Open Collections

UBC Faculty Research and Publications

Engaging the Canadian public on reimbursement decision-making for drugs for rare diseases: a national… Polisena, Julie; Burgess, Michael; Mitton, Craig; Lynd, Larry D May 26, 2017

Your browser doesn't seem to have a PDF viewer, please download the PDF to view this item.

Item Metadata


52383-12913_2017_Article_2310.pdf [ 386.57kB ]
JSON: 52383-1.0361970.json
JSON-LD: 52383-1.0361970-ld.json
RDF/XML (Pretty): 52383-1.0361970-rdf.xml
RDF/JSON: 52383-1.0361970-rdf.json
Turtle: 52383-1.0361970-turtle.txt
N-Triples: 52383-1.0361970-rdf-ntriples.txt
Original Record: 52383-1.0361970-source.json
Full Text

Full Text

RESEARCH ARTICLE Open AccessEngaging the Canadian public onreimbursement decision-making for drugsfor rare diseases: a national online surveyJulie Polisena1*, Michael Burgess2, Craig Mitton3,4 and Larry D. Lynd5,6AbstractBackground: Funding of drugs for rare diseases (DRDs) requires decisions that balance fairness for all individualswithin the healthcare system with compassion for affected individuals. Our study objective was to conduct anational online survey to determine the Canadian public’s perspective, including regional variations, associated withDRD decision-making.Methods: The survey collected responses from 1631 Canadians. Respondents were asked to rank at least three andup to five DRD decision-making priorities, out of a total of eight priorities presented. They were also asked tocompare and rate their agreement level on a 5-point Likert scale with four funding scenarios described. Thefrequency of each priority, independent of where it was ranked in relation to the other priorities, was calculated.Regression analyses were conducted to measure the association between respondents’ demographics and selectedpriorities with their agreement level for each funding scenario.Results: Among the survey respondents, Improved Quality of Life and Effective Health Care were most frequentlyselected as top priorities. Also, 79.2% of respondents agreed with equal access to DRDs across Canada, and 73.0%agreed with DRD funding if additional expenses are justified in the DRD’s cost-effectiveness. Approximately halfagreed to pay for DRDs independent of their effectiveness. There were no geographic differences in priorities.Selecting Effective Health Care in the top priorities was positively associated with both prioritizing other programsover programs for rare diseases and DRD funding only if deemed as cost-effective. Respondents, who selectedNational Access as one of the top priorities, were less likely to agree to fund DRDs only if deemed as cost-effectiveand were more likely to agree with the scenario to provide national access to DRDs.Conclusions: The survey results suggest the level of public support for funding decisions and programs thatincorporate assessment of the effectiveness of drugs for improving quality of life, and to promote similar accessacross Canada. The responses anticipate public responses to different policy scenarios and the priorities thatunderlie them. Decision-makers may find it useful to consider whether and how to incorporate these results intopolicy decisions and their justification to citizens and patients.Keywords: Drugs, Reimbursement, Rare diseases, Survey, Public engagement* Correspondence: juliep@cadth.ca1Canadian Agency for Drugs and Technologies in Health, 600-865 CarlingAve, Ottawa K1S 5S8, ON, CanadaFull list of author information is available at the end of the article© The Author(s). 2017 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0International License (, which permits unrestricted use, distribution, andreproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link tothe Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver( applies to the data made available in this article, unless otherwise stated.Polisena et al. BMC Health Services Research  (2017) 17:372 DOI 10.1186/s12913-017-2310-4BackgroundFunding of expensive drugs for rare diseases (DRDs) re-quires decisions that balance fairness for all individualswithin the healthcare system with compassion for individ-uals with rare diseases. As there is no universal definitionof a rare disease, we applied the average prevalence usedin Canada (38 cases per 100,000 people; minimum= 1 andmaximum= 50 across jurisdictions) [1]. In Canada’s pub-licly funded healthcare system, the multiple processes toassess reimbursement of DRDs are generally comparable,but access to specific drugs differs across jurisdictions.The variation can, in part, be attributed to the prevalenceof the rare disease in specific provinces or territories [2].For instance, Alberta defines a rare disease as a geneticlysosomal storage disorder that is prevalent in fewer than1 per 50,000 Canadians, while Ontario and Saskatchewandefine it as a disease with an incidence rate of fewer than1 in 150,000 live births or new diagnoses annually, andBritish Columbia consider a rare disease with a prevalenceas fewer than 1.7 per 100,000 [2, 3]. The availability ofhigh quality evidence is a challenge due to the low preva-lence of these diseases, and manufacturers typically assignhigh prices to recover research and production relatedcosts [4].Even though an increasing number of countries haveimplemented