The state of Washington Health Technologies Coverage Determination:

ABA based behavioral interventions for treatment of Autism Spectrum Disorder is a covered benefit with Evidence Development (CED)

HTCC Reimbursement Determination ␣ Limitations of Coverage

␣ Certain Applied Behavioral Analysis based behavioral interventions are a covered benefit for the treatment of Autism Spectrum Disorder when the following conditions are met:

• The Early Intensive Behavioral/developmental Intervention (EIBI) using either the UCLA/Lovaas model or Early Start Denver Model (ESDM) are both covered with evidence development

Briefing Background: The Health Technology Clinical Committee has made a draft coverage decision for Applied Behavioral Analysis for Autism Spectrum Disorder for conditional coverage: the condition is coverage with evidence development. The HTCC requested staff provide a briefing document to assist the HTCC with finalizing the coverage decision and potentially adding more specific information.

This document provides: ␣ a brief background and description of coverage with evidence development (CED) ␣ example parameters for CED ␣ Resources

BACKGROUND Health care payers often face a situation where a decision on coverage of a medical service or item is requested, but there is inadequate information about efficacy, effectiveness, safety, and/or cost. Coverage with data collection, or evidence development, or post market/field evaluation (collectively CED) is a potential policy approach to reduce uncertainty by permitting access to the technology in an evaluation setting appropriate to questions that patients, clinicians, and payers have. Use of this policy approach by payers is relatively new and has been tried by Medicare (since 2005) and some US Payers, and in Canada (e.g. Ontario since 2003) and other European health systems. CED provides interim access to medical interventions while generating clinical evidence to inform a decision about whether coverage is appropriate with or without limitations.

In most instances, the HTCC uses the health technology assessment report (a summary and evaluation of the current medical evidence) to determine whether sufficient evidence of a technologies’ safety, effectiveness, and cost-effectiveness exists to support coverage. Principles and decision factors considered by the HTCC related to the sufficiency of evidence; determination of health benefit; and other factors for consideration are included in the HTCC decision tool document. Generally, if evidence is not adequate to support a coverage decision, non-coverage would result. In some cases however, the evidence may be sufficient, together with other decision factors, to determine that coverage in the context of further evaluation and study is appropriate. When that occurs, coverage is appropriate when provided within a research setting where added safety, patient protections, monitoring, and clinical expertise are present; and important outcomes are being appropriately studied.

CED Considerations

Once a decision has been made to cover with evidence development, a payer needs to identify the key uncertainties that need to be addressed by the evidence; and then either design the CED program or provide guidance or criteria for studies that could qualify. For the HTA process, the HTCC decides on coverage conditions and agencies decide on implementation. For the ABA Therapy for Autism Spectrum Disease, the HTCC has made a draft decision to cover with the condition of evidence development and requested this briefing on CED to determine what further conditions, if any should be included.

Key uncertainties: The HTCC may want to highlight the key uncertainties and outcomes, or the information may already be captured in the HTCC decision tool; meeting materials; discussion at the public meeting; the draft minutes; and/or the committee findings and conclusions.

CED Program Criteria, Conditions or Guidance: CED programs have ranged from payers designing, financing, and implementing evaluations, studies and trials; to issuing guidance for when the payerwill participate through reimbursement for the intervention. Guidance ranges from general outcomes information to detailed study criteria. Excerpted examples are below, followed by a reference list for the full citations.

CED programs should be accompanied by a clear explanation of the evidence required to warrant conventional coverage. In addition, they should be governed by research plans that identify the institution(s) responsible for the research, define the primary and secondary outcomes under study, estimate the number of patients needed to have adequate power to answer the coverage-relevant research questions, and specify methods of data analysis. In the case of randomized trials, guidelines for early termination based on interim data should be established and the data should be monitored by an independent Data Monitoring Committee. Miller, et.al, July 2008 Medical Care, V47,7; 746 ///

Clinical trials sponsored by other entities may also be covered if approved by the benefits administrator, provided that the clinical trial has passed independent scientific review and has also been approved by an Institutional Review Board (IRB) that would oversee the trial; and the clinical trial must be conducted in a setting and by personnel who maintain a high level of expertise because of their training, experience and volume of patients. An IRB is an independent ethics committee usually associated with a university or physician-accrediting organization formally designated to approve, monitor and review biomedical and behavioral research involving humans with the aim to protect the rights and welfare of the subjects. CMTP Framework 2008

To qualify for reimbursement, such a study must be designed to produce evidence that could be used in a future national coverage decision that would focus on whether the item or service should be covered by Medicare under 1862(a)(1)(A). Payment for the items and services provided in the study will be restricted to the Medicare qualified patients involved as human subjects in the study. CMS

CMS will not routinely be involved in the design, review, or execution of research studies. CMS will only provide payment for clinical research that meets the standards of a qualified trial as will be outlined in the revision of the Clinical Trial Policy. We anticipate this NCD will include the following principles:

1. The primary purpose of the trial is to test whether the intervention potentially improves the participants' health outcomes.

2. The trial is well supported by available scientific and medical information or is intended to clarify or establish the health outcomes of interventions already in common clinical use.

