IT S ALL ABOUT EVIDENCE COVERAGE WITH EVIDENCE DEVELOPMENT IN CANADA DEVIDAS MENON, SCHOOL OF PUBLIC HEALTH, UNIVERSITY OF ALBERTA 2012 CADTH SYMPOSIUM, Ottawa, 17 April 2012
What s this all about? Experience in other jurisdictions How well have these schemes worked? What are the challenges? How could we do this in Canada?
Available health care resources should be used in a way that improves health of populations Uncertainties about clinical benefit and cost challenge decision-making on access, coverage and reimbursement System incentives should encourage innovations for improving health care Research findings and coverage decisions are closely linked (need the former in order to optimize the latter) Collecting data offers one way of acquiring evidence to reduce uncertainty and increase the probably of making a good decision
THE UNCERTAINTY GAPS 1) Clinical benefit - Safety and effectiveness over the short and long term when used in the real world 2) Value for money - cost effectiveness or utility placed on health gains and the opportunity costs of those gains 3) Adoption and diffusion - Eligible patients, anticipated uptake rates, whether the technology is a replacement for an existing technology or an addition to current practice 4) Affordability - Overall cost to the health system, taking into account any savings that may be realized
KEY QUESTIONS How do we develop a decision or policy framework that better deals with uncertainty? How do we decide when to invest in data collection to inform a definitive coverage decision? How do we decide if we have enough evidence to make a coverage decision?
ACCESS WITH EVIDENCE DEVELOPMENT (AED) Conditional or provisional funding decision that links payment to the collection and analysis of additional information needed to make a definitive funding decision Risk of exposing patients to ineffective or harmful technologies Risk of non-coverage depriving patients of access to beneficial technologies
ADVANTAGES Patients gain access to technologies that would otherwise not be funded Generates clinical evidence to inform future patients and providers Payers share responsibility for generating evidence they need to make fair decisions when market forces have failed to do so Industry gains limited reimbursement to bridge the period between regulatory approval and public funding May reduce perceived biases against promising, but less well proven technologies May help to improve population health by ensuring better use of scarce resources
WHAT DO WE KNOW ABOUT AED? Not new Many different names (e.g., field evaluations, 3-C funding, coverage with evidence development (CED), Only in Research (OIR)) Has been applied in several countries Examples from the Australia, Canada and the United States
RESULTS: INTERNATIONAL EXAMPLES OF COVERAGE AS PART OF A STUDY Country Technology Indication Uncertainty Australia Endovascular aortic aneurysm repair (EVAR) Deep brain stimulation (DBS) Positron Emission Tomography (PET) Abdominal aortic aneurysm Parkinson s disease unresponsive to pharmaceutical therapy Diagnosis and staging of various cancers Safety; long term clinical effectiveness Safety; long term clinical effectiveness; cost Safety; clinical effectiveness; costeffectiveness Netherlands Spinal Cord Stimulation (SCS) Chronic non-oncological pain Technology adoption & diffusion; cost Autologous bone marrow transplant (BMT) Advanced breast cancer Safety; clinical effectiveness Lung volume reduction surgery (LVRS) Emphysema Safety; clinical effectiveness; cost Carotid artery angioplasty+ stenting (CAS) Prevention of stroke due to carotid artery disease Safety; clinical effectiveness United States Positron Emission Tomography (PET) Suspected dementia Clinical effectiveness Positron Emission Tomography (PET) Diagnosis and staging of various cancers Clinical effectiveness Implantable Cardioverter Defibrillator Prevention of sudden cardiac death Safety; clinical effectiveness Long term home oxygen treatment Patients with severe COPD Clinical effectiveness; cost Artificial hearts Biventricular end stage heart disease Safety; clinical effectiveness
COVERAGE AS PART OF A STUDY: TYPE OF EVIDENCE DEVELOPMENT RCT Registry Case series Autologous BMT (US) EVAR (Australia) DBS (Australia) LVRS (US) Long term home oxygen treatment PET for cancer (Australia) Spinal Cord Stimulation (SCS) (Netherlands) Carotid angioplasty (US) PET for dementia (US) PET for cancer (US) Implantable Cardioverter Defibrillators Artificial hearts
COVERAGE AS PART OF A STUDY: OTHER CHARACTERISTICS Scheme management and implementation 1) University-based, academic units 2) Non-profit, publicly funded research institutes (e.g., NIH) 3) Professional societies (e.g., American College of Cardiology) Funding 1) Payer funds everything 2) Industry funds technology; payer funds service; non-industry organization funds data collection 3) Industry funds technology; payer funds service; non-industry organization funds data collection 4) Payer funds technology and service; non-industry organization funds data collection