regulations for the development and marketapproval for DRDs, Canada does not have national pol-icies or a pan-Canadian legislative framework in place or areimbursement review process for DRDs. They do, how-ever, exist in five provinces: British Columbia, Alberta,Saskatchewan, Ontario, and New Brunswick [3]. Unlikeother provinces with DRD programs, a coverage decisionwill impact access to the drug for all patients with the raredisease in Ontario, and not an individual patient. Acrossthe programs, a panel or committee that includes at leastone disease expert or specialist will review an applicationto understand the disease severity and alternative treat-ments, assess the potential effectiveness, or evaluate thebudget and cost impact. Submissions by patient groupscan also be reviewed. Recommendations will usually in-clude options for coverage with conditions or do notprovide coverage. Coverage with conditions usually im-plies continued monitoring for clinical outcomes or thepatient’s response to treatment [3]. Similar to Canada,most 20 OECD countries with a socially funded healthcare system or universal health care do not have a nationalprogram for funding DRDs, except for the UnitedKingdom. Instead, many apply a “safety net” program oruse modified decision criteria when their common drugreview processes are insufficient for funding decisions forDRDs [5].In 2012, Health Canada presented a draft frameworkfor orphan drug regulatory approval, engaged in a pan-Canadian discussion, and posted it online for comments.The framework aims to develop and implement a com-prehensive approach meant to support industry in theirsubmission for market access approval without com-promising patient safety. It is based on an integratedapproach that incorporates information collected frompatients, health care professionals, researchers, payers,and international regulators to enhance the quality ofknowledge for decision making and reduce uncertainty.Similar to DRD legislation established by other inter-national regulatory agencies, key components of theframework involve orphan drug designation, regulatoryand expert advice and patient representation fromHealth Canada, and increased transparency in the mar-ket authorization process [6]. At the time of our study,the framework had not been finalized.Paulden et al. conducted a scoping review published in2015 to identify the medical and grey literature tounderstand the societal values associated with DRD re-imbursement, what potential role they can play, and out-lined a decision-making framework that identifies howsocietal values can be introduced to guide discussionsand increase transparency on reimbursement decisions.The authors recognized that there are many places inthe decision-making framework where public values arerelevant [4].Dionne et al. developed a framework to operationalizea multi-criteria decision analysis (MCDA) method thatincorporates societal values on drug reimbursement de-cisions as an alternative to cost-effective analysis [7].MCDA is used to primarily measure value based onnumerous criteria. According to the results of two work-shops held in Canada with pharmaceutical, industry,patient, healthcare, and decision-making representatives,ten criteria were identified. They were as follows: com-parative effectiveness, adoption feasibility, risks of ad-verse events, patient autonomy, societal benefit, equity,strength of evidence, incidence/prevalence/severity ofcondition, innovation and disease prevention/health pro-motion [7]. In addition to drug reimbursement decisionsfor common diseases, the authors suggest that this frame-work is applicable to funding DRDs. At the time of publi-cation, the proposed framework had not been finalized.Public engagement helps to inform decision-makersabout the diverse perspectives of the Canadian population,including differences among stakeholder, patient, and citi-zen perspectives [8]. Since it is necessary to consider thefair distribution of health services or funds across healthneeds and how that is to be weighed against access toDRDs, it is important to include citizens or public whoseinterests are outside the access to DRDs. This broadrepresentation needs to be considered when the trade-offsbetween providing access for DRDs are weighed againstother possible uses (i.e., the opportunity cost) of the healthcare funds. Wider representation may add legitimacy toPolisena et al. BMC Health Services Research  (2017) 17:372 Page 2 of 9decisions and policies by involving diverse citizenry onwhat counts as fair and reasonable and by adding trans-parency to decision-making processes [9–11].Survey approaches to identify the perspectives ofrespondents tend to characterize their views based on asmall amount of information. Surveys can be distin-guished from deliberative approaches that emphasizewell-informed and civic-minded input of diverse, smallgroups, such as citizen juries and study circles. Carefulinforming and facilitation of small group deliberationproduces advice that balances the diversity of intereststo deliver civic-minded recommendations directed tospecific decisions or trade-offs. Nevertheless, publicviews aggregated from a survey reflect help decisionmakers understand how a wider public might respond topolicy changes. They many also