The present study was designed for obtaining Canadian pricing and reimbursement stakeholders’ opinion on a list of proposed recommendations for updating the 2007 Patented Medicine Prices Review Board (PMPRB) Budget Impact Analysis guidelines. Methods: Stakeholders from different perspectives including policymakers (public and private payers) and industry experts/consultants were invited to participate in the study (private payer and industry perspectives were not included in the PMPRB 2007 BIA guidelines). Using a mixed methods approach, an interview guide and a written survey were developed based on discordance between the PMPRB 2007 BIA guidelines recommendations and Canadian provincial, and other national or transnational BIA guidelines. A thematic content analysis was applied for the qualitative data analysis. Results: We conducted nine interviews with policymakers and twenty-seven surveys with industry experts/consultants. Most interviewees were positive about the usefulness of BIA in disinvestment decisions and believed that reviewing cost-effectiveness analysis (CEA) and BIA together, at the same time, could be particularly informative for setting value-based prices. Fifty-six percent of the proposed recommendations were approved (e.g., the use of post-market real-world data for assessing the reliability of BIAs first-year forecasts), whereas, 30% were not supported by stakeholders (e.g., indirect costs). Some recommendations will need further input from public and private payers before being included in a revised version of the PMPRB BIA guidelines (e.g., inclusion of cost offsets). Conclusions: In the present study, Canadian payers and manufacturers’ views on the BIA recommendations, obtained through qualitative and quantitative methods, provide additional insight to help define BIA guidelines from a Canadian perspective. This information may also be of value for updating or creating BIA guidelines worldwide.

1. Comparative analysis of stakeholder views on proposals for updating the Patented Medicine Prices Review
Board (PMPRB) Budget Impact Analysis guidelines: public or private payer versus industry perspectives
Naghmeh Foroutan1,2, Jean-Eric Tarride1, 2, 3, Feng Xie1, 3, 4, Bismah Jameel1, Fergal Mills5, Mitchell Levine1,2, 3
1Department of Health Research Methods, Evidence and Impact, McMaster University, Hamilton, 2Programs for Assessment of Technology in Health, Research Institute of St. Joe’s Hamilton, Hamilton, ON; 3Centre for
Health Economics and Policy Analysis (CHEPA), McMaster University, ON, Canada; 4Program for Health Economics and Outcome Measures (PHENOM), Hamilton, ON, Canada; 5Innomar Consulting, Oakville, ON, Canada
BAC KG RO U N D
 We launched a project for updating the PMPRB BIA guidelines in early 2017.
 The project was consisted of two literature reviews of BIA guidelines (the
interim results were reported in CAPT 2017) and a stakeholder analysis.
 Objectives: The present study is a comparative assessment of stakeholders’
feedback on a list of proposed recommendations for updating the PMPRB
BIA guidelines.
R E S U LT S
Survey: Policymakers vs manufacturers
R E S U LT S ( C O N T ’ D )
Proposal
(Penultimate version of the guidelines)
Stakeholder analysis (A mixed method study)
Face-to-face interviews Online survey
Systematic literature reviews
National and transnational guidelines Canadian (F/P/T) guidelines
M E T H O D S
Note: Gold refers to” policy-makers” and “Blue bars” represent Manufactures/consultants
 We found support for inclusion of 56% (n=21) of Discordance List
 11 recommendations (30%) were not supported by stakeholders
 Inconclusive: 14% (n=5)
-100% -50% 0% 50% 100%
Opportunity cost by the manufacturers
Off-label indications are recommended
Risk sharing agreement are included
Cost transfer from other jurisdictions is allowed
A BIA should use probabilistic sensitivity analysis
Patient’s perspective is included
A longer introduction phase for early BIAs
Subpopulation analysis should be conducted
Outcomes should be presented in natural units
The 3-year time horizon is recommended.
Outcomes be presented for different payers
A schematic representation of the uncertainty
Open or dynamic populations should be used
LCA price for relevant drug comparators
Outcomes should be presented in monetary units
The impact of uncertainty should be reported
A BIA should use deterministic sensitivity analysis
Inflation should NOT be included
Discounting should NOT be included in the BIA
Aggregated and disaggregated results
Gross and net impact on the budget are reported
Data from manufacturer are allowed
A BIA should use scenario analysis
-100% -50% 0% 50% 100%
An appropriate rate of tax (e.g. HST) should be applied
Training or introduction cost should be included in the BIA
Transportation, productivity and caregiver related costs are included
Data from other jurisdictions are reliable
The total and incremental impact on the budget should be included
Expert opinions are reliable
Extrapolating data from similar drug experience are reliable
-100% -50% 0% 50% 100%
Patient adherence should be included
Cost offsets should be included
Rate of mortality and disease progression
should be reported
A cap or threshold for budget impact is
recommended
A reassessment process of BIAs in a future
real-world is recommended
Including the treatment switch in BIAs is
recommended
A BIA guideline should provide a list of
acceptable databases
Survey: Policymakers Survey: Manufacturers
R E S U LT S
Survey
Interview
Through a thematic content analysis, we identified four major themes as
following:
 BIA is used by both public and private payers for drug reimbursement
decisions and price adjustments.
 BIA could be useful and one of the several aspects for disinvestment
decisions.
 Linking incremental cost-effectiveness ratio (ICER) and BIA (affordability)
using any formula is not recommended.
 Additional recommendations for improving the Canadian BIA guidelines. F U N D I N G
Mitacs Accelerate; Patented Medicine Prices Review Board (PMPRB)