Abstract
IntroductionReimbursement decisions on orphan drugs carry significant uncertainty, and as the amount increases, so does the risk of making a wrong decision, where harms outweigh benefits. Consequently, patients often face limited access to orphan drugs. Managed access programmes (MAPs) are a mechanism for managing risk while enabling access to potentially beneficial drugs. Patients and their caregivers have expressed support for these programmes and see patient input as critical to successful implementation. However, they have yet to be systematically involved in their design.ObjectiveThe aim of this study was to co‐design with patients and caregivers a tool for the development of managed access programmes.MethodsBuilding upon established relationships with the Canadian Organization for Rare Disorders, the project team collaborated with patients and caregivers using the principles of participatory action research. Data were collected at two workshops and analysed using a thematic network approach.ResultsPatients and caregivers co‐designed a checklist comprised of six aspects of an ideal MAP relating to accountability (programme goals); governance (MAP‐specific committee oversight, patient input, international collaboration); and evidence collection (outcome measures and continuation criteria, on‐going monitoring and registries). They recognized that health‐care resources are finite and considered disease or drug eligibility criteria for deciding when to use a MAP (eg drugs treating diseases for which there are no other legitimate alternatives).ConclusionsA patient and caregiver‐designed checklist was created, which emphasized patient involvement and transparency. Further research is needed to examine the feasibility of this checklist and roles for other stakeholders.
Highlights
Reimbursement decisions on orphan drugs carry significant uncertainty, and as the amount increases, so does the risk of making a wrong decision, where harms outweigh benefits
International collaboration Given the nature of rare diseases, patients and caregivers proposed collaborating with other countries on certain Managed access programmes (MAPs), using the experiences of patients across multiple countries
If my son doesn’t make it, I’ll be fighting for the other ones” (C2, W1). This theme was apparent in both patients’ and caregivers’ enthusiasm around the workshop dedicated to the design of an ideal MAP and in their discussions about uncertainty and the difficulty it creates for decision-makers
Summary
Reimbursement decisions on orphan drugs (ie medicines for treating rare diseases affecting less than 5 in 10 000 people in the European Union1) carry significant uncertainty.[2,3] Uncertainty typically arises from a lack of high-quality information on (i) clinical benefit, (ii) value for money, (iii) potential adoption/diffusion and (iv) affordability.[4,5] The natural histories of many rare diseases, which tend to be life- threatening or severely debilitating, remain poorly understood, and high-q uality randomized clinical trials are often difficult to conduct because of small patient populations and limited validated outcome measures.[5,6]. Patients may be harmed and resources may be wasted when a treatment provided turns out to be ineffective or unsafe or when a treatment not provided turns out to be effective.[4] to manage risk while enabling access to potentially beneficial drugs, innovative ways of introducing these drugs have been developed,[7,8,9] one of which may be referred to as managed access programmes (MAPs).[4] MAPs provide patients with a drug while information needed to address uncertainties is collected to inform a definitive coverage decision. As an outcome-based arrangement, they resemble complex patient access schemes offered through NHS England.[10] In Canada, patients, caregivers and patient organizations have expressed support for MAPs. As an outcome-based arrangement, they resemble complex patient access schemes offered through NHS England.[10] In Canada, patients, caregivers and patient organizations have expressed support for MAPs They perceive their input to be critical to successful implementation, should such a policy option be adopted. They have yet to be systematically involved in their design.[4,11]
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