be used to inform futuredeliberative engagement.The main objective of this study was to engage a diversesample of Canadians to determine their perspectives, in-cluding any regional variations, associated with DRDdecision-making, as determined through a national onlinesurvey. The responses provided insight into public per-spectives related to DRD funding that decision-makersmight want to consider when reviewing policies and deter-mining the allocation of scarce resources. This study wasreviewed and approved by the University of British Co-lumbia’s Behaviour Research Ethics Board (UBC BREBnumber H15-02972).MethodsSurvey questionnaireThe survey sought to characterize a sample of Canadians’priorities and perspectives associated with decision-making for DRDs. An online survey, available in Englishand French, was administered in January and February2016 (Additional file 1).The priorities and scenarios in the survey were devel-oped over 2 years of collaboration with researchers anddecision makers involved in provincial drug fundingdecisions and Canadian federal recommendations, andwith the Canadian Institutes for Health Research (CIHR)New Emerging Team for Rare Diseases [12]. Supportingrespondents’ ability to identify their priorities and re-ceive descriptions of how the priorities would be pro-moted or mitigated by the scenarios provided somedecision-support that is a rough parallel with the struc-ture of a deliberation. This approach moved from theidentification of participants’ hopes and concerns toassessing how these were affected by different policychoices. The survey design by MetroQuest encouragedrespondents to think about priorities in relation to DRDfunding decisions, and provided estimates of how theeach respondent’s selected priorities would be affectedby each of the funding scenarios. This structure aimedto guide respondents through a basic understanding ofthe policy challenge, identify their priorities, and relatethose priorities to policy decisions in a survey intendedto take less than 10 min. The final version of the surveywas reviewed and revised by the CIHR New EmergingTeam [12].Survey respondents were asked to rank priorities, andthen use the priorities to indicate their degree of supportfor the funding scenarios. This method may not achievethe degree of reflection and reason-giving typical of adeliberative engagement, but it provides an opportunityfor analysis to assess the priorities in relation to thescenarios [9, 13].Respondents ranked, in order of importance to them,at least three and up to five DRD decision-making prior-ities, out of a total of eight potential priorities listed inthe survey. The eight priorities presented included PainReduction, Improve Quality of Life, Longer Life, NationalEqual Access, Lack of Current Treatment, Severity ofSymptoms, Cost Containment, and Effective Health Care.They were also asked to compare and rate their agree-ment level with each scenario using a 5-point Likertscale, ranging from 1 = completely disagree to 5 = com-pletely agree. Each scenario represented a different prop-osition associated with funding for DRDs. The titles ofthe scenarios were: Fund Drug if Justified, CanadawideEquality, Pay for Drugs, and Prioritize Other Programs.Table 1 describes the full description of each scenario asincluded in the survey.Respondents were able to view an estimate of howeach funding scenario agreed or disagreed with theirselected priorities. Providing estimates of how eachscenario promoted or compromised the priority selec-tion was a decision support that helped survey respon-dents to more efficiently consider the scenarios based ontheir selected priorities. It did not, however, precludethem from rating the funding scenarios using a 5-pointLikert scale independently of the projected effect on theirselected priorities. Prior to submitting the survey, respon-dents had the opportunity to revise any of their responsesin previous screens and to submit comments in anopen-ended question with regards to DRD funding.Survey sampleA third-party organization, Asking Canadians, was respon-sible for recruiting the survey sample of approximately1600 individuals from across Canada. Respondents weresolicited from AskingCanadians’ panel. The company hadseveral quality assurance checkpoints to ensure the integ-rity of the survey respondents. For our study, once Asking-Canadians’ panel members were recruited, they were askedto confirm their desire to participate in the survey beforebeing redirected to the MetroQuest survey. A survey wasclassified as complete if at least three priorities werePolisena et al. BMC Health Services Research  (2017) 17:372 Page 3 of 9ranked, and all four scenarios were rated. Demographicdata was also collected for each respondent which includedage, annual household income, geographic region, sex, andeducation level.Survey data analysisDescriptive statisticsSurvey respondents were asked to rank at least threeand up to five DRD decision-making priorities in orderof importance from their own personal perspective. Wedetermined the proportion of respondents that rankedeach of the priorities (i.e., selected in their top three tofive priorities), independent of where it was ranked inrelation to the other priorities.Respondents’ agreement with each funding scenario