Public reimbursement for orphan drugs is an emergent challenge in health care decision making. A Nova Scotia approach to multi-criteria decision analysis could leverage existing national drug approval resources while also yielding a consistent, transparent, and fair approach to drug reimbursement decisions.

Expensive Drugs for Rare Diseases in Nova Scotia Results and Recommendations

Case #6: Policy Hack Competition

June 8, 2018

Team Members: Melissa Goertzen Nicholas Patocka

Morgan Wheeldon

1

Executive Summary Rare diseases are serious, chronic conditions that are debilitating or life-threatening. Estimates suggest that 1 in 12 Canadians are affected by a rare disorder. Provinces are placed in a difficult position when they must determine which drugs for rare diseases are funded through public programs. Pressure points include budget limitations, public opinion, limited evidence to justify decisions, and the absence of a universal standardized decision framework to support reimbursement decisions.

The Department of Health and Wellness (DHW), Government of Nova Scotia accepts claims for rare disease treatments and makes funding decisions regarding orphan drugs, also known as pharmaceuticals that treat rare diseases. The department faces the emergent challenge of price escalations associated with drug therapies developed to treat rare diseases.

When working with rare disease claims, the DHW navigates a complex landscape. The department must maximize resource use to address healthcare needs across the province, negotiate with pharmaceutical companies who escalate the costs of rare disease treatments to offset low market demands, make decisions based on limited clinical evidence, and account for the opinions of stakeholders and citizens. Currently, DHW does not have a formal policy in place to address this issue and decisions are made on a case-by-case basis. The goal of this project is to provide decision framework options that allow DHW to account for budgets, collect supporting evidence, and meet the healthcare needs of Nova Scotians battling rare diseases. The recommended solutions support the core responsibilities of decision-makers at DHW and promote the values of transparency, accountability, and service to the province.

A jurisdictional scan revealed that the use of multiple criteria decision analysis (MCDA) improves the decision-making process and is used throughout the world to assess the value of new medical technologies. This technique helps decision makers quantify benefits and risks associated with medical interventions and incorporates a range of criteria that account for stakeholder interests. The scan also identified three key pressure points associated with rare diseases: budget impact; political content; availability of evidence.

The Policy Hack team developed three MCDA frameworks that combat abovementioned pressure points. They also leverage drug assessments conducted by the Canadian Agency for Drugs and Technologies in Health (CADTH) to support transparent, flexible, and agile decision making. Please see the chart below for a summary of our findings. To view the full proposal, please see page 11.

We recommend implementing Solution #1 in the short-term, starting with a consultation process to finalize the MCDA framework to guide orphan drug request decisions. The finalized decision framework would be transparent, accountable, and would leverage existing resources.

In the long-term, we recommend that the province advocate for the implementation of Solution #3 among federal, provincial, and territorial partners. While a national pharmaceutical plan has proven to be difficult to establish, it is noted that a limited program focused on orphan drugs presents a number of benefits. This decision framework promotes consistent access to orphan drug therapies across Canada; moreover, a national orphan drug plan has the potential of delivering cost efficiencies through bulk purchasing and partnerships among funders.

2

Solution #1: Implement a Standard MCDA Framework Description Evaluation Criteria Pros Cons

Based on evidence and cost information supplied by CADTH and the lead physician submitting a claim to DHW.

After a claim is received, DHW and the submitting physician compare the claim against evaluation criteria. Each criterion receives a score on a scale of 1 to 7 (see next column). The combined scores of DHW and the lead physician determine if a drug is funded through the Exception Drug Fund.

x Patient eligibility x Impact of treatment x CADTH recommendation x Precedence x Budget analysis

x Leverage information compiled by national review bodies

x Customize claim decisions based on provincial standards and budgets

x Minimize costs x Reduce time required to

reach a decision

x Pharmacoeconomic analysis results in criteria and valuation that are uncontrolled

x Low number of criteria may allow for biases

x Evidence and cost data may not be available

x Does not eliminate political pressure

Solution #2: Implement an MCDA Framework that Accounts for Patient Quality of Life with A Review Panel Description Evaluation Criteria Pros Cons

Utilizes the same evidence and scoring criteria as Solution #1, also accounts for the drug’s impact on quality of life. This element is not considered during a traditional CADTH evaluation.

