Session 5.4 – Prioritizing Orphan and Expensive Drugs

Thursday October 30 15:30-17:00

Understanding the Factors Influencing Variation in Priority Setting for Cancer Drug Coverage Across Canadian Provinces

(Abstract 0012) Roger Chafe1, Peter Coyte1, Mark Dobrow2 1University of Toronto, Canada, 2Cancer Care Ontario, Canada Background: Questions concerning the coverage and funding of new cancer drugs are among the most difficult faced by health care decision makers. In Canada, provincial governments have ultimate responsibility for choosing which drugs they will cover and despite usually drawing on the same scientific evidence base, inter-provincial variation exists in both the processes used for making coverage decisions and in access to new cancer drugs. Canada is currently establishing a national centralized review process specifically to allow for a shared priority setting process for cancer drugs in the expectation that this will lessen the level of variation. Objectives: This presentation will present some of the preliminary findings from a three-stage, three-year research project examining variation in priority setting for cancer drugs. Methods: These findings will be based on case studies examining the factors influencing the coverage or non-coverage of four cancer drugs in three Canadian provinces. Results: The presentation will highlight the influence of contextual factors in the adoption of drugs, even when decision makers are drawing on the same evidence-base and are participating in shared priority setting processes. Policy Implications: Variations in priority setting exercises and in resulting coverage decisions are difficult for the public to understand. Better understanding the factors which drive variation will likely provide greater transparency into the real reasons why variations occur. Greater understanding of the impact contextual factors play in priority setting will also hopeful allow for recommendations to improve priority setting for new cancer drugs.

Valuing Rarity: A Case Study

(Abstract 0019)

Zahava Rosenberg-Yunger1, Abdallah Daar2, Halla Thorstiendottir3, Douglas Martin4
1Department of Health Policy, Management and Evaluation and the Joint Centre for Bioethics, University of Toronto, Canada, 2McLaughlin-Rotman Centre for Global Health at University Health Network/University of Toronto and McLaughlin Centre for Molecular Medicine, Canada, 3McLaughlin-Rotman Centre for Global Health at University Health Network/University of Toronto, Canada, 4Department of Health Policy, Management and Evaluation and the Joint Centre for Bioethics, University of Toronto, Canada

Background: Orphan drug (OD) reimbursement committees are challenged with making recommendations based on insufficient evidence due to the small population size affected by the disease.

Objective: To describe PS decisions regarding the drug reimbursement recommendations for two expensive ODs: Cerezyme and Fabrazyme.

Methods: We conducted a qualitative case study using 23 key informants (i.e. committee members, patient groups and industry) to describe PS of two ODs at three international drug advisory committees (Israeli Basket Committee; Australia’s Pharmaceutical Benefits Advisory Committee; & Canadian Expert Drug Advisory Committee).

Results: OD reimbursement recommendations in the committees were based on similar criteria and publicized through various methods (including media, Internet, and publications). Clinical evidence, cost, and efficacy were used and considered relevant decision-making values. Many committee members expressed preference for the current process but were concerned with fairness and desired an improved process for OD. Other informants suggested the inclusion of other relevant values (e.g., drug’s life-saving ability) would improve the PS process.

Policy Implications: Policy makers and recommendation committees need to better identify and incorporate relevant values when making PS decisions. This will enable fair and legitimate decisions despite insufficient evidence because PS for drugs is not solely a technical process (involving purely the weighing of evidence).

Market Access for Orphan Drugs: Managing Scarce Resources in a Regionalised System

(Abstract 0085)

Claudio Jommi, Simona Bartoli
Università L. Bocconi, Italy

Background: Diverse policies have been implemented to enhance R&D for rare diseases. This has noticeably increased the number of orphan drugs designations. However, once the orphan drug is available, some hurdles (diagnosis, clinical pathways and organisational aspects, costs) can limit patients access. In addition, equity issues arise when access is influenced by regional policies, as it happens in the Italian National Health Service.

Objective: This paper aims to compare Italian regional policies on rare diseases and orphan drugs. The analysis is focused on regional and local drug formularies management and orphan drugs funding. These policies are put into the general context of rare diseases management by regions.

Methods: We selected eleven regions according to size of population and institutional arrangements of health care system (e.g. regions can either split providers from purchasers or adopt a vertically integrated model, with important effects on health care funding). Regional policies are scrutinised according to regional documents, grey literature and a semi-structured interviews (validated through key-informant) administered to regional medicines officers.

Results: Regions show important variability in the way they manage drug formularies and finance orphan drugs. This could have an important implication on equity: e.g. the average length of time between drugs availability and its inclusion into regional formulary could undermine the access to new drugs.

Policy Implications: The analysis allows identifying key factors facilitating or delaying market access to orphan drugs and could address actions aimed at facing equity issues, taking regional best practices as a benchmark.

The Process and Politics of Prioritising a New Orphan Drug

(Abstract 0145)

Jonathan Howell
West Midlands Specialised Commissioning Team, NHS, United Kingdom

The very high cost orphan drugs are challenging prioritisation processes and creating ethical problems for the funders of healthcare.

This presentation is about using a carefully structured approach so as to support a local commissioning decision on an expensive orphan drug for a rare genetic condition. This is also about the politics around following through a rational process for a drug that appears to work but is clearly not cost effective and may conflict with previous national policy. Are there any special criteria to justify funding? If not, can a reasonable process be followed that resists legal challenge?

The history of the commissioning of enzyme replacement therapies in the UK is used to show how the earlier lessons were worked through into a policy that could be used by local healthcare commissioners in an English health region. The links with other local decisions and national policies are explored as well as the political and communications issues behind an orphan drug policy.