under MAC discretion (e.g. LCDs).

The coverage request from the ASBMT and NMDP raises a tone of urgency and states that:

   The need for finding a more effective pathway toward coverage for HSCT is urgent. Every week, physicians and our patient advocates hear from patients and their families from across the country who can no longer access HSCT once they turn 65 years old and become Medicare beneficiaries. Since time to transplant is a critical determinant of the success of a transplant, a delay or permanent barrier to transplant can lead to relapse and even death.
    Based upon information from SEER and other clinical resources, we estimate that more than 2,000 Medicare beneficiaries who need a transplant are unable to access one each year because of the lack of clarity in the national coverage policies. Based on discussion with our network transplant centers, it has become clear that expanded coverage guidance is critical to beneficiaries in need of transplantation.

The letter states that sickle cell disease, myelofibrosis, and lymphoma are conditions where SCT is "routine approved [by] the commercial payer population."  They write that while "age alone is not a factor in determining eligibility for HSCT" the Medicare population "appears to be underutilizing HSCT."  The present January 23, 2015 letter refers to a prior January 12, 2015 letter.

LCD coverage is described as inequitable and no clear guidance is provided in most MAC jurisdictions.

The NCD webpage states that new coverage will be reviewed only for (1) SCT in patients with sickle cell disease and (2) SCT in patients with myelofibrosis.

The Other World of Stem Cell Transplantation and Treatment

International interest in new horizons for stem cell transplantation is high.  California invested billions of dollars in a state-funded California Institute for Regenerative Medicine (here; see also IOM report on its governance, here).   To a large degree, stem cell transplantation is left outside of FDA regulation, as are laboratory-developed tests.   This spring, the Los Angeles Times ran several articles decrying the hype or even sham therapies proliferating in the absence of regulation.   You can see this for yourself if you run a Google search on stem cell transplants, where you can expect to get top links intermixed for a leukemia transplant program at your regional medical school and some place called "Paradise Rejuvenation Clinic."

The Los Angeles Times articles describe a doctor with a revoked licenses allegedly continuing to solicit patients for treatment in Mexico (here) and an editorial about a field "infected with hype" (here).  Science Translational Medicine recently ran a critical report about journalism in the field (Kamenova & Caulfield, 2015, here), but focused on hype of actual scientific work, not even broaching the hype of completely unregulated claims from off beat rejuvenation clinics.

For links on the FDA regulation issue, entry points include an FDA website (here), a 2015 blog on "unapproved, for profit" treatments given to humans (here), similarly WebMD on "false hope warning signs" here, and finally see also a December 2014 FDA document hoping to regulate "fat stem cells" as biological drugs (here).

Stem cell therapy is available for your dog (here).