x Criterial from Solution #1 x Impact of technology on

quality of life x Patient’s ability to

continue daily activities x Pain management x Reduction in levels of

psychological distress x Patient’s ability to

maintain dignity x Out of pocket expense

x Greater control in the evaluation process

x Better patient representation

x Access to more up to date information.

x Cost of implementation x Longer processing times x Potential redundancy in

work x Does not eliminate

political pressure

Solution #3: Implement a National MCDA Framework and Coverage Program for Orphan Drugs Description Evaluation Criteria Pros Cons

Accounts for evidence from CADTH, quality of life, and the overall impact on the national budget. The framework promotes information sharing across the country. It also allows for shared costs for orphan drugs.

x Criteria from Solution #1 (minus CADTH)

x Clinical evidence x Drug eligibility x Treatment plan eligibility x Impact of technology on

quality of life x Urgency of treatment x Rarity of the disease x Patient’s ability to

maintain dignity x Review of clinical

evidence

x Standardizes evaluation frameworks in Canada

x Removes political and personal influences from decision making

x Avoids provincial duplication

x Mitigates political pressure associated with inconsistent decisions

x Centralizes data collection to support evidence-based decisions

x Provincial autonomy threatened

x Difficult to ensure patient representation

x Requires coordination among many stakeholders with diverse interests

x Costs associated with establishing a national framework at the federal and provincial levels

3

Table of Contents

Executive Summary ............................................................................................................... 1

Background ........................................................................................................................... 4

Summary of the Assignment ...........................................................................................................4

Summary of the Problem ................................................................................................................4

Methodology & Scope ....................................................................................................................5

Rare Diseases in Canada & Nova Scotia ...........................................................................................5

Jurisdictional Scan of Rare Disease in Canada ..................................................................................8

Current Issues & Problems ............................................................................................................ 10

Proposed Solutions ...................................................................................................................... 11

Solution 1: Develop an MCDA Framework ..................................................................................... 11

Solution 2: Develop an MCDA Framework that Accounts for Quality of Life ................................... 13

Solution 3: Develop National MCDA Framework and Orphan Drug Coverage Program ................... 14

Our Recommendation .......................................................................................................... 15

Conclusions ......................................................................................................................... 16

References........................................................................................................................... 17

4

Background Summary of the Assignment For the 2018 Policy Hack Case Competition, the team was provided with a case study from the Department of Health and Wellness (DHW). Over the past five weeks, we researched the following problem statement:

Pharmaceuticals that treat or manage rare diseases typically have high per patient costs and often limited clinical evidence. Provinces must determine which drugs for rare diseases are funded through public programs. Decisions on funding are challenging and given the multitude of perspectives to be considered in the funding decision. Nova Scotia currently has no policy for publicly funded expensive drugs for rare diseases. What can guide funding decisions for expensive drugs for rare diseases?

At this time, the DHW does not have a formal policy in place and decisions are made on a case-by-case basis. It faces the emergent challenge of price escalations associated with drug therapies developed to treat rare diseases as it makes decisions in response to requests for public coverage for drugs that are not included in the provincial formulary.

The goal of this report is to present policy options that are informed by a multi-criteria decision analysis (MCDA) framework that leverages evidence from drug assessments conducted by the Canadian Agency for Drugs and Technologies in Health (CADTH). By leveraging these assessments, Nova Scotia can implement a modified MCDA to support transparent, flexible, fair and agile decision making.

Our proposed solutions operate with the assumption that rare diseases are defined as occurring in fewer than 5 in 10,000, as per Health Canada’s proposed definition.1 In addition, DHW staff identified a high cost drug as one that is priced above $100,000. It is important to note that this report is not recommending the exclusive assessment of price to screen prescriptions for orphan drugs that are not on the provincial formulary. This figure is provided to support readers’ understanding of thresholds that create pressure points when making decisions regarding public reimbursement.

The policy recommendations support the DHW’s commitment to the ongoing improvement of Nova Scotia’s health care system through planning, legislation, resource allocation, policy and standards development, monitoring and evaluation, and information management. 2

Summary of the Problem An underlying feature of this policy challenge is that there is a price escalation associated with public reimbursement of drugs for rare diseases. Pharmaceuticals that treat rare diseases, also known as orphan drugs, are rare enough that they are considered commercially unviable under normal market conditions. The cost is high due to the significant benefit to the patient, the small number of patients yielding a high average cost of drug development per patient, and the cost of continuing investment for rare disease drug research.3

Within the context of a publicly funded medical system, devoting resources to the purchase of expensive drugs is also a decision to direct those resources away from other priorities. With orphan drugs

1 CADTH, 2016 2 Department of Health and Wellness, n.d. 3 EvaluatePharma, 2014

5

forecasted to take up to 19.1% of global prescription sales in 2020,4 balancing the benefit of new drug therapies against the escalating cost is required when deciding about public coverage of these drugs.

