Patient-Centred Decision Making with MCDA: Should We Be Trying to Quantify the Patient Voice for Use in HTA? ISPOR 2016, Vienna, 31 October 2016 Prof. Dr. Peter Kolominsky-Rabas, M.D.; M.B.A. Interdisciplinary Center for Health Technology Assessment (HTA) and Public Health Friedrich-Alexander-University Erlangen-Nürnberg Executive Board, National Cluster of Excellence Medical Technologies - Medical Valley EMN "Whenever a theory appears to you as the only possible one, take this as a sign that you have neither understood the theory nor the problem which it was intended to solve. The Logic of Scientific Discovery, 1959 2 1
Sir Karl Popper born 1902 in Vienna died 1994 in London Austrian and British philosopher Professor at the London School of Economics He is considered one of the most influential philosophers of science of the 20th century 3 Key Questions How are patients involved in the HTA- Sytems? To which extent is MCDA today part of the current HTA processes? 4 2
Systematic Review from 2016 Abelson J et al. Public and Patient Involvement in HTA. International Journal of Technology Assessment in Health Care, 32:4 (2016) 5 Systematic Review - Results Broader view Public AND Patient involvement in HTA 53 HTA agencies, 34 countries Research questions - How are HTA oranizations involving public and patients? - How are they doing this in terms of approaches, specific mechanism - With are the results? What are the barriers or facilitators? Public and patient involvement approaches ( n = 13) Goals and rationales for public and patient involvement (n = 5) Evidence of public and patient involvement effectiveness ( n = 2) Public and patient involvement feasibility ( n = 0) Abelson J et al. Public and Patient Involvement in HTA. International Journal of Technology Assessment in Health Care, 32:4 (2016) 6 3
Patient Focused Decision-Making Canada Patient Involvement CADTH s first Call for Patient Input was issued in May 2010 CADTH Common Drug Reviews (CDR) CADTH s pan-canadian Oncology Drug Review (pcodr) British Columbia Your Voice Program Ontario Patient Evidence Submission 7 England & Wales Participation at each strand of NICE s work - Public Board Meetings - Scoping Process - Advisory Committees (f.e. Technology Appraisal Committee) NICE s Public Involvement Programme (PIP) - developing and supporting opportunities to involve patients, carers, service users and the public in NICE s work June 2016 - Myeloma UK has awarded a research grant to NICE - to fund a two year exploratory study - Myeloma UK is a registered charity since 1997 Project Methods for patient-centred decision-making on the availability of new treatments (Lead: Prof Sarah Garner, NICE) https://www.nice.org.uk/media/default/about/nice-communities/public-involvement/public-involvement-programme/pip-process-guide-apr-2015.pdf 8 4
Belgium Belgian Healthcare Knowledge Centre KCE REPORT 195C Models for Citizen and Patient Involvement in Health Care Policy, Part I: Exploration of their feasibility and acceptability Excellent methodological framework based on concepts International Association for Public Participation (IAP2) Different levels of involvement according to different decisions - for priority setting in the allocation of scarce health care resources: citizen representation - for reimbursement decisions of new treatments: patient representation https://kce.fgov.be/sites/default/files/page_documents/kce_195c_citizen_and_patient_participation.pdf http://c.ymcdn.com/sites/www.iap2.org/resource/resmgr/imported/iap2%20spectrum_vertical.pdf 9 Germany G-BA Coordination Committee of Patients Representatives - in total 220 patient representatives involved in G-BA subcommittees Institute for Quality and Efficiency in Health Care (IQWiG) - Written comments to benefit assessments (direct) - Hearings (PRO) (via Coordination Committee of Patients Representatives) - Patient s perspective in benefit assessements (via questionnaire) 10 5
MCDA in Germany s HTA 2010 IQWiG initiated a study on Analytic Hierarchy Process (AHP) Integrating patients views into HTA Topic Diagnosis of major depression Rating of preferences with respect to the importance of different endpoints of antidepressant treatment Recommendations: AHP useful in HTA to give a quantitative dimension to patients preferences for treatment endpoints Danner M et al. Integrating patients views into health technology assessment: Analytic hierarchy process (AHP) as a method to elicit patient preferences. Int J Tech Assess Health Care, 27:4 (2011), 369 375. Hummel MJ et al. Using the analytic hierarchy process to elicit patient preferences: prioritizing multiple outcome measures of antidepressant drug treatment. Patient. 2012;5(4):225-3 11 Patient Focused Decision-Making USA Patient Preference Initiative While patient representatives participate in many of the FDA's advisory committees, [ ] the FDA has never had a committee that was wholly focused on patients. Center for Biologics Evaluation and Research (CDER) - working on integrating qualitative data into review processes Center for Devices and Radiological Health (CDRH) - working on quantitative approach - Pilot project on medical devices (weight-loss device) Ref: Surg Endosc (2015) 29:2984 2993 Final Guidance in effect October 23, 2016 FDA encourages medical device manufacturers to voluntarily include in their premarket submissions information on Patient Preference Information (PPI) http://www.fda.gov/aboutfda/centersoffices/officeofmedicalproductsandtobacco/cdrh/cdrhpatientengagement/ucm462830.htm Ho et al. Incorporating patient-preference evidence into regulatory decision making. Surg Endosc (2015) 29:2984 2993 http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm446680.pdf 12 6
Conclusion Patients perspectives Considering patients perspectives is increasingly essential as healthcare systems move toward more patient-centred approach This trend is part of broader political context of consumer protection However the majority of HTA agencies lack systematic evaluation on - structures - impact - facilliators and barriers of patient involvement 13 Conclusion MCDA in HTA Technical MCDA techniques can support a broad variety of decision problems in HTA Political - transparency of the preference-elicitation process - involvement of ALL stakeholders into the value-assessment process - increasing the legitimacy of decision process Lack of familiarity with the deversity of MCDA techniques No sufficient methodological guidance how to - design - conduct and - implement MCDA a spart of HTA 14 7