Accelerated Access to Innovative Medicines for Patients in Need

Abstract

There is broad agreement among health-care stakeholders that more must be done to ensure that patients have timely access to new and innovative medicines. Assuming that industry will continue to develop such medicines at a sustainable rate, regulators and payers become the gatekeepers. Regulators, starting in the late 1980s/early 1990s, and, more recently, payers have implemented a variety of early-access pathways or initiatives, and this practice is continuing even today. This article describes the specific approaches that have been taken in four economically developed regions, reviews their success rates, and suggests possible new directions.

The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal

“…11 These new regulatory pathways can also improve competitiveness; shortened product development times prior to licensing can be very beneficial and more appealing to emerging small and medium-sized enterprises.…”

“…Such a 'life-cycle approach' to acquiring and (re)assessing evidence will consider the basis of decision-making in the following stages of a product's life cycle: development, licensing, reimbursement, monitoring/post-licence evidence and drug utilisation. 11 Importantly, the approach encompasses both the authorised indication and the potential further therapeutic uses of the medicine. The EMA changed the name of the pilot project from 'adaptive licensing' to 'adaptive pathways' to better reflect the idea of a lifespan approach.…”

Cross-Comparison of Cancer Drug Approvals at Three International Regulatory Agencies

“…Before accelerated approval mechanisms can be implemented in a meaningful way, a number of issues have to be addressed, including data quality, completeness, and clear guidelines for post-marketing surveillance of drugs approved through such pathways 6 . In the present study, the drug with the shortest tta was cabazitaxel.…”

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“…This is partly linked to changes in the regulatory landscape in which regulators and payers have produced new approaches in an effort to provide patients with timely access to new medicines. For example, the European Medicines Agency has introduced the "Adaptive Pathways" approach that is open to interventions in the early stages of development [1,2]. Inevitably, this means that coverage decisions in many different jurisdictions and health care systems are being made when there may be substantial uncertainty surrounding the technology's effectiveness, costeffectiveness, and potential for harm.…”