wassummarized into two categories: agree (i.e., 4 = agree and5 = completely agree) and do not agree (i.e., 1 = completelydisagree, 2 = disagree, and 3 = neither agree nor disagree).Descriptive statistics were used to present the frequencyof the individual priority selected, agreement level witheach scenario and respondents’ demographics.Logistic regression analysesUnivariate analyses were performed to measure the asso-ciation between respondent demographics and theselected priorities with respondents’ agreement witheach funding scenario using logistic regression analyses.For each regression analysis, the dependent variable wasthe agreement level with a specific funding scenario, andthe reference category was “Do not agree”. Demograph-ics and priority selection with p-values less than 0.05from the univariate analyses were included in multiplelogistic regression analyses to obtain the association be-tween funding scenarios and demographics and priorityselection. All analyses were conducted using RStudio®(Version 0.99.878, Boston, USA).Content analysis on open-ended question on funding forexpensive drugsContent analysis, a qualitative research method, wasconducted to review and categorize the open-ended re-sponses submitted by 410 survey respondents [14, 15].The responses were reviewed, defined and organizedinto themes. An emergent list of codes of distinct ideaswas maintained, as well as their definitions and a sampletext passage that illustrates the application of each code.The codes were reviewed using a constant comparativetechnique to identify all instances and appropriatenessof the coding framework, and to determine how toexpand or merge them into themes. The responses werereviewed again to ensure that all the relevant informa-tion was coded. The themes derived from the contentanalysis were summarized narratively.ResultsDescriptive statisticsTable 2 presents the demographics of the 1631 respon-dents who completed the survey. An even proportionof males and females participated in the survey, andthe mean age was 48.0 years old (standard deviation =16.0 years). Close to 60% of respondents had com-pleted college or university, obtained a post-graduatedegree, or had a professional degree. Over 25% had anannual household income of more than CDN$100,000,and 22.9% of respondents (n = 373) preferred not toreveal their income. Among the 1631 respondents,Alberta (n = 162; 9.9%), Manitoba (n = 157; 9.6%), andSaskatchewan (n = 145; 8. 9%) represented the greatestproportion of respondents across all provinces, and32.7% of respondents (n = 533) preferred not identifytheir location.Table 1 Description of each funding scenarioFunding Scenario Description in SurveyPay for Drugs Provincial decision-makers fund newexpensive drugs. They do not consideralternative uses of funds but argue thatANY improvement for patient care mustbe funded, even if that means that newprograms cannot be started. This giveshigh priority to all benefits to patientswith rare diseases, at the expense offunding to other programs and patients.It does not address health care budgetsor provincial differences.Prioritize Other Programs Provincial decision-makers do not fundaccess to new expensive drugs. Instead,funding is allocated to programs thattarget reduced sickness and death inunderserved populations, often throughaccess to basic health care. Treatment forpatients with rare diseases are only fundedwhen they are highly effective and nottoo expensive. Health care budgets arecontained. Provincial differences are notequalized.Fund Drug if Justified Fund the drug ONLY if the extra expenseis well justified. Effective health care andcost containment are promoted. Prioritiesrelated to patients with rare diseases arepromoted only when they compare wellto other uses of funds and significantlyimprove treatments that are currentlyavailable. Provincial differences infunding are not changed.Canadawide Equality All provincial and territorial Ministriesof Health are required by a new lawto use recommendations of a federaldrug assessment agency. Canadiansreceive the same access across theprovinces and territories. Benefits forpatients with rare diseases are promotedwhen they compare well to alternativeuse of the funds and are significantimprovements over current treatments.This supports assessing cost containmentand effective health care on a national level.Polisena et al. BMC Health Services Research  (2017) 17:372 Page 4 of 9Surveys that ranked at least three priorities and ratedall scenarios were labelled as complete, but 24 com-pleted surveys were missing the scenario ratings. Thisfigure represents 1.47% of responses, so the impact onthe analyses was minimal.The priorities that were most frequently selected byrespondents as being important when considering fund-ing of drugs were Improve Quality of Life (n = 1304;80.0%) and Effective Health Care (n = 1232; 75.5%). Thethird most frequently selected priority was NationalEqual Access (n = 701; 45%). Less than 25% of respon-dents selected Lack of Current Treatment (n = 372;22.8%) and Longer Life (n = 376; 23.1%) (Table 3).Among the four scenarios presented, over 75% ofrespondents agreed with the scenario that described afunding model that grants equal access to treatment toall Canadians, independent of where