3. The trial does not duplicate existing studies unjustifiably. 4. The trial design is appropriate to answer the research question being asked in the trial. 5. The trial is sponsored by a credible organization or individual capable of executing the proposed trial

successfully. 6. The trial is in compliance with Federal regulations relating to the protection of human subjects. 7. All aspects of the trial are conducted according to the appropriate standards of scientific integrity. 8. The trial is listed in the National Library of Medicine clinical trials database. 9. The sample of study subjects in the trial should include individuals representative of the Medicare population

with the condition described in the NCD. ///

Clinical Trial Coverage Criteria (Regence) Costs for medical services (e.g., inpatient hospitalization, outpatient services, and other ancillary services, such as laboratory tests and imaging) that are associated with an emerging technology that Regence considers investigational may be eligible for coverage when all of the following criteria (A-F) are met:

A. The treatment being studied is therapeutic, intended to directly improve health outcomes, and not for diagnosis or supportive care. B. The treatment being studied is for a condition that is either life-threatening or severely and chronically disabling and that has a poor prognosis with the most effective available treatment. 1) A condition is considered to be life-threatening if it has a substantial probability of causing premature death; 2) A condition is considered severely and chronically disabling if the individual with the condition is unable to perform the functions that are required for daily life and if the severe disability is not expected to improve with the most effective available treatment. C. The member is enrolled and participating in a phase II or phase III clinical trial that is approved by a national body, such as the National Institutes of Health, the National Cancer Institute, or the FDA. D. The clinical trial is conducted under a written research protocol with Institutional Review Board approval. E. There is published evidence from phase II clinical trials that indicates the treatment has a substantial probability of providing a clinically significant and substantial improvement in net health outcomes compared to the most effective available treatment. F. It is likely that the evidence from well-designed clinical trials, including the trial in which the member is participating, will contribute to determining whether the treatment meets the Regence technology assessment criteria detailed in the Introduction to the Regence Medical Policy Manual (see Cross References). Regence

The Centers for Medicare & Medicaid Services (CMS) is expanding coverage for Medicare beneficiaries by requiring national coverage for certain anticancer drugs in National Cancer Institute (NCI) sponsored trials. CMS will cover the studied drug and routine costs of the trials using the following anticancer drugs: oxaliplatin (EloxatinTM), irinotecan (Camptosar®), bevacizumab (AvastinTM), and Cetuximab (ErbituxTM). Consult the following NCI web page for details of the trials: http://www.cancer.gov/clinicaltrials

␣ C80405 is a phase III, first-line metastatic colorectal cancer trial. It consists of multiple arms: FOLFOX, FOLFIRI, CAPOX, or CAPIRI plus bevacizumab; FOLFOX, FOLFIRI, CAPOX, or CAPIRI plus cetuximab; and FOLFOX, FOLFIRI, CAPOX, or CAPIRI plus both bevacizumab and cetuximab. ... etc

An FDG PET scan is considered reasonable and necessary in patients with MCI or early dementia (in clinical circumstances other than those specified in subparagraph 1) only in the context of an approved clinical trial that contains patient safeguards and protections to ensure proper administration, use and evaluation of the FDG PET scan. The clinical trial must compare patients who do and do not receive an FDG PET scan and have as its goal to monitor, evaluate, and improve clinical outcomes. In addition, it must meet the following basic criteria:

Written protocol on file;

1. 2. 3.

Institutional Review Board review and approval; Scientific review and approval by two or more qualified individuals who are not part of the research team; and, Certification that investigators have not been disqualified.

CED by private payers requires each payer to deem a trial worthy of reimbursement of clinical care costs. Doing that individually for every CED project (assuming CED becomes more common) is quite burdensome and likely to be unsuccessful. However, it still needs to be done independently among participating health plans, and since many health plans do not have staff who can evaluate study design, the role of an independent third party becomes more crucial. Plans may be more interested in engaging a neutral organization like CMTP in a coordinator role that would certify study designs against a set of payer/stakeholder generated CED requirements. CMTP – Lessons 2010

Resources␣

Miller, et..al. Coverage With Evidence Development: Ethical Issues and Policy Implications; Medical Care •July 2008, Volume 46, Number 7, 746.

Levin, et al, Coverage with evidence development: the Ontario experience, Int J Technol Assess Health Care. 2011 Apr;27(2):159-68.

Guidance for the Public, Industry, and CMS Staff; National Coverage Determinations with Data Collection as a Condition of Coverage: Coverage with Evidence Development, Issued July 12, 2006 Downloaded from https://www.cms.gov

Research Urgent Treatments – Regency Policy.

http://blue.regence.com/trgmedpol/medicine/med74.html

Coverage for Evidence Development: A Conceptual Framework, Center for Medical and Technology Policy(CMTP) http://www.cmtpnet.org/cmtp-research/applied-policy-and-methods/coverage-with-evidence- development/20090108%20-%20CMTP%20-%20CED%20Issue%20Brief.pdf

Coverage for Evidence Development in the Private Sector: Lessons in Design and Implementation (CMTP) http://www.cmtpnet.org/recent-articles/CMTP%20-%20CED%20Issue%20Brief.pdf