Methodology for constructing scenarios for health policy research: The case of coverage decision-making for drugs for rare diseases in Canada

https://doi.org/10.1016/j.techfore.2021.120960Get rights and content

Highlights

  • Here specific scenario methodology are described to help inform health policy.

  • Methods are demonstrated in coverage decision-making for drugs for rare diseases.

  • Scenarios are used in policy focus groups to explore impacts of external factors.

  • Scenarios are shown to help policy makers identify areas for positive change.

  • They help to identify additional challenges and think differently about the future.

Abstract

When futures are uncertain and technological developments stand to have large impacts on healthcare systems, scenario studies can be deployed as a tool for policy exploration. We describe the important differences between firm-based and policy-oriented scenarios, and show how to adapt them for health policy. Our approach involves a five-phase process. Background interviews with a diverse group of stakeholders are used to identify focal points and future challenges. Interview data is analyzed to identify the key drivers of future challenges, which are then used to develop a set of scenarios. The scenarios are deployed in a focus group setting with relevant health policy makers and advisors. We illustrate this approach with our scenario study on coverage decision-making for drugs for rare diseases in Canada. Our focus here is on the extension and application of this scenario method and a sharing of our experiences while applying this method. We demonstrate how scenarios can be applied to identify targets of robust health policy strategies that will be of value irrespective how the future unfolds. Our post-focus group questionnaire show that the majority of participants found the scenario focus group useful, and helped them to identify additional challenges regarding drugs for rare diseases.

Introduction

The adoption of new healthcare technologies often leads to substantial improvements in patient outcomes (Skinner and Staiger, 2015, Cutler and McClellan, 2001, Salas-Vega and Mossialos, 2016, Howard et al., 2016, Liu et al., 2017, Vissers et al., 2016), but also leads to an increase in healthcare spending that can threaten the sustainability of health systems (Lehoux et al., 2016). A current challenge faced by health policy practitioners tasked with managing technological change is that the high rate of innovation in biomedical technologies like genomics (Wetterstrand, 2016) makes it extremely difficult to forecast the budgetary impact of new healthcare technologies in the medium-to-long-term. This hampers policy-makers’ ability to engage in strategic planning to ensure health system sustainability.

A useful planning tool in such circumstances is the scenario study, which has been successfully deployed for decades in business and management studies as a method for strategic foresight when futures are uncertain and technological developments stand to have large impacts on industrial sectors (Agami et al., 2008, Bishop et al., 2007, Porter and Xu, 1990, Ringland, 2010, Tenaglia and Noonan, 1992). Scenario studies are ideally suited to assist health system policy-makers in envisioning possible futures and developing strategies to manage technological change. Scenarios have been used to facilitate long-range strategic planning for a number of health-related technologies, including telemedicine (Kiran, 2012), drug therapy for obesity (Swierstra et al., 2009), and bionanotechnology (Boenink et al., 2010), as well as by major pharmaceutical companies (Bradfield and El-Sayed, 2009).

The scenario study approach is based on the premise on the uncertainty of the future, and as a result exploring a diversity of possible futures allows managers and decision-makers to prepare for an array of plausible social and technological changes (Tenaglia and Noonan, 1992). When managers and decision-makers engage with scenarios, they not only stand to accelerate organizational learning about future challenges, but they can also work to build consensus on a mental model of the key variables that will influence their organization's future environment (Tenaglia and Noonan, 1992). This facilitates rigorous and creative strategic planning by leadership teams faced with technological uncertainty.

Scenario studies have, however, faced significant methodological criticism focused on the lack of transparency in most published reports regarding the specific processes through which scenarios are constructed (Bishop et al., 2007, Bradfield et al., 2005, Wright et al., 2008, Wright et al., 2013, Wright and Goodwin, 2009). This makes it difficult for others to assess the utility and broader relevance of published scenario studies and to replicate them in different organizational contexts. In response to this critique, Phadnis et al. outlined a systematic scenario construction methodology for industry (Phadnis et al., 2014). They acknowledge that the use of scenarios in policy settings presents a distinct set of challenges (e.g., the diverse group of stakeholders and values that must be taken into account in scenario development in a policy setting) that require further methodological work (Phadnis et al., 2014, van and MVV, 2012).

To this end, this article provides a detailed account of how a Canadian scenario study that focused on the health policy challenges associated with the growing number of expensive drugs for rare diseases (DRD) was designed and conducted. Specifically, we describe in detail our approach to developing a set of plausible scenarios for the future DRD landscape in Canada. These four scenarios were deployed in several focus groups in 2015 to help Canadian reimbursement decision-makers and health policy-makers reflect on a wide range of possible futures, and on how coverage decision-making processes could adapt to ensure future health system sustainability. Our detailed methodological description should allow other researchers and practitioners to adapt our systematic approach to developing useful scenarios for policy-oriented studies to different health policy contexts in which uncertain technological futures are a challenge to strategic planning. While the full results of the scenario deployment are reported on separately, our primary focus here is on the extension and application of this scenario method and a sharing of our experiences while applying this method. Irrespective of this methodological focus, we provide a glimpse into the kind of qualitative data generated from these kinds of scenario focus groups and how that data can be used as the foundation for future orientated health policy. We also present questionnaire data from focus group participants about the value and utility of the scenario exercise.