Methodology & Scope The team consulted a variety of sources in order to understand the issue and develop recommendations that provide short-term and long-term solutions. This investigation included:

x Consulted with DHW to document processes used to evaluate claims for expensive and/or orphan drugs;

x Examined peer reviewed literature, online research repositories about rare diseases, and websites developed by organizations that fund rare disease treatments or compile drug assessments;

x Jurisdictional scan of the policies, strategies, and frameworks in place across Canada; x Evaluated frameworks that support fair, transparent, flexible, and agile decision making; x Attended Policy Hack professional development sessions including Policy 101 and Human

Centered Design; x Consulted with the following five stakeholders:

� Angela Purcell, Kathleen Coleman, and Lara Grant at DHW; � The Honourable Kevin K. Ogilvie, former Senator, Senate of Canada; � Lisa Farrell, Nova Scotia Liaison for CADTH; � Dr. Larry Lynd, Professor and Director, Collaboration for Outcomes Research and

Evaluation (CORE), Faculty of Pharmaceutical Sciences, University of British Columbia; � Dr. Christopher R. McMaster, Professor and Head of the Department of Pharmacology,

Dalhousie University.

It is worth noting that due to the time allotted to this project, the team was not able to address each challenge associated with rare diseases and orphan drugs. One area that is absent for the report is a discussion of ethics surrounding treatment or funding options for rare diseases. While this topic was discussed with stakeholders, the team identified three key pressure points that could reasonably be addressed within the scope of this project:

x Budget impact; x Decision making is politically charged; x Evidence for rare disease drugs is limited.

To address these challenges, the team developed a concise, transparent, and flexible decision framework to support decision makers reviewing public reimbursement of expensive drugs for rare diseases. This framework is intended to trigger consultations with stakeholders to finalize an MCDA framework tailored to fit Nova Scotia’s reality.

Rare Diseases in Canada & Nova Scotia Rare diseases, also known as orphan diseases, are serious, chronic conditions that are often debilitating or life-threatening. Most have a genetic basis and patients often display symptoms early in life.

4 EvaluatePharma, 2014

6

Estimates suggest that 1 in 12 Canadians are affected by a rare disorder. Currently, 7,000 rare diseases have been identified and new conditions are discovered each year.5

Orphan drugs are used to treat rare disorders; these drugs are specifically designed in response to a public health need and do not always have economic benefits to the pharmaceutical industry.6 Because of this, pharmaceutical companies often escalate the prices of orphan drugs.

Within the context of the Canadian drug approval system, a drug must first be approved by scientists at Health Canada's Health Products and Food Branch (HPFB) and occasionally external experts supporting the assessment of drug safety, efficacy, and quality.

Next, the Canadian Agency for Drugs and Technologies in Health (CADTH) Common Drug Review (CDR) evaluates evidence to determine if an orphan drug qualifies for public reimbursement (Figure 1). The CADTH Canadian Drug Expert Committee (CDEC), a pan-Canadian advisory body made up of experts in disease management, also evaluates the drug and considers health economics. Finally, participating drug plans, like provincial initiatives in Nova Scotia, may determine whether to fund patient claims based on individual mandates, local priorities, and budget impact. 7

Figure 1. Summary of CADTH drug approval process.

Nova Scotia has an existing drug formulary that can be prescribed and funded through public reimbursement if no private coverage is available. The drugs addressed in this briefing are exceptions to the formulary; currently, they are funded under the Exception Drug Plan.

