Evidence-Based Medicine – Key Trends and Lessons Learned from CMS :Evidence-Based Medicine – Key Trends and Lessons Learned from CMS Mass MEDIC Brian Carey April 20, 2006


Overview :Overview Medicare coverage basics When is evidence adequate for coverage and reimbursement Coverage with evidence development Lessons learned from case studies


CMS Quality Initiative :CMS Quality Initiative Administrator Mark McClellan wants to transform CMS from a payer into a public health agency Promising new technologies may have insufficient evidence for informing clinical practice Premarket randomized clinical trials may be too limited to represent outcomes in real practice Health system seeks better outcomes at lowest possible costs Health IT will allow for great advances in data management, utility for research


Statutory Basis for Coverage :Statutory Basis for Coverage Sect. 1862 (a)(1)(A), Title 18, SSA “Notwithstanding any other provisions of law . . .no payment may be made…for items or services . . [which] are not reasonable and necessary for the diagnosis or treatment of illness or injury.”


Reasonable and Necessary :Reasonable and Necessary CMS has been developing evidence based framework for coverage decisions “case law” derived from NCDs since 2000 Safe and effective FDA Adequate evidence to conclude that the item or service improves net health outcomes generalizable to the Medicare population as good or better than current covered alternatives


CMS Follows General Principles of EBM :CMS Follows General Principles of EBM Published and non-published studies, expert opinion, technology assessments, professional societies, recommendations from the Medicare Coverage Advisory Committee (MCAC) Key areas of focus include: Methodological considerations, including study design, implementation, and analysis Relevance of chosen outcomes; preference for those experienced by the patient Generalizable to the Medicare population Qualitative assessment of net risks and benefits based on individual studies


Ladder of Evidential Strength :Ladder of Evidential Strength ________Meta-analysis of individual patient data ___________ Large, multi-center RCTs ______________ Meta-analysis of grouped data ______________ Smaller, single site RCTs ______________ Prospective cohort studies ____________ retrospective cohort ____________ Poorly controlled studies _______________Uncontrolled studies (case-series or reports)


CMS Does Not Consider Cost Effectiveness :CMS Does Not Consider Cost Effectiveness CMS released Guidance document on April 11, 2006 on NCD Process “Cost effectiveness is not a factor CMS considers in making NCDs. In other words, the cost of a particular technology is not relevant in the determination of whether the technology improves health outcomes or should be covered for the Medicare population through an NCD.”


Frequently Unanswered Questions :Frequently Unanswered Questions Adverse events in broad use Off-label uses or new combinations of approved uses Risks and benefits in subgroups Outcomes for patient and provider types excluded from trials Comparative effectiveness Outcomes not measured in trials Clinical utility of diagnostic tests Surgery, some devices and other technologies with limited regulatory review


Practical Clinical Trials :Practical Clinical Trials CMS encourages use of data from Practical Clinical Trials (JAMA article by Dr. Tunis) Greater emphasis on health outcomes actually experienced by patients, such as quality of life, functional status, duration of disability, morbidity and mortality less emphasis on outcomes that patients do not directly experience, such as changes in laboratory values, radiographic response, sensitivity/specificity, physiologic parameters and other intermediate or surrogate outcomes


Case Study – Coverage of MTWA :Case Study – Coverage of MTWA Microvolt T-Wave Alternans is a non-diagnostic test for stratification of patients who may be at risk of sudden cardiac death MTWA had limited Medicare coverage that varied by local carrier Cambridge Heart requested NCD in 2005 and met with CMS numerous times


MTWA Coverage :MTWA Coverage CMS reviewed peer reviewed article and existing technology assessment Out of 1028 citations in support of MTWA CMS focused on 12 peer reviewed articles CMS performed systematic review of literature Final NCD issued in March, 2006 expanding coverage of MTWA using spectral analytic method.


Observations :Observations BCBS TA had concluded that MTWA did not meet criteria for coverage. CMS focused on Medicare eligible population CMS found there was not sufficient evidence to cover modified moving average (MMA) technique for determining MTWA Aetna followed CMS’s policy


CMS Coverage with Evidence Development (CED) :CMS Coverage with Evidence Development (CED) Support creation of better evidence Links Medicare coverage with requirement for prospective data collection Builds on existing Evidence Based Medicine coverage framework


CMS Evidence Objectives :CMS Evidence Objectives Provide more and better information for health care decision makers Expand capacity of clinical research enterprise to produce information oriented to decision makers Support innovation while addressing frequently unanswered questions Move toward system in which care delivery and evaluation are more integrated


Use of “CED” by CMS :Use of “CED” by CMS Lung volume reduction surgery PET for suspected dementia ICD for primary prevention of SCD PET for non-covered oncology uses


Case Study – National Oncologic PET Registry :Case Study – National Oncologic PET Registry What is a CMS approved “Coverage with Evidence Development” Program In 2000, the Centers for Medicare and Medicaid Services (CMS) expanded its coverage of positron emission tomography (PET) with F-18 fluorodeoxyglucose (FDG) to a wide variety of indications for several common cancers – but not all cancers. In November 2004, CMS proposed expanding PET coverage to most other cancers, if providers collect relevant data in a CMS-approved clinical registry.


National Oncologic PET Registry (Cont.) :National Oncologic PET Registry (Cont.) NOPR is rune by American College of Radiology and Academy of Molecular Imaging All PET facilities can participate (for a fee) Requires Pre-PET and Post-PET Case report forms completed timely Will assess change in intended management


Lessons Learned :Lessons Learned CED policy can result in significant expansion Registries are very complicated; numerous logistic issues such as IRB, informed consent and Privacy Act CMS likely to issue new guidance on CED


Section 1013 – Clinical Effectiveness Studies :Section 1013 – Clinical Effectiveness Studies MMA included specific provision on the comparative clinical effectiveness and appropriateness of health care items HHS developed 10 priority areas Agency for Healthcare Research and Quality (AHRQ) is conducting studies through EPCs Systematic reviews and syntheses of the scientific literature


Section 1013 AHRQ Studies :Section 1013 AHRQ Studies Management strategies for gastroesophageal reflux disease Benefits and safety of analgesics for osteoarthritis New diagnostic technologies for evaluation of abnormal breast cancer screening Epoetin and Darbepoetin for managing anemia in patients undergoing cancer treatment Off-label use of atypical anti-psychotic medications Renal artery stenting compared to aggressive anti-hypertensive medical therapy for mild renal artery stenosis Therapies for localized prostate cancer Oral medications for diabetes management Medications for depression management Drug therapies and behavioral interventions for osteoporosis and osteopenia


EBM at State Level :EBM at State Level States have been further ahead of CMS Center for Evidence-based Policy at Oregon Health and Science University Performed systematic reviews for 10 to 15 states on drug effectiveness Starting to look at devices such as imaging and diagnostic tests


Conclusion – Recent Trends :Conclusion – Recent Trends Increased transparency of coverage process Focus on evidence and data generation beyond what has traditionally been required for FDA approval Initiative to increase data collection to use for future coverage decisions or refinements Increased collaboration between CMS and other government agencies such as FDA and NCI


Contact info :Contact info Brian P. Carey Foley Hoag LLP bcarey@foleyhoag.com (617) 832-1712 (202) 261-7398