The rapidly-declining cost of genomic sequencing (Wetterstrand, 2016) has enabled dramatic advances in our ability to discover and diagnose rare monogenic disorders (Vissers et al., 2016, 24, Deciphering Developmental Disorders Study, 2017, Dragojlovic et al., 2018). Today there are an estimated 7000 known rare diseases, most of which have a genetic cause (Boycott et al., 2013). This expanded ability to diagnose rare genetic disorders in patients has been accompanied by a shift in the pharmaceutical industry away from developing traditional blockbuster drugs for common conditions and towards “niche busters” that target smaller populations, but provide higher return on investment (Long and Works, 2013) due to their extremely high prices (Collier, 2011, Dolgin, 2010). Indeed, driven by incentives included in the U.S. Orphan Drug Act of 1983, advances in biotechnology, and the demonstrated profitability of DRD (Kesselheim, 2011), the fraction of new molecular entities approved by the U.S. Food and Drug Administration (FDA) that had an orphan designation moved from less than 15% in the 1980s to more than 35% in the 2010s (Kinch et al., 2014). These developments stand to provide hope for some patients where treatments were previously unavailable. However, drugs used in the treatment of rare diseases are typically very expensive and too costly for most patients and their families to pay for out-of-pocket. A Canadian review of costs of drugs for ultra-rare diseases (i.e. drugs used to treat diseases that affect ≤ 1 per 100,000 people) approved from 2004-2016 “were found to have a median annual cost per patient of CAN$330,395 (range = CAN$934,000)” per year (Richter et al., 2018).

In countries with universal publicly-funded healthcare systems, the increased availability and high cost of new DRD poses a major challenge for payers. In 2016 the “health expenditure share of total provincial/territorial government program expenditures (e.g., on health, education, transportation/communication, social services) was around 37%” (Canadian Institute for Health Information, 2018), and by 2018 the percentage of prescription drugs covered by the public system was 42.7% (or $14.4 billion), which “reflects an annual increase of 5.0% [from 2017], compared with 3.6% growth in private-sector spending” and up 4.6% from 20161 (Canadian Institute for Health Information, 2018). Overall, drugs continue to account for the second largest shares of health dollars (15.7%) in Canada, and “drugs expenditure has outpaced that of Hospitals or Physicians, due in part to the increased use of high-cost patented drugs” (Canadian Institute for Health Information, 2018). In 2017, Canada public drugs programs spent 36.6% of their budget on drugs costing $10,000 or more, which treated 2.3% of individuals covered by drugs programs (Canadian Institute for Health Information, 2018). While at the time of writing Canada lacked an framework for drugs for rare disease (or a national drug framework of any kind) in which it could coordinate its response to this health policy challenge, the 2019 Federal Budget has proposed that the Canadian government will invest up to one billion dollars over two years, starting in 2022-2023, with up to five hundred million dollars invested in DRD per year ongoing. The government has indicated that this budget would include "the creation of a national strategy for high-cost drugs for rare diseases, to gather and evaluate evidence on high-cost drugs for rare diseases, improve the consistency of decision-making and access across the country, negotiate prices with drug manufactures, and ensure that effective treatments reach the patients who need them" (Government of Canada D of F. Budget, 2019).

Added to the weight that drugs carry in the health policy context, decisions surrounding market authorization of DRD occur at the national level in Canada (Menon and Stafinski, 2009), but decisions on their reimbursement by public insurance programs are a responsibility of the thirteen provinces and territories. As is done internationally (Cleemput et al., 2012, Clement et al., 2009), provincial health ministries use health technology assessment (HTA) methods to assess the cost-effectiveness of new drugs and to inform their coverage decisions for more commonly used drugs (Menon and Stafinski, 2009). However, if traditional HTA methods were strictly applied to DRD, then almost none of them would be covered due to their extremely high incremental cost, and patients would remain untreated given the dearth of pharmaceutical alternatives. For example, the incremental cost-effectiveness ratio (ICER) of eculizumab (Soliris) for the treatment of Paroxysmal Nocturnal Hemoglobinuria has been estimated at around $2 million (Canadian dollars) per quality adjusted life year (QALY) gained (Coyle et al., 2014). Such costs are orders of magnitude higher than the willingness-to-pay thresholds typical used by payers in the United States (US$50,000/QALY (Neumann et al., 2014)) and the United Kingdom (between £20,000 and £30,000 per QALY (McCabe et al., 2008)), and proposed for Canadian healthcare systems (C$20,000 to C$100,000 per QALY (Laupacis et al., 1992, Cape et al., 2013)). As a result, payers in Canada and elsewhere are increasingly taking into account other considerations, such as equity concerns and broader conceptions of “societal value” when making coverage decisions for DRD, and often approve funding for DRD despite extremely high ICERs (Menon et al., 2015, Simoens, 2011).