5 Ontario Ministry of Health and Long-Term Care, 2017 6 Orphanet, 2018 7 Government of Canada, 2015; CADTH, 2018

Manfacturersubmits

application for drug

Health Canada's

Health Products and Food

Branch approval

CADTH CDR Review Team

evaluates drugapplication:

Common Drug Review (CDR)

CADTH's CDEC reviews drugapplication & CDR reports (next step of

CDR)

Pan-Canadian Pharmaceutical

Alliance negotiates withmanufacturer on pricing and

conditions

Territorial/ Provincial

jurisdiction on whether to

cover drug or not

Summary of CADTH drug approval process (non-cancer)

Early meetings to discuss intent to submit application CADTH

7

There are no existing policies or frameworks in place to support requests for pharmaceuticals that are exceptions to the existing drug formulary. The process used by the province when evaluating claim requests for orphan drugs is neither standardized nor transparent (Figure 2).

Figure 2. Current decision-making process at the DHW.

Orphan drugs are funded directly by the DHW, but can also be funded through Nova Scotia Pharmacare, with an associated co-pay and deductible charged to the patient. If the drug’s price has been negotiated by the pan-Canadian Pharmaceutical Alliance (pCPA), a rebate is sought after the drug has been purchased. There are also cases where the manufacturer offers a rebate.

8

Figure 3. Current Reimbursement Procss

In Nova Scotia, budget allocations are mixed, but most orphan drugs fall under special status expenditures in the health budget. An exception subcategory of approximately $40 million covers most orphan drug expenditures. Some of these exceptions, however, include treatment protocols and criteria for drugs included on the provincial formulary, so these numbers are mixed and do not represent a formal ear marked fund or commitment to orphan drugs alone.

Jurisdictional Scan of Rare Disease in Canada Health Technology Assessment & Multiple Criteria Decision Analysis Due to limited resources, health care systems are constantly seeking new methods to fairly and efficiently allocate their resources. The use of health technology assessment (HTA) has greatly improved the process of decision making and is used throughout the world to assess the value of new medical technologies.

The complexity of health-related problems lead decision makers to under-utilize important information or omit it altogether. A recent approach for assessing the value of health care interventions is the use of multiple criteria decision analysis (MCDA). 8 This technique helps decision makers quantify benefits associated with a certain intervention as well as the risks by considering a wide range of criteria and incorporating many stakeholder interests.

8 Nicod, Elena et al. 2017

9

There are currently more than 10 countries that are either using or implementing MCDA in their decision framework for purchasing and reimbursement (P&R) of drugs.9 These frameworks vary from country to country in the number of criteria used to evaluate drugs, the scoring and weighing techniques of each criterion, and the recommendation conditions associated with the treatment. The focus of most MCDAs used in HTAs is on clinical evidence and economic efficiency analysis.

Several countries utilize a separate evaluation process that may be more appropriate for orphan drugs and allow for more flexibility and transparency when making decisions about P&R. For example, Scotland implemented the ultra-orphan medicine product (ultra-OMP) framework and the Scottish Medicine Consortium (SMC) and Patient and Clinician Engagement Committee (PACE) group. These systems avoid the challenges and pitfalls associated with frameworks used to evaluate more common disease drugs. The ultra-OMP framework has a reduced number of criteria while the SMC-PAGE group focuses on the impact of the new technology on quality of life.

The National Landscape of HTA in Canada Canada has an HTA body that evaluates all drugs that wish to enter the marketplace. The evaluation process is a rigorous assessment of the clinical data and economic impact of the drug on the patients and society.

This body will then make recommendations as to whether or not a certain drug should be covered by the insurance plans and what criteria (if any) should be included. However, there is no guarantee that a drug, even if recommended, would be included in the provincial drug plan; price and criteria are first negotiated by the Pan Canadian Pharmacological Committee and then provinces determine if a drug will be placed on the formulary.

Citizens can request coverage for drugs that are not covered through the formulary. They apply to the province and ultimately, decisions are made to approve or deny funding on a case-by-case basis. The use of a MCDA would be extremely beneficial in provinces making case-by-case reimbursement decisions, as it could help guide their decision making, while leveraging the analysis already performed by CADTH.

The Provincial Landscape of HTA At the provincial level, five provinces in Canada (BC, AB, SK, ON, and NB) are known to have established rare disease drug P&R processes. These frameworks typically assess whether a disease is rare enough to trigger these distinct drug approval processes. All of them require input from groups of experts in order to review the drug’s effectiveness. In AB, for example, treating specialists and health care professionals with related experience sit on the Rare Disease Clinical Review Panel. Decisions are made on a case-by-case basis; claims are either denied or approved with conditions (e.g. follow-ups to assess clinical outcomes with the possibility of discontinuing coverage if conditions worsen).