they live, followedby 73.0% who agreed that a DRD should be funded ifdeemed to be cost-effective. Moreover, half of respon-dents support DRD funding to improve patient care forTable 2 Survey respondent characteristicsCharacteristic Frequency (N = 1631) (%) Mean (SD)Age (in years) 48.0 (16.0)Age Category (in years)18–34 448 (27.5)35–50 536 (32.9)51–75 545 (33.4)76–100 97 (6.0)No response 5 (0.3)SexFemale 819 (50.2)Education LevelLess than Grade 9 10 (0.6)Grade 9 to Grade 13 179 (11.0)Trades certificate or diploma 139 (8.5)Some college or university 268 (16.4)Completed college oruniversity662 (40.6)Post-graduate or professionaldegree310 (19.0)Prefer not to say 61 (3.7)No response 2 (0.1)Annual Household IncomeLess than $20,000–$44,999 254 (15.6)$45,000–$99,999 563 (34.5)$100,000–$499,999 416 (25.5)$500,000–$1,000,000+ 9 (0.6)Prefer not to say 373 (22.9)No response 16 (1.0)Province of ResidenceBritish Columbia 138 (8.5)Alberta 162 (9.9)Saskatchewan 145 (8.9)Manitoba 157 (9.6)Ontario 130 (8.0)National Capital Regiona 20 (1.2)Quebec 130 (8.00)Nova Scotia 55 (3.4)New Brunswick 42 (2.6)Prince Edward Island 6 (0.4)Newfoundland and Labrador 113 (6.9)Preferred not to say 533 (32.7)aIncludes Ottawa and Gatineau; SD standard deviationTable 3 Frequency of priority selection and agreement level forfunding scenarios (N = 1631)Priority SelectionDescription Frequency (%)Improve Quality of Life 1304 (80.0)Effective Health Care 1232 (75.5)National Equal Access 701 (43.0)Pain Reduction 642 (39.4)Cost Containment 543 (33.3)Severity of Symptoms 430 (26.4)Longer Life 376 (23.1)Lack of Current Treatment 372 (22.8)Funding Scenario by Agreement LevelDescription Agreement Level Frequency (%)Canadawide Equality Agree 1292 (79.2)Neither agree nor disagree 229 (14.0)Disagree 95 (5.8)No response 15 (0.9)Fund Drug if Justified Agree 1190 (73.0)Neither agree nor disagree 311 (19.1)Disagree 115 (7.1)No response 15 (0.9)Pay for Drugs Agree 818 (50.2)Neither agree nor disagree 484 (29.7)Disagree 305 (18.7)No response 24 (1.4)Prioritize Other Programs Agree 601 (36.8)Neither agree nor disagree 549 (33.7Disagree 460 (28.2)No response 21 (1.3)Agree: 4 = agree and 5 = completely agree; Disagree: 1 = completely disagreeand 2 = disagree; Neither agree nor disagree: 3 = neutralPolisena et al. BMC Health Services Research  (2017) 17:372 Page 5 of 9rare diseases, while over 35% agreed that provincialfunding should be allocated to programs that targetreduced sickness and death in underserved populationsinstead of funding DRDs.Logistic regression analysesIn our initial analyses, we evaluated the relationship be-tween agreement with each funding scenario and respond-ent demographics including geographical location. SeeAdditional file 2 for frequency of priority ranking andratings of scenarios by province. We did not, however, findany significant associations between demographic charac-teristics and respondents’ agreement with any specificscenario.Table 4 presents the results of multiple logistic regres-sion analyses that included variables in which the associ-ation between the funding scenarios and individualpriority selections were statistically significant. As therewas no association between any demographic character-istics and the level of agreement with each scenario, onlythe relationship between priority and agreement levelwith the scenarios were modelled.The selection of Cost Containment as one of the toppriorities was associated with the level of agreement onthree of the four funding scenarios. Specifically, thosewho selected Cost Containment in their top prioritieswere less likely to agree with paying for all drugs for rarediseases and Canada-wide equality of drug access versusthose who did not select this priority. Respondents, whoranked Cost Containment in their top priorities, werealso 67% more likely to agree with the prioritization ofother programs over programs for rare diseases. Thosewho selected Longer Life were more likely to agree withboth paying for drugs for rare diseases as a matter ofprinciple, and the necessity of Canada-wide equality foraccess to treatment. Selecting Effective Health Care inthe top priorities was positively associated with bothprioritizing other programs over programs for rarediseases, and only funding a drug treatment if justified.As well, respondents, who selected National Access asone of the top priorities, were four times more likely toagree with the scenario to provide equal access to drugsacross Canada.Content analysis on open-ended question on funding forexpensive drugsRespondents had the opportunity to submit their com-ments on DRD funding. Among the 1631 respondentswho completed the survey, 410 responded to the open-ended question (25.14%).Several themes that emerged from the commentssubmitted on funding for expensive drugs were alignedwith the priorities listed in the survey, such as nationalaccess to DRDs and cost containment. Other themescaptured in the comments were directly related to thepatient’s well-being and welfare. They included the fi-nancial burden imposed on patients and considerationfor their age and autonomy. For example, several re-spondents wrote that families experience a heavy finan-cial burden when drugs are not funded, and individualsshould not have to choose between paying for