Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review
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REVIEW
Open Access
Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review Andrea Young1*, Devidas Menon1, Jackie Street2, Walla Al-Hertani3 and Tania Stafinski1
Abstract Background: Patients and their families have become more active in healthcare systems and research. The value of patient involvement is particularly relevant in the area of rare diseases, where patients face delayed diagnoses and limited access to effective therapies due to the high level of uncertainty in market approval and reimbursement decisions. It has been suggested that patient involvement may help to reduce some of these uncertainties. This review explored existing and proposed roles for patients, families, and patient organizations at each stage of the lifecycle of therapies for rare diseases (i.e., orphan drug lifecycle). Methods: A scoping review was conducted using methods outlined by Arksey and O’Malley. To validate the findings from the literature and identify any additional opportunities that were missed, a consultative webinar was conducted with members of the Patient and Caregiver Liaison Group of a Canadian research network. Results: Existing and proposed opportunities for involving patients, families, and patient organizations were reported throughout the orphan drug lifecycle and fell into 12 themes: research outside of clinical trials; clinical trials; patient reported outcomes measures; patient registries and biorepositories; education; advocacy and awareness; conferences and workshops; patient care and support; patient organization development; regulatory decision-making; and reimbursement decision-making. Existing opportunities were not described in sufficient detail to allow for the level of involvement to be assessed. Additionally, no information on the impact of involvement within specific opportunities was found. Based on feedback from patients and families, documentation of existing opportunities within Canada is poor. Conclusions: Opportunities for patient, family, and patient organization involvement exist throughout the orphan drug lifecycle. However, based on the information found, it is not possible to determine which opportunities would be most effective at each stage. Keywords: Rare diseases, Orphan drugs, Patient involvement, Scoping review
Background In recent years, patients have become active in healthcare systems and research, which have increasingly recognized the benefits of their involvement [1]. Such benefits include: improving the credibility and relevance of research [2]; enhancing the translation of research results into clinical practice; establishing more responsive services leading to better outcomes of care [3]; and identifying benefits and costs often missing from health technology assessments of * Correspondence: [email protected] 1 Health Technology & Policy Unit, School of Public Health, University of Alberta, Edmonton, AB, Canada Full list of author information is available at the end of the article
new therapies [4]. While recognized by all patien
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