ON took the review process one step farther by implementing a seven-step evaluation framework. The status of submissions and decisions are transparent, and while decisions cannot be appealed, patient organizations, physicians, and manufacturers can provide feedback to the Ministry of Health and Long-Term Care, which can determine whether re-evaluation is necessary.

In BC, the Minister is responsible for making decisions based on recommendations from the Expensive Drugs for Rare Diseases (EDRD) Advisory Committee. Requests are made by physicians specific to patients 9 Short, H. et al. 2015

10

and are reviewed, unlike Ontario, on a case-by-case basis by pediatric and adult rare disease specialists, a geneticist, pharmacists, health administrators, a health economist, and an ethicist.

Current Issues & Problems The team encountered a number of pressure points while experimenting with decision frameworks. Our background research discovered the same challenges listed in strategic reports published by the research community.

The first pressure point is budget. Resources are limited and the high cost of orphan drugs direct finances away from other priorities. A report by EvaluatePharma notes that:

Within the context of a publicly funded medical system, devoting resources to the purchase of expensive drugs is also a decision to direct those resources away from other priorities. With rare diseases, it is not uncommon that expensive drugs are the only available and effective option to treat the disease, rather than manage the symptoms. This means that a decision to deny treatment may have dire consequences, including death. On the other hand, a decision to cover every request for expensive drugs may eventually lead to unsustainable budget expenditures as an increasing number of drugs with high price tags come to market.10

Decision to fund rare drugs are not based solely on resources; political contexts place pressure on decision makers. In many cases, this involves balancing societal preferences, economic evaluation, and evidence. The Canadian Society of Pharmacology and Therapeutics notes that:

Doubts have been expressed about whether standard methods of health technology assessment are suitable for the evaluation of drugs for rare diseases. Under conditions of rarity, it may be more difficult to conduct large randomized trials in order to gather adequate evidence on efficacy, and the standard methods of economic evaluation may not adequately reflect societal preferences for the treatment of serious and/or life-threatening rare diseases.11

Finally, decision makers require evidence to justify funding decisions. In many cases, clinical trials or medial evidence is limited or unavailable. The Orphanet Journal of Rare Diseases notes that:

The economic evaluation of orphan drugs is inhibited by the existence of often limited and weak clinical data at launch time. In the context of rare diseases, it may prove difficult to recruit a sufficient number of patients and medical centers in clinical trials, thus raising costs.12

The abovementioned pressure points impact allocations of resources, transparency in decision making, and evidence required to justify decisions. The team developed recommendations that address these challenges through proposed decision frameworks.

10 EvaluatePharma, 2014 11 Drummond et al., 2009 12 Simoens, 2011

11

Proposed Solutions Solution 1: Develop an MCDA Framework In this framework, the request for a drug comes from the acting physician. The evaluation would be on a case-by-case need and be funded through the Exception Drug Fund. This proposal would implement an MCDA that would leverage the current work being performed by Canada’s health technology assessment body, CADTH, while incorporating the applying physician. A panel of specialists and stakeholders would be consulted before finalizing the evaluative criteria, but the MCDA performed by the DHW could consist of the following domains:

x Eligibility of the patient; x Impact on the patient; x CADTH recommendation; x Precedence; x Budget analysis.

Each domain would have a set of criteria that would be scored on a scale of 1 to 7 (1 being lowest/worst impact, 7 being best/highest impact). We would require the applying physician to score the eligibility of the patient and the impact of the treatment on the quality of life. These would be considered with the scores of DHW as they evaluate the drug. It is important to note that these criteria were chosen based on literature reviews of HTAs and adapted to our current situation.

When looking at CADTH recommendations, we would want to ask the following questions:

x Was the treatment recommended by CADTH; x Were there any criteria accompanying the recommendation; x What were the main clinical findings; x What is the incremental cost-utility ratio (ICUR); x What effect would approval have on the healthcare budget?

Once a score (mean of the department and physician) was determined for each criterion, their sum would represent the domains relative score (divided by the best possible score) and would be multiplied by the weight (% out of 100%) of each domain to obtain a domain specific score. The sum of these scores would represent the value score of the proposed treatment.