drugs fortheir health versus paying for their food. Respondentsalso commented on drug coverage decisions based oncost-effectiveness versus decisions driven by compas-sion for the patient, and they questioned how the valueof life was measured. The impact of media coverage onthe individual cases and ensuring the accountability ofindustry, who charge high costs for DRDs, werehighlighted as concerns by numerous respondents. Forinstance, one respondent wrote that decisions shouldbe based on real data and not on emotions or “over-the-top” media-coverage. Themes identified also fo-cused on the balance between funding for DRDscompared with funding drugs for common diseases thataffect a larger population, coverage through private in-surance versus public sources, and increased frequencyof bulk purchases to obtain large volume discounts.One respondent commented that a national drug pro-gram was needed, and another respondent suggested aCanada-wide purchase scheme to reduce the cost ofdrugs. Additional themes that surfaced from the re-spondents’ comments included placing more emphasison preventive care and the availability of non-pharmaceutical therapies and generic drugs to treatpatients with rare diseases. Additional file 3 presentsTable 4 Association between priority selection and agreementwith each funding scenarioFunding Scenarioa Priority OR 95% CIPay for Drugsb Longer Life 1.52 1.20–1.93Cost Containmentb 0.71 0.58–0.88Prioritize Other Programs Effective Healthcare 1.42 1.17–1.82Cost Containment 1.67 1.34–2.06Lack of Treatment 0.78 0.61–1.00Fund Drug if Justified Effective Healthcare 1.55 1.21–1.98Canada-wide Equality Longer Life 1.57 1.14–2.20Cost Containment 0.50 0.38–0.65National Access 4.14 3.07–5.65Severity of Symptoms 0.70 0.53–0.93aReference category = Do not agreebInterpretation example: Respondents who ranked cost containment as onetheir top priorities were 29% less likely (OR 0.71) to agree that provincialpayers should pay for DRDs if there is any improvement in health, withoutconsidering the potential uses of these funds. Odds ratios represent the oddsof agreeing or strongly agreeing with the scenario if that priority was selectedas a top priority versus if it was notAgree: 4 = agree and 5 = completely agree; CI: confidence interval; Do notagree: 1 = completely disagree, 2 = disagree, and 3 = neither agree nordisagree; OR = odds ratioPolisena et al. BMC Health Services Research  (2017) 17:372 Page 6 of 9sample comments provided by the survey respondentsfor each theme.DiscussionOur survey results based on 1631 responses acrossCanada were that Improved Quality of Life and EffectiveHealth Care were priorities most frequently selectedamong the survey respondents that should be consid-ered when making decisions about funding of DRDs.Although reimbursement decisions are made by the pro-vincial and territorial drug plans, a majority of respon-dents also agreed with the scenarios that all Canadiansshould have equal access to the same drugs and to funda DRD only if it is justified in terms of the drug’s cost-effectiveness. As per their descriptions, both scenariossupport the Effective Health Care and Cost Containmentpriorities (Table 1). Our analyses identified associationsbetween the funding scenarios and several priority selec-tions. Although access to DRDs varies by jurisdiction,we did not find a significant relationship between theagreement with the funding scenarios and geographiclocation. Regardless of the geographic location, a major-ity of the respondents support federal drug assessmentthat would determine which drugs are deemed as pro-viding value for money for coverage, and that coverageshould serve the goal of ensuring that Canadians havethe same access to DRDs across all jurisdictions.Our survey findings were aligned with the results of a2014 study that presented 13 trade-off scenarios to 2111participants to measure values of the Canadian publicon drug prioritization and reimbursement for rare dis-eases versus common diseases and other non-health careprograms [16]. More specifically, both studies found thatparticipants supported national equity for DRDs [16].LimitationsThe interpretation and application of our study may beinfluenced by several limitations. The survey presented adescription of each priority and scenario, but it is uncer-tain how the respondents interpreted the descriptionsand if they required additional information for clarifica-tion purposes. Moreover, they did not have much of anopportunity to consider expert and stakeholder input,and our methodology did not use time-trade-off re-quired by decision makers when prioritizing or allocat-ing finite resources. The survey responses suggested thata majority would support national consistency of fund-ing decisions based on evidence of comparative cost-effectiveness, and 50% agree to fund DRDs. Althoughthe survey responses highlighted a diversity of perspec-tives, it is not feasible to identify all Canadian perspec-tives based on the number of limited number ofrespondents [9, 11]. In the current sample, a greater pro-portion of our survey respondents had a college diplomaor university degree or earned at least $100,000 com-pared with the Canadian population [17, 18]. Althoughthe survey sampling targets were not based on the actualCanadian