Abstract
Early access to medicines allows the prescription of a medicine before it is available in the public formulary to patients with severe or rare diseases with high unmet needs who have no authorised therapeutic alternatives available. In this context, consistent decision making is difficult, and a systematic assessment procedure could be useful to tackle complex situations and guarantee the equity of medicines’ access. A multidisciplinary panel (MP) conducted four workshops to develop an early access framework based on a reflective multiple criteria decision analysis (MCDA). A set of 12 criteria was agreed: eight quantitative (severity of disease, urgency, efficacy, safety, internal and external validity, therapeutic benefit and plausibility) and four qualitative (therapeutic alternative, existence of precedents, management impact and costs). Quantitative criteria were weighted using a five-point scale. The relative importance of quantitative criteria had mean weights from 4.7 to 3.6, showing its relevance in the decisions. The framework was tested using two case studies, and reliability was assessed by re-test. The re-test revealed no statistical differences, indicating the consistency and replicability of the framework developed. MCDA may help to structure discussions for heterogeneous treatment requests, providing predictability and robustness in decision making involving sensitive and complex situations.
Highlights
Medicines in European countries require a marketing authorisation granted to confirm quality, efficacy and safety before they can be placed on the market [1,2]
Medical urgency: The rationale for including this criterion was to assess whether a window of opportunity in prognosis was relevant for early access in order to avoid irreversible adverse consequences of the disease
Therapeutic alternatives: the use of medicines in special situations (MSS) should be limited to situations where no therapeutic alternatives or no suitable alternatives are available for the individual patient, including clinical trials
Summary
Medicines in European countries require a marketing authorisation granted to confirm quality, efficacy and safety before they can be placed on the market [1,2]. Other milestones have to be reached before the medicine is commercially available to patients, including health technology assessment (HTA) and decisions on pricing and reimbursement (P&R) [2–4]. HTA measures the added value of a new health technology compared to existing ones to formulate health policies and support decisions about coverage at a national level. HTA assesses the best evidence on relative efficacy/effectiveness, safety, relevance to the quality of life and economic aspects [2,4,5]. Post-authorisation procedures aim to guarantee the provision of treatments with added value and benefit to the maximum number of patients while ensuring public health care sustainability [5].
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