Extracorporeal Photopheresis (ECP)

CMS NCA document | source_status=Closed | review_type=2nd Recon | public_comment_open=False | document_id=CAG-00324R2

CMS covers extracorporeal photopheresis (ECP) for the treatment of bronchiolitis obliterans syndrome (BOS) following lung allograft transplantation only when ECP is provided under a clinical research study that meets the following conditions. The clinical research study meets the requirements specified below to assess the effect of ECP for the treatment of BOS following lung allograft transplantation. The clinical study must address one or more aspects of the following question: Prospectively, do Medicare beneficiaries who have received lung allografts, developed BOS refractory to standard immunosuppressive therapy, and received ECP, experience improved patient-centered health outcomes as indicated by: improved forced expiratory volume in one second (FEV1), or decreased rate of decline of FEV1; improved survival after transplant; and/or improved quality of life? This decision does not modify existing requirements for any other covered or non-covered indication for ECP (Section 110.4 Medicare National Coverage Determinations Manual). The required clinical study must adhere to the following standards of scientific integrity and relevance to the Medicare population: The principal purpose of the research study is to test whether ECP potentially improves the participants’ health outcomes. The research study is well supported by available scientific and medical information or it is intended to clarify or establish the health outcomes of interventions already in common clinical use. The research study does not unjustifiably duplicate existing studies. The research study design is appropriate to answer the research question being asked in the study. The research study is sponsored by an organization or individual capable of successfully executing the proposed study. The research study is in compliance with all applicable Federal regulations concerning the protection of human subjects found at 45 CFR Part 46. If a study is regulated by the Food and Drug Administration (FDA), it must also be in compliance with 21 CFR parts 50 and 56. All aspects of the research study are conducted according to appropriate standards of scientific integrity (see http://www.icmje.org). The research study has a written protocol that clearly addresses, or incorporates by reference, the standards listed here as Medicare requirements for CED coverage. The clinical research study is not designed to exclusively test toxicity or disease pathophysiology in healthy individuals. Trials of all medical technologies measuring therapeutic outcomes as one of the objectives meet this standard only if the disease or condition being studied is life threatening as defined in 21 CFR § 312.81(a) and the patient has no other viable treatment options. The clinical research study is registered on the ClinicalTrials.gov website by the principal sponsor/investigator prior to the enrollment of the first study subject. The research study protocol specifies the method and timing of public release of all prespecified outcomes to be measured including release of outcomes if outcomes are negative or study is terminated early. The results must be made public within 24 months of the end of data collection. If a report is planned to be published in a peer reviewed journal, then that initial release may be an abstract that meets the requirements of the International Committee of Medical Journal Editors (http://www.icmje.org). The research study protocol must explicitly discuss subpopulations affected by the treatment under investigation, particularly traditionally underrepresented groups in clinical studies, how the inclusion and exclusion criteria effect enrollment of these populations, and a plan for the retention and reporting of said populations on the trial. If the inclusion and exclusion criteria are expected to have a negative effect on the recruitment or retention of underrepresented populations, the protocol must discuss why these criteria are necessary. The research study protocol explicitly discusses how the results are or are not expected to be generalizable to the Medicare population to infer whether Medicare patients may benefit from the intervention. Separate discussions in the protocol may be necessary for populations eligible for Medicare due to age, disability or Medicaid eligibility. Consistent with section 1142 of the Social Security Act, AHRQ supports clinical research studies that CMS determines meet the above-listed standards and address the above-listed research questions. Any clinical study undertaken pursuant to this NCD must be approved no later than April 30, 2014, two (2) years from the effective date of this NCD. If there are no approved clinical studies on or before April 30, 2014, this NCD for ECP treatment for patients who have received lung allografts and then developed progressive BOS refractory to immunosuppressive drug treatment will expire, and coverage will revert to the policy in effect prior to April 30, 2012. Any clinical study approved by April 30, 2014, will adhere to the timeframe designated in the approved clinical study protocol.