The weight of each domain should be established by a panel of stakeholders to assure a consistent and transparent process. In most cases, a total score greater than 50% would carry a positive recommendation (approve) from the department. A final recommendation is prepared after consultation with the finance department and then it is given to the minister for final approval.

12

Pros:

x Leveraging the information already found through other review bodies x Ability to personalize the decision based on provincial standards (budget) x Minimize costs and time to reach decision

Cons:

x Choice of criteria and valuation are uncontrolled for (clinical and pharmacoeconomic analysis) x Low number of criteria may allow for biases x May not have up to date information at hand x Does not eliminate political pressure

The final score would lead to an initial department recommendation (approve or deny) which after consultation with the finance department, would lead to the preparation of a final report which goes to the minister for approval.

13

Solution 2: Develop an MCDA Framework that Accounts for Quality of Life with Panel Review This solution proposes the formation of an expert advisory committee comparable that that found in ON or BC, but that would conduct a more extensive MCDA decision making process. The basis for this framework will be the work done by CADTH coupled with additional criteria focusing on the impact on the quality of life the drug will have as well as a budget impact analysis (specific to the province). By focusing on the impact and on the quality of life, the MCDA will bring forth elements that are not typically considered or have a minor weight during the CADTH evaluation. A province specific budget analysis is required to understand the impact any decision will have on the DHW’s annual health care budget as well as future costs associated with a potential positive reimbursement decision.

Below is a list of the criteria proposed in the MCDA:

The scoring of each criterion would be done by a panel of specialists (physician, geneticist, health economist, ethicist, patient advocate) to be decided in advance following the same process as that

14

discussed above in Solution 1. The weights should also be decided before evaluation takes place and be consistent for all drug evaluations.

Pros:

x Greater control in the evaluation process x Better patient representation x Access to more up to date information

Cons:

x More expensive x Longer processing time x Potential redundancy in work x Does not eliminate political pressure

The recommendation process would follow the same steps as outlined in solution 1.

Solution 3: Develop National MCDA Framework and Orphan Drug Coverage Program A third option would be to implement a separate drug review framework from the existing CDR framework that is conducted by CADTH. This new decision framework, also conducted by CADTH, would be coupled with additional criteria focusing on the impact on the quality of life the drug will have as well as a national budget impact analysis. The framework will promote the sharing of information and costs associated with rare drugs across the country. To ensure cost efficiencies are achieved, we also propose a national orphan drug coverage system. This would give universal access to approved treatments across Canada and additionally promote cost sharing for provinces, territories, and other authorities that opt to participate in the program.

The creation of a national framework would create a system of transparent and accountable decisions across the country. Also, the collective evaluation of cost data, occurrence of disease, and treatment options allow for an analysis of trends that inform resource allocations. For instance, decision makers could create a baseline of rare drug expenditures, document expenditures over time, and identify trends in spending (e.g. % of budget is dedicated to specific drug treatments). Captured trends can be used to predict future expenditures, flag areas for research funding, or negotiate drug prices.

As proposed in our first option, consultation with stakeholders would be essential to establishing a national MCDA framework. Below is a list of potential criteria proposed:

x Eligibility of the patient; x Eligibility of the drug; x Eligibility of the treatment plan; x Eligibility for drug funding; x Impact of new technology on quality of life; x Urgency of treatment; x Rarity of the disease; x The ability to self-care or maintain independence and dignity, time for accompanied visits for

treatment, requirement for assisting the patient with personal care and support;

15

x Review of available clinical evidence; x Precedence; x Budget impact analysis.

The scoring of each criterion would be done by a panel of specialists (physician, geneticist, health economist, ethicist, patient advocate) to be decided in advance following the same process as discussed in solution 1.

Pros:

x Consistency across Canada x Removes political and personal influences from decision making x Faster implementation (avoids provincial duplication) x Mitigates political pressure associated with inconsistent decisions x Centralize data collection to support evidence based decisions

Cons:

x Provincial autonomy threatened x Difficult to ensure patient representation x Requires coordination among many institutions and bodies with varying interests and

preferences

16

Recommendation We recommend implementing Solution 1 in the short-term, starting with a consultation process to finalize an MCDA framework to guide orphan drug reimbursement decisions. The sample framework submitted here would require input from experts, patients, and other stakeholders, but is presented as an interim proposal that could be used to trigger consultation. The finalized decision framework would be transparent and would leverage existing resources. These are areas identified as priorities by DHW.