While this approach to dealing with DRD is feasible at the moment, it is uncertain whether it can be maintained over the medium- and long-term. There were an estimated 1800 DRD in the development pipeline in 2012 (Long and Works, 2013). As the number of DRD on the market and of known rare genetic diseases continue to grow, the DRD share of total spending on pharmaceuticals will also grow. Indeed, this class of drug was forecasted to account for 15.9% of the global prescription drug market in 2018, as compared to 5.1% in 1998 (EvaluatePharma, 2013). This is likely to put severe pressure on healthcare payers’ budgets, since more requests will be made to private and public drug programs for coverage of a growing number of novel drugs that are often very expensive and of uncertain efficacy. Given limited healthcare budgets, decision-makers face difficult choices about which DRD to cover and for which patients.

Indeed, restricted access to DRD has already come under criticism, particularly in Canada (Clarke, 2006), and while changes are being proposed at the federal level in the way in which DRD can be regulated (Lee and Wong, 2013, Lee, 2013, Canada PMPRB, 2020), this provides little respite for provincial drug coverage decision-makers who must make final coverage decisions and bear the responsibility for the sustainability of the healthcare system. While the trends outlined above are clear, a number of uncertainties remain that complicate strategic planning by Canadian healthcare systems on how to manage the impact of DRD. How many rare diseases are health systems likely to be dealing with in the future? How fast will new treatments for those diseases emerge on the market, what will the pricing models for those products be, and what role will subsequent-entry biologics play in this dynamic? In this context of uncertainty, major technological change, and high impact, scenario studies can be a valuable tool in exploring possible futures, which can then be used to in the development of health policy and strategic planning for health ministries.

Section snippets

Theory & methods

Scenario methods have been used by private sector companies (Schoemaker and van der Heijden, 1992) as well as major research and governing bodies (Dingwall et al., 2011, European Patent Office, 2007) to explore uncertain futures and address knowledge and policy gaps when systems and institutional apparatuses face transformative challenges. In their overview of scenario types and techniques Börjeson and colleagues create a classification of scenarios based on the principal questions they believe

Phase 1 – Background interviews & project orientation

At the outset of any scenario exercise, the focal issue (or decision) must be identified in close collaboration with the organization in question (Phadnis et al., 2014, Schwartz, 1991). We approached leaders in four provincial pharmaceutical insurance programs about the idea of a scenario project regarding DRD, and with their support it was agreed that the focal issue was how coverage decision-making for DRD could be made in the next fifteen years. Notably, having a specific future timeframe is

Preliminary findings from our scenario study on drugs for rare diseases

Two of the objectives of our scenario study were to explore how advisory systems might respond to current trends and future challenges, and to foster reflection on the part of participating advisors / decision-makers. The results of our anonymous questionnaire assessing participants’ views of the scenario exercise suggest the construction and deployment of scenarios in this manner is valuable in health policy settings (see Table 4). We collected views from all of our participants both before

Conclusion

Here we have provided a detailed methodological description of our approach to explorative external scenario development and demonstrated how it can help to identify the key drivers of future challenges in an area of health policy that is characterised by high levels of future uncertainty and technological change. While we have demonstrated how this approach has been used on a particularly challenging area of health policy (i.e. coverage of DRD), the methodological approach outlined here allows

CRediT authorship contribution statement

Conor M.W. Douglas: Conceptualization, Methodology, Validation, Formal analysis, Investigation, Writing - original draft, Writing - review & editing, Supervision, Project administration, Funding acquisition. Dimitra Panagiotoglou: Methodology, Validation, Formal analysis, Investigation, Data curation, Writing - original draft, Writing - review & editing, Visualization. Nick Dragojlovic: Conceptualization, Methodology, Data curation, Writing - original draft, Writing - review & editing,

Acknowledgments

This work was supported by the Canadian Institutes of Health Research (CIHR) Emerging Team for Rare Diseases (Funding Reference Number 119193), and together with the Michael Smith Foundation for Health Research (MSFHR) Partnership Grant “Informing Future Orphan Drug Coverage Using Scenario Studies” (Funding Reference Number 135919).

References (87)

  • EHM Moors et al.

    Orphan drugs: Unmet societal need for non-profitable privately supplied new products

    Res Policy.

    (2007)
  • S Phadnis et al.