population density, targeted demographics bygeographic area, as well as age and sex, were specifiedand were within 5% of the targets. This approach helpedensure that a variety of perspectives were representedfor comparative purposes.Directions for future researchThe survey responses will help to inform the planningand implementing a deliberative public engagementworkshop on decision-making processes for DRDs. Thetheory of deliberative democracy argues that the mini-forums created for the deliberative engagement presentadvice that is the best estimate of how citizens wouldadvise knowledge users if citizens were informed, civic-minded and deliberative [19]. It is a common belief thatcitizens are not generally well informed, nor are theysupported to consider the perspectives of others and thecommon good. That said, the online survey provides anestimate of advice from non-deliberating informed citi-zens. The responses can be considered by the deliberationparticipants, and by knowledge users as they decidewhether and how to incorporate advice from these differ-ently constituted forums. One objective of this study wasto use demographic associations, including geographicallocation, with different the survey responses to guide thesample selection for the deliberative engagement. Initialanalyses did not identify any statistically significant associ-ations that can be used for this purpose. However, themesthat emerged from the content analysis can help to iden-tify information that participants might require to supportdeliberation, which can be provided prior to the workshopand during the deliberations. For example, several com-ments were associated with the promotion of preventivecare and use of generic drugs as a treatment option. Alarge proportion of rare diseases have genetic origins, andDRDs usually have extended patent protection comparedwith therapies for more common indications. The currentsurvey uses the Likert scale to gauge the respondents’ toppriorities and level of agreement with the funding scenar-ios associated with DRD funding. It remains uncertain ifthe results of this survey align with the perspectives of thedecision-making bodies in Canada, especially in jurisdic-tions with programs for DRDs. An analysis on the Canad-ian public’s perspectives compared with the values andprinciples of these programs is merited. Studies on thepublic’s perspectives in other countries and how theycontrast with those among Canadians are also warranted.Future research can apply the trade-off technique tomeasure the Canadian public’s perspectives in which re-spondents must consider and compare all key attributesPolisena et al. BMC Health Services Research  (2017) 17:372 Page 7 of 9associated with each priority and funding scenario. Theirresponses can then be compared with those derived fromthis survey.ConclusionsAn online national survey was conducted to inquire aboutthe Canadian publics’ priorities and perspectives on fund-ing decisions for expensive drugs for rare diseases. Amongthe 1631 respondents, Improved Quality of Life and Effect-ive Health Care were most frequently selected as the toppriorities. Ensuring equal access across Canada and fund-ing for DRD only when the drugs were deemed to becost-effective had the highest agreement level among thesurvey respondents, while 50% agreed that DRDs shouldbe funded independent of their effectiveness. Our analysesdid not identify any significant relationships between therating of the scenarios and demographics, including geo-graphic location. The survey results suggest that while apolicy that provided national equity for cost-effectivedrugs would be widely supported, there are likely to be asignificant number of citizens who will object to anyrestrictions to access to DRDs.Additional filesAdditional file 1: Screen Captures of Online National Survey.Supplementary file 1 presents the screen captures of the online nationalsurvey on drugs for rare diseases. (DOCX 1070 kb)Additional file 2: Frequency of Priority Rankings by Province.Supplementary file 2 presents the results of the frequency of priorityrankings by jurisdiction. (DOCX 26 kb)Additional file 3: Comments from Survey Respondents. Supplementaryfile 3 presents a sample of the written comments from the surveyrespondents. (DOCX 55 kb)AbbreviationsCDN: Canadian; CIHR: Canadian Institutes of Health Research; DRDs: Drugs forrare diseases; MCDA: Multi-criteria decision analysisAcknowledgementsThe authors are grateful for input from Stirling Bryan and Bob Nakagawa,and from the members of the CIHR Emerging Team for Rare Diseases.FundingThis research was funded by the Canadian Agency for Drugs andTechnologies in Health (C-150390) and a Canadian Institutes for HealthResearch Grant Team (119193). It has also been completed in partialfulfillment of the requirements of Julie Polisena’s Doctoral Fellowship withthe Society for Medical Decision-Making. The authors take sole responsibilityfor the final form and content of this document. The views expressed hereinare those of the authors and do not necessarily reflect the views of CADTH.Availability of data and materialsSurvey data are available upon request to the corresponding author.Authors’ contributionsJP led the data analysis of the survey results and the manuscript preparation.MB