There is no way to avoid the potential variable that a patient would contact the news media and/or their political representative in this model. Additionally, this is not the most rigorous decision framework available. Despite these challenges, this option presents a practical, consistent and flexible framework that would support decision makers and could be made transparent by publishing the rubric and decisions (anonymously). While this concise MCDA scoring mechanism is not proposed as the final arbiter of reimbursement decisions, it would provide decision makers at the DHW with sound guidance to support transparent, but flexible decision making.

In the long-term, we recommend that DHW advocate the implementation of Solution 3 among federal, provincial, and territorial partners. While a national pharmaceutical plan has proven to be difficult to establish, it is proposed that a limited program focused on orphan drugs presents a number of benefits. A decision framework implemented at the federal level promotes consistent access to orphan drug therapies across Canada while promoting cost efficiencies through bulk purchasing and partnerships among funders. This approach also allows Canada to pilot a national drug program on a smaller scale while potentially mitigating the price escalation associated with orphan drug therapies. Finally, the decision-making process would be transparent, consistent, and evidence collection on orphan drugs and prior decisions would be built into the system. This option would require the buy-in of many provinces in order to achieve cost-savings, but the limited scale reduces the risks while directly addressing an emergent budget challenge.

Conclusions The health and wellbeing of all Canadians is the goal that all levels of government strive to achieve. A combination of factors has led to conditions where not all available treatments are possible. Limited resources, imperfect information, profit motives, and political pressures lead to decisions that are difficult and often without precedent.

We propose a concise and transparent decision framework that will help DHW better evaluate drug reimbursement submissions. However, a provincial framework does not address all identified pressure points across the country. A national framework that supports orphan drugs and the treatment of rare diseases is a goal that we should aim to achieve.

17

References CADTH. (2018). CADTH common drug review. Retrieved from

https://cadth.ca/about-cadth/what-we-do/products-services/cdr CADTH. (2016). Drugs for rare diseases: Evolving trends in regulatory health technology assessment

perspectives. Retrieved from https://www.cadth.ca/drugs-rare-diseases-evolving-trends-regulatory-and-health-technology-assessment-perspectives

Department of Health and Wellness. (n.d.). Provincial programs. Retrieved from

https://novascotia.ca/dhw/provincial_programs.asp Drummond, M., Evans, B., LeLorier, J., Karakiewicz, P., Martin, D., Tugwell, P., MacLeod, S. (2009).

Evidence and values: Requirements for public reimbursement of drugs for rare diseases. Canadian Society of Pharmacology and Therapeutics, 16(2). Retrieved from: http://www.cameroninstitute.com/wp-content/uploads/2014/11/020_Evidence-and-Values.pdf

EvaluatePharma. (2014, October). Orphan drug report 2014. Retrieved from

http://info.evaluategroup.com/rs/evaluatepharmaltd/images/2014OD.pdf

Government of Canada. (2015). How drugs are reviewed in Canada. Retrieved from https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/fact-sheets/drugs-reviewed-canada.html

Nicod E, Annemans L, Bucsics A, Lee A, Upadhyaya S, Facey K. (2017, March 28). HTA programme

response to the challenges of dealing with orphan medicinal products: Process evaluation in selected European countries. Retrieved from https://www.sciencedirect.com/science/article/pii/S0168851017300842

Ontario Ministry of Health and Long-Term Care. (2017). Rare Diseases Working Group Report. Retrieved

from http://www.health.gov.on.ca/en/common/ministry/publications/reports/rare_diseases_2017/ Orphanet. (2018). About orphan drugs. Retrieved from

https://www.orpha.net/consor/cgi-bin/Education_AboutOrphanDrugs.php?lng=EN Richeter T, Janoudi G., Amegatse W., Nester-Parr S. (2018). Characteristics of drugs for ultra-rare

diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR. Orphanet Journal of Rare Diseases 13(15). Retrieved from https://ojrd.biomedcentral.com/articles/10.1186/s13023-018-0762-1

Simoens, S. (2011). Pricing and reimbursement of orphan drugs: The need for more transparency.

Orphanet Journal of Rare Diseases, 6(42). Retrieved from https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-6-42

Short H, Stafinski T, Menon D. (2015). A national approach to reimbursement decision-making on drugs

for rare diseases in Canada? Insights from across the ponds. Retrieved from https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/26142357/