    Axiomatic Foundation and a Structured Process for Developing Firm-Specific Intuitive Logics Scenarios

    Technol Forecast Soc Change

    (2014)
  • G. Ringland

    The role of scenarios in strategic foresight

    Technol Forecast Soc Change

    (2010)
  • DKR Robinson et al.

    Policy lensing of future-oriented strategic intelligence: An experiment connecting foresight with decision making contexts

    Technol Forecast Soc Change

    (2021)
  • E Wollenberg et al.

    Using scenarios to make decisions about the future: anticipatory learning for the adaptive co-management of community forests

    Landsc Urban Plan

    (2000)
  • D Wright et al.

    Policy scenarios as an instrument for policymakers

    Technol Forecast Soc Change

    (2020)
  • G Wright et al.

    Does the intuitive logics method – and its recent enhancements – produce “effective” scenarios?

    Technol Forecast Soc Change

    (2013)
  • G Wright et al.

    Decision making and planning under low levels of predictability: Enhancing the scenario method

    Int J Forecast

    (2009)
  • G Wright et al.

    Scenario planning interventions in organizations: An analysis of the causes of success and failure

    Futures

    (2008)
  • A Barak et al.

    Orphan drugs: pricing, reimbursement and patient access

    Int J Pharm Healthc Mark

    (2011)
  • P Bishop et al.

    The current state of scenario development: an overview of techniques

    Foresight

    (2007)
  • Tom Blackwell

    World's Most Expensive Drug - Which Costs up to $700,000 Per Year - Too Expensive, Canada Says. National Post [Internet]

    (2015 Feb)
  • M Boenink et al.

    Anticipating the interaction between technology and morality: a scenario study of experimenting with humans in bionanotechnology

    Stud Ethics Law Technol [Internet]

    (2010)
  • KM Boycott et al.

    Rare-disease genetics in the era of next-generation sequencing: discovery to translation

    Nat Rev Genet

    (2013)
  • R Bradfield et al.

    Four scenarios for the future of the pharmaceutical industry

    Technol Anal Strateg Manag

    (2009)
  • Canada PMPRB

    PMPRB Guidelines [Internet]. aem

    (2020)
  • Canadian Institute for Health Information

    National Health Expenditure Trends, 1975 to 2018 [Internet]

    (2018)
  • Canadian Institute for Health Information

    Prescribed Drug Spending in Canada, 2018: A Focus on Public Drug Programs. [Internet]

    (2018)
  • JD Cape et al.

    Introduction to cost-effectiveness analysis for clinicians

    Univ Tor Med J [Internet].

    (2013)
  • K. Charmaz

    Constructing Grounded Theory: A Practical Guide Through Qualitative Analysis

    (2006)
  • JTR. Clarke

    Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy

    Can Med Assoc J

    (2006)
  • I Cleemput et al.

    European drug reimbursement systems’ legitimacy: five-country comparison and policy tool

    Int J Technol Assess Health Care

    (2012)
  • F Clement et al.

    Using effectiveness and cost-effectiveness to make drug coverage decisions: a comparison of Britain, Australia, and Canada

    JAMA.

    (2009)
  • R. Collier

    Bye, bye blockbusters, hello niche busters

    Can Med Assoc J

    (2011)
  • D Coyle et al.

    Opportunity cost of funding drugs for rare diseases: the cost-effectiveness of eculizumab in paroxysmal nocturnal hemoglobinuria

    Med Decis Making

    (2014)
  • DM Cutler et al.

    Is technological change in medicine worth it?

    Health Aff (Millwood)

    (2001)
  • Deciphering Developmental Disorders Study

    Large-scale discovery of novel genetic causes of devleopmental disorders

    Nature

    (2015)
  • Deciphering Developmental Disorders Study

    Prevalence and architecture of de novo mutations in developmental disorders

    Nature

    (2017)
  • A Denis et al.

    Issues surrounding orphan disease and orphan drug policies in Europe

    Appl Health Econ Health Policy

    (2010)
  • R Dingwall et al.

    BBSRC sustainable bioenergy bioenergy scenario tool [Internet]

    Biotechnology and Biological Sciences Research Council (BBSRC)

    (2011)
  • E. Dolgin

    Big pharma moves from “blockbusters” to “niche busters.”

    Nat Med

    (2010)
  • Douglas CMW, Wilcox E, Burgess M, Lynd LD. Why Orphan Drug Coverage Reimbursement Decision-Making Needs Patient and...
  • N Dragojlovic et al.

    The cost and diagnostic yield of exome sequencing for children with suspected genetic disorders: a benchmarking study

    Genet Med

    (2018)
  • Cited by (4)

    • Forecasting spending on orphan diseases to maintain the long-run financial sustainability of healthcare system

      2023, Zhournal Novoi Ekonomicheskoi Associacii /Journal of the New Economic Association
    View full text