COVERAGE AS PART OF A STUDY: OTHER CHARACTERISTICS Time investment - Several AEDS incomplete - From date announced to decision based on results: 5½ yrs (2-11 yrs) - Time invested in organizing and planning AEDs varies 1) Political factors 2) Stakeholder buy-in 3) Complexity of study design 4) Technology characteristics
EVAR AUSTRALIA August 1997: Medical Services Advisory Committee (MSAC) asked to undertake a review of EVAR February 1999: MSAC review of EVAR completed; Concluded that safety and long term outcomes of the procedure are not clear and informed consent protocol must be formulated explore data collection with the view of obtaining the data required MSAC recommended linking funding to collection of information on all procedures performed; Also recommended collecting information on any open surgical repairs (OSR) performed over same time period 1999: Australian Government Department of Health and Ageing agreed to fund national audit (registry) to be overseen by the Australian Safety and Efficacy Register of New Interventional Procedures Surgical (ASERNIP-S) November 1999 - May 2001: Information collected on 961 EVAR patients; Information also collected on some OSR patients
EVAR AUSTRALIA 2007: Results of audit reported to MSAC - 54% of patients were deemed suitable for OSR, 93% of procedures considered technical successes, 4% of patients required additional endovascular procedures, 2% of patients had EVAR converted to OSR, and 60% were still alive after 5 years of follow-up MSAC recommended that EVAR be publicly funded Minister endorsed recommendation 2009: New registry on EVAR announced
EVAR AUSTRALIA Issues that emerged: 1) Contract with ASERNIP-S not signed until most of the procedures had been performed 2) New and acceptable data set for open procedures needed 3) Difficult getting surgeons to use and complete repetitive data collection form 4) Recognized differences in baseline characteristics between OSR and EVAR patients, which would make it impossible to draw any conclusions on the effectiveness of EVAR, relative to open repair
COVERAGE AS PART OF A STUDY HOW WELL HAS IT WORKED? Definitive coverage decision consistent with AED results Technology EVAR DBS PET - cancer SCS Autologous BMT LVRS CAS PET - dementia PET - cancer ICD Home oxygen Artificial hearts Yes No Unable to assess
LESSONS LEARNED 1. AED can facilitate access to technologies that would otherwise not be funded 2. AED schemes could generate evidence needed to make fair decisions when market forces/commercial interests have failed to collect such evidence 3. AED schemes offer manufacturers limited reimbursement to bridge the period between regulatory approval and introduction into routine, publicly funded practice 4. Implementation of AED as a policy option can be scientifically and politically complex
5. Without careful consideration of study design, it may not answer the questions it was set up to address 6. Considerable upfront investment of time and resources may be needed (managing conflicts of interest, determining outcome measures that all stakeholder groups are agreeable to, stopping points, etc.) 7. Costs can be significant, so there is a need to consider whether the costs of investing in an AED are offset by savings anticipated as a result of collecting the evidence needed to fill the uncertainty
SO WHERE DOES THAT LEAVE US? 1. AED might be a suitable option when there is a health technology which a payer is considering funding, but a review shows that there are significant uncertainties for a policy decision to be made. 2. AED to date has not been universally successful, mainly because there has not been a systematic and considered approach to its development and implementation.
3. In Ontario, there is a field evaluation program which has reviewed a number of new and existing technologies. 4. In Quebec, there appear to have been hospital-based studies in which health technologies have been provided with a condition that additional evidence be collected. 5. In Alberta, the government has commissioned the development of a provincial AED framework
The 2004 Health Technology Strategy 1.0 recommendation on Field Evaluations and Infostructure: 1.That a coordinated Field Evaluation system be established to collect primary research data on new and experimental technologies where data needed for decision-making is insufficient. 2.That Canada's health information resources and infostructure be developed and leveraged to guide innovation, health quality, and diffusion of health technology. 3.That common health quality indicators be used to track the performance of Field Evaluations wherever possible.