Evidence-Based Medicine – Key Trends and Lessons Learned from CMS

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

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

3. 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

4. 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.”

5. 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

6. 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

7. Higher STRENGTH OF EVIDENCE Lower 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)

8. 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.”

9. 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

10. 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

11. 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

12. 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.

13. 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

14. 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

15. 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

16. 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

17. 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.

18. 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

19. 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

20. 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

21. 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

22. 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

23. 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

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