led the survey design and contributed to the manuscript preparation.CM and LDL contributed to the survey design and the manuscriptpreparation. All authors read and approved the final manuscript.Competing interestsThe authors declare that they have no competing interests.Consent for publicationNot applicable.Ethics approval and consent to participateThis study was reviewed and approved by the University of BritishColumbia’s Behaviour Research Ethics Board (UBC BREB number H15-02972).AskingCanadians asked the panel members to confirm their consent toparticipate in the online survey.Publisher’s NoteSpringer Nature remains neutral with regard to jurisdictional claims inpublished maps and institutional affiliations.Author details1Canadian Agency for Drugs and Technologies in Health, 600-865 CarlingAve, Ottawa K1S 5S8, ON, Canada. 2W. Maurice Young Centre for AppliedEthics, School of Population and Public Health, University of British Columbia,Kelowna, BC, Canada. 3School of Population and Public Health, University ofBritish Columbia, Vancouver, BC, Canada. 4Centre for Clinical Epidemiologyand Evaluation, Vancouver, BC, Canada. 5Faculty of Pharmaceutical Sciences,University of British Columbia, Vancouver, BC, Canada. 6Centre for HealthEvaluation and Outcomes Sciences, Providence Health Research Institute,Vancouver, BC, Canada.Received: 11 June 2016 Accepted: 16 May 2017References1. Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, Hughes DA.Rare Disease Terminology and Definitions-A Systematic Global Review: Reportof the ISPOR Rare Disease Special Interest Group. Value Health. 2015;18:906–14.2. Menon D, Clark D, Stafinski T. Reimbursement of Drugs for Rare Diseasesthrough the Public Healthcare System in Canada: Where Are We Now?Healthc Policy. 2015;11:15–32.3. Loorand-Stiver L, Cowling T, Perras C. Drugs for rare diseases: evolvingtrends in regulatory and health technology assessment perspectives.Ottawa: CADTH; 2016.4. Paulden M, Stafinski T, Menon D, McCabe C. Value-Based ReimbursementDecisions for Orphan Drugs: A Scoping Review and Decision Framework.Pharmacoeconomics. 2014;33(2):255–69.5. Short H, Stafinski T, Menon D. A national approach to reimbursementdecision-making on drugs for rare diseases in Canada? Insights from acrossthe ponds. Healthc Policy. 2015;10:24–46.6. Office of Legislative and Regulatory Modernization. Initial draft discussiondocument for a Canadian orphan drug regulatory framework. Ottawa:Health Canada; 2012.7. Dionne F, Mitton C, Dempster B, Lynd LD. Developing a multi-criteriaapproach for drug reimbursement decision making: an initial step forward. JPopul Ther Clin Pharmacol. 2015;22:e68–77.8. Kahane D, Loptson K, Herriman J. Stakeholder and Citizen Roles in PublicDeliberation. J Public Deliberation. 2013;9:1–37.9. Longstaff H, Burgess MM. Recruiting for representation in publicdeliberation on the ethics of biobanks. Public Underst Sci. 2010;19:212–24.10. Molster C, Maxwell S, Youngs L, Kyne G, Hope F, Dawkins H, O'Leary P.Blueprint for a deliberative public forum on biobanking policy: weretheoretical principles achievable in practice? Health Expect. 2013;16:211–24.11. O'Doherty K, Gauvin FP, Grogan C, Friedman W. Implementing a publicdeliberative forum. Hastings Cent Rep. 2012;42:20–3.12. The changing landscape of treatments for rare diseases. Vancouver (BC):CIHR New Emerging Team for Rare Diseases; 2016.13. O'Doherty KC. Synthesising the outputs of deliberation: Extractingmeaningful results from a public forum. J Public Deliberation. 2013;9:1–16.14. Hsieh HF, Shannon SE. Three approaches to qualitative content analysis.Qual Health Res. 2005;15:1277–88.15. Morgan DL. Qualitative content analysis: a guide to paths not taken. QualHealth Res. 1993;3:112–21.Polisena et al. BMC Health Services Research  (2017) 17:372 Page 8 of 916. Rizzardo S. An evaluation of Canadians values and attitudes towardsexpensive drugs for rare diseases. Vancouver (BC): University of BritishColumbia; 2014.17. Education in Canada: attainment, field of study and location of study.Ottawa: Statistics Canada; 2011.18. Individuals by total income level, by province and territory (Canada).Ottawa: Statistics Canada; 2015.19. Goodin RE, Dryzek JS. Deliberative impacts: the macro-political uptake ofmini-publics. Polic Soc. 2006;34:219–44.•  We accept pre-submission inquiries •  Our selector tool helps you to find the most relevant journal•  We provide round the clock customer support •  Convenient online submission•  Thorough peer review•  Inclusion in PubMed and all major indexing services •  Maximum visibility for your researchSubmit your manuscript your next manuscript to BioMed Central and we will help you at every step:Polisena et al. BMC Health Services Research  (2017) 17:372 Page 9 of 9


Citation Scheme:


Citations by CSL (citeproc-js)

Usage Statistics



Customize your widget with the following options, then copy and paste the code below into the HTML of your page to embed this item in your website.
                            <div id="ubcOpenCollectionsWidgetDisplay">
                            <script id="ubcOpenCollectionsWidget"
                            async >
IIIF logo Our image viewer uses the IIIF 2.0 standard. To load this item in other compatible viewers, use this url:


Related Items