Mapping Patient and Caregiver Involvement across the Orphan Drug Lifecycle and in Programs that Provide Access to Promising Therapies While Ensuring Prudent use of Scarce Health Care Resources

  • Author / Creator
    Dunn, Andrea L
  • Patients with rare diseases face significant challenges in accessing orphan drugs in Canada because of the uncertainties that decision-makers face when deciding whether or not to provide coverage for a new drug. While all coverage decisions are made under uncertainty, this uncertainty is often greater for orphan drugs due to the poorly understood natural histories of many rare diseases and the lack of rigorous clinical trials. To address these challenges, Canada’s Minister of Health announced the creation of a Canadian Orphan Drug Regulatory Framework, which aims to provide Canadians with better, timelier access to orphan drugs, and encourage and facilitate clinical research on rare diseases. Still under development, a draft of the Framework indicates that patient involvement will be incorporated into several stages of the orphan drug life cycle. Although this involvement is emphasized as a key component of the Framework, the exact ways in which patients will be involved has not yet been described. The purpose of this thesis was to explore opportunities for patient and caregiver involvement through the orphan drug life cycle and to identify how they believe they should be involved. It is comprised of 3 papers. The first paper contains the results of a scoping review of existing and proposed opportunities for patients, caregivers and patient organizations at each stage of the orphan drug life cycle in Canada and internationally. The review demonstrated a wide variety of opportunities that fell into 12 themes: research, clinical trials, patient reported outcome measures, patient registries and biorepositories, stakeholder relationships and collaborations, education and awareness, advocacy, conferences and workshops, patient care and support, patient organization development, regulatory decision-making, and reimbursement decision-making. During a consultative webinar in which the results of the scoping review were described, Canadian patients and caregivers demonstrated a willingness to take advantage of these opportunities and become more involved in the life cycle. The second paper focuses on how patients and caregivers believe they should be involved, describing the results of 5 workshops with various stakeholders, including patients, caregivers, physicians, and representatives from government, pharmaceutical companies, and patient organizations, and of a deliberative session and webinar with patients and caregivers alone. The results revealed significant patient and caregiver interest in the ways in which they could be involved to help improve coverage decision-making. Participants recognized the need for prudent decision-making, but emphasized that a lack of evidence of effectiveness is not the same as evidence of a lack of effectiveness. They believed that managed access programs (MAPs) are a reasonable means of reducing uncertainty in coverage decision-making while providing patients with timely access to potentially effective orphan drugs. Participants also felt that beyond participating in MAPs, they should be involved in the design of the programs. Building on the patient and caregiver interest in MAPs demonstrated in the results of the second paper, the third paper focuses on a patient and caregiver-designed framework for MAPs. It describes the results of two workshops with patients and caregivers from across Canada. Through the workshops it was discovered that patient and caregiver interest in MAPs is motivated by their lack of trust in decision-makers and physicians to ensure they receive access to effective orphan drugs, desperation to gain access to potentially effective drugs when no alternatives exist, and hope that access to effective orphan drugs can be improved in Canada. Participants identified 7 aspects of an ideal MAP: program goals, disease/drug priorities, program-specific governing committees, incorporation of individual patient input, learning from other countries, ongoing monitoring and registries, and appropriate outcome measures and stopping criteria. They emphasized the need for patient involvement and transparency in all aspects of the program.

  • Subjects / Keywords
  • Graduation date
    Fall 2016
  • Type of Item
  • Degree
    Master of Science
  • DOI
  • License
    This thesis is made available by the University of Alberta Libraries with permission of the copyright owner solely for non-commercial purposes. This thesis, or any portion thereof, may not otherwise be copied or reproduced without the written consent of the copyright owner, except to the extent permitted by Canadian copyright law.