Bending the Cost Curve Through Comparative Effectiveness Research. ASCO 2012 Sean Tunis MD, MSc June 2, 2012

Transcription

1 Bending the Cost Curve Through Comparative Effectiveness Research ASCO 2012 Sean Tunis MD, MSc June 2, 2012

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3 Evidence Summary: Radiation Therapy for Comparisons Clinically Localized Prostate Cancer Disease specific survival Freedom from biochemical failure GU/GI toxicity RT vs NT insufficient insufficient insufficient SBRT vs EBRT insufficient insufficient insufficient SBRT vs HDBRT insufficient insufficient insufficient SBRT vs LDBRT insufficient insufficient insufficient EBRT vs HDBRT insufficient insufficient insufficient EBRT vs LDBRT insufficient insufficient insufficient HDBRT vs LDBRT insufficient insufficient insufficient Combined mod. insufficient insufficient insufficient Intra SBRT insufficient insufficient insufficient Intra EBRT insufficient moderate moderate Intra LDBRT insufficient insufficient insufficient Source: Tufts Evidence-based Practice Center: Draft AHRQ Technical Assessment, March 25, 2010

4 Off-label Uses of Oncology Drugs Draft systematic review of off-label use of oncology drugs Duke EPC, Amy Abernathy 428 pages, several thousand trials Because of the paucity of high quality evidence, the data available though voluminous may have little meaning or value for informing clinical practice PROs missing or not comparable 4

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6 High Hopes for CER Better information about the costs and benefits of different treatment options, combined with new incentive structures reflecting the information.is essential to putting the country on a sounder long-term fiscal path. Peter Orszag, CBO (later OMB) Congressional Testimony, June

7 Overview for Center for Medical Technology Policy PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE Baltimore, MD August 24, 2011 Joe Selby, MD MPH Executive Director

8 The Evidence Paradox 18,000+ RCTs published each year Tens of thousands of other clinical studies Systematic reviews intended to inform clinical and health policy decisions routinely conclude that evidence is inadequate

9 The CER Hypothesis Gaps in evidence will be reduced with greater engagement of end users (decision makers = patients, clinicians, payers) in Identifying most important uncertainties Designing study protocols Useful CER studies must incorporate views of patients, clinicians, payers in study design 9

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11 PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE The Patient-Centered Outcomes Research Institute helps people make informed health care decisions and improves health care 1 delivery and outcomes by producing and promoting high integrity, evidence-based information that comes from research guided by patients, caregivers and the broader health care community

12 Patient Perspectives: Psoriasis Regulatory studies report extent and severity of total body surface area affected by plaques FDA does not allow any QoL measures on label Patients state that face and joint involvement had biggest impact on QoL More pragmatic design would include measure of face/joint severity as secondary endpoint and high priority to develop and validate a measure for severity of face/joint involvement 12

13 Clinician Perspective Dementia ACP guideline reviewed 5 drugs approved by FDA based on cognitive function (ADAS-cog) Weak evidence for clinical recommendations No convincing comparative studies Outcomes used in trials not used in routine clinical practice.and not clinically important Follow-up too short: generally less than 1 year. More pragmatic design would include active comparator, include cognitive measures used in practice, and longer follow up 13

14 Payer Perspective - Artificial Disc Medicare denied coverage of cervical artificial discs based on FDA-approved PMA trial Trial limited to patients age 18 to 60 Non-inferiority comparison to spinal fusion was not most clinically relevant alternative SF-36 and radiographic changes have no clinical meaning More pragmatic design would enroll older patients, compare to conservative care, and measure patients activity level 14

15 High Hopes for CER Better information about the costs and benefits of different treatment options, combined with new incentive structures reflecting the information.is essential to putting the country on a sounder long-term fiscal path. Peter Orszag, CBO (later OMB) Congressional Testimony, June

16 Implications for Clinical Practice The Learning Healthcare System Every clinical encounter generates knowledge Depends on accurate and complete data entry Bundled payments and quality-adjusted pay Clinical income linked to outcomes and efficiency Risks, benefits and costs of alternative diagnostic and therapeutic options are essential resource Increased patient-cost sharing Clinical decisions must be cost sensitive

17 High Hopes for CER Better information about the costs and benefits of different treatment options, combined with new incentive structures reflecting the information.is essential to putting the country on a sounder long-term fiscal path. Peter Orszag, CBO (later OMB) Congressional Testimony, June

18 Who is to Blame? Health insurance profits and salaries? Overpriced drugs and devices? Greedy malpractice lawyers? Incompetent federal bureaucrats? Demanding patients? (Physicians?) My theory, a the tragedy of the commons

19 Contact Info x120 (W) (M)

20 Extra Slides

21 21 CANCERGEN Structure

22 RxPonder Trial Node-positive (1-3 nodes), HR-positive, and HER2-negative breast cancer (N= 600) RS already Available (N= 8,800) Patients consent to study-sponsored RS testing, discussion of potential trials, tumor tissue submission and linkage to cancer registry data (N= 3,800 Discuss alternative trials for high risk patients RS < 25? RS > 25 RS < 25 N= 5,600 Physician and patients discuss randomization knowing the RS Refuse Accept N= 4,000 Randomization stratified by 1. RS 0-13 vs Menopausal status 3. Sentinel LNB only vs. complete LND N= 2,000 Chemotherapy; appropriate endocrine therapy N= 2,000 No Chemotherapy; appropriate endocrine therapy 22 N= 1,600 Record chosen therapy and followed for vital status using cancer registry

23 CER Defining Characteristics (IOM) Objective of directly informing clinical or health policy decision Compares at least 2 alternative, each with potential to be best practice Results at population and subgroup level Measures outcomes important to patients Methods and data sources appropriate for the decision of interest Conducted in real world settings 23

24 Methods Emphasized in CER Heavy focus on extracting more knowledge from existing studies and data Meta-analysis / systematic reviews Modeling Analysis of EHR and administrative data More efficient, informative prospective studies Registries Adaptive designs Attention to heterogeneity / subgroup effects Pragmatic clinical trials