Update from IMI PREFER: Patient preferences in benefitrisk assessments during the medical product lifecycle

Transcription

1 1 1 Update from IMI PREFER: Patient preferences in benefitrisk assessments during the medical product lifecycle Presenter: Chiara Whichello on behalf of the PREFER consortium June 5, 2018 The statements made in this presentation are those of the presenter and co-authors and not necessarily those of their employers or institutions, the view of IMI, the EU, or EFPIA.

2 2 2 Overview 1. What is PREFER and its goal? 2. What is patient preference information (PPI)? 3. How will PREFER achieve its goal? 4. Results from Work Package 2: Assessing patient preference elicitation and exploration methods

3 3 3 About the PREFER project The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) is a five year project that has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA.

4 4 4 PREFER 5-Year Public-Private Partnership ( ) 33 partners, 8 countries 10 Academic research institutions 4 Patient representatives 1 Health Technology Assessment body 2 SMEs (small and medium sized enterprises) 16 Pharmaceutical companies Additional Stakeholders Advisory Groups: Patients, HTA/Payers, Regulators Scientific Advisory Board / Extended Network

5 5 5 Goal of IMI PREFER Project To strengthen patient-centric decision making throughout the medical product life cycle by developing expert and evidence-based recommendations on how patient preferences should be assessed and inform decision making.

6 6 Three Types of Patient Preference Information Type Attributes Relative Importance Tradeoffs What it Measures What Matters How much it matters (relative to something else) What tradeoffs patients are willing to make between benefits, harms, and other aspects (e.g.. convenience of dosing) Adapted from RTI-HS

7 7 7 PREFER Stepwise Work Packages Work Package 2 Stakeholder Needs / Methods Review Determining stakeholder needs for patient preference use in MPLC Identifying and appraising methods for clinical case and simulation studies Methods: Literature reviews, Interviews, focus groups, (stakeholders and health preference experts) Work Package 3 Clinical case and simulation studies Designed to address issues / questions raised in needs assessment Academia-led studies Industry-led studies Simulations Work Package 4 Recommendations Document(s) Preference assessment and application to decisions throughout the lifecycle For health authorities, industry and payers/hta Work Package 1: Overall project management, including communication / dissemination

8 8 8 2 Results from WP2: Assessing patient preference elicitation and exploration methods

9 9 9 T2.4: Preference exploration methods

10 10 10 T2.4: Preference elicitation methods

11 11 11 T2.6: Q-Methodology Results Criteria to be included in AHP A typical survey can be conducted at relatively low costs Data can be collected during quick sessions with participants Low frequency of sessions required by patients Relatively quick delivery of preparation, data collection, and analysis A large number of attributes can be explored Suitable to study preferences in a small sample size A low cognitive load on patients Does not need an education tool or preparatory instructions in order to enhance participant comprehension Publically acknowledged by your organisation s guidelines as an acceptable method to study preferences New attributes can be added without making prior results invalid Can be used to collect data from more than one participant in a single session The analysis can calculate risk attitudes, like risk tolerance, and calculate how value functions bend due to the presence of uncertainty in the participant Explores the reasons behind a preference in detail Can estimate weights for attributes Estimates trade-offs that patients are willing to make among attributes Can quantify heterogeneity in preferences Establishes internal validity Establishes external validity Early development A (mechanism known) Early development B (mechanism not known) Late phase III Postmarketing

12 12 12 T2.6: Analytic Hierarchy Process (AHP) Results

13 13 13 T2.7: Identifying candidate methods Criteria (from Q- methodology) Identified in WP 2.6 Identified in WP 2.7 MPLC Stages AHP Results Method Assessment Weights Method Performance Publication Frequency Theory Concerns Identification of 12 candidate exploration and elicitation methodologies (suitable/likely to meet most decision makers needs) + Identification of 11 promising methods (identified potential theoretical or publication frequency issues of which decision-makers must be aware)

14 14 14 T2.7: Identifying candidate methods eg. Appraising threshold techniques * Informed exclusively by literature, and not expert interviews Red indicates that these methods would likely not suit most decision makers needs during this stage + + means 1.51, + means , - means , - - means 0

15 15 15 T2.7: Twelve most promising candidate PP methods Exploration methods Elicitation methods Group methods Individual methods Discrete choicebased Threshold techniques Focus groups Semistructured interviews In-depth interviews Discrete choice experiments / best-worst scaling 3 (Probabilistic) threshold technique Time trade-off Standard gamble Ratingrelated Rankingrelated Swing weighting Visual analogue scale Best-worst scaling 1 Best-worst scaling 2 Analytical hierarchy process

16 16 16 T2.7: Eleven potential candidate PP methods Exploration methods Elicitation methods Group methods Individual methods Discrete choice-based Threshold techniques Nominal group technique Public meetings Dyadic interviews Adaptive conjoint analysis Test trade-off Starting known efficacy Ratingrelated Rankingrelated Outcome prioritization tool Constant sum scaling Control preferences scale Q- methodology Qualitative discriminant process

17 17 17 Clinical Case and Simulation Studies Methodological questions identified during WP2 will be investigated using patient preference studies Studies to be conducted in at least three disease areas where patients and clinical research partners already provide expertise: Cancer Rheumatoid arthritis Neuromuscular disorders Partners from the pharmaceutical industry will provide additional patient preference studies to cover disease areas from the companies portfolio.

18 18 18 Scope of Recommendations Industry internal decisions Product strategy (e.g. TPP), stage gate decisions, B-R assessments Clinical trial design Endpoint selection, effect size, Regulatory benefit-risk assessments, Industry preparation, regulatory assessments, post-approval benefit-risk Health technology expert assessments Recommendations for individual products or classes of products Payer reimbursement decisions, Reimbursement for a specific product or classes of products Public agency decisions (e.g. public private partnerships, regulatory agencies)

19 19 19 In summary, PREFER Will establish recommendations for Industry, Regulatory Authorities, and HTA bodies on how and when to perform patient preference studies include patient preferences in decision-making Includes a diverse consortium that involves many stakeholder groups (including patients): both as partners and advisors Link to PREFER website:

20 20 20 Acknowledgements PREFER consortium PREFER partners Stakeholder advisory groups Scientific advisory board Extended stakeholders Contact information: