Access to expensive treatments and the rising cost of medicines are a global challenge. But are there good ideas and best practices for dealing with them? To answer this question, a team of guest editors from Frontiers in Pharmacology has joined forces to provide a platform for all insights and ideas. This team consists of experts from various organisations. Atse Huisman from Zorgverzekeraars Nederland and the LUMC, Denise Van Den Berg from the Treatmeds foundation, Saco De Visser from FAST), Bettina Ryll from Melanoma Patient Network Europe and Stockholm School of Economics and Sahar Barjesteh Van Waalwijk Van Doorn-Khosrovani from LUMC, CZ and the European consortium Prime-Rose.

For nine months, researchers could submit their articles for peer-review. Eventually, a selection of relevant ideas and solutions was made. In the newly published editorial , titled ‘Towards continued and affordable accessibility of innovative drugs: sustainable development and efficient use of medicines,’ the team writes about different categories of ideas and solutions published in the collection. Here we explain the main topics covered in the publication with a few examples:

1. Addressing uncertainty about clinical value
Many health insurers and Health Technology Assessment (HTA) bodies evaluate the clinical value of new drugs before reimbursement. They stress the importance of evaluating clinically relevant variables, such as survival and quality of life, rather than relying on surrogate endpoints, which often lead to uncertainty.
2. Promote (cost) effectiveness by preventing overtreatment
Treatment optimisation studies are crucial to refine drug use, improve patient outcomes and promote cost-effectiveness. Overtreatment, for example through longer treatment duration or higher doses than necessary, leads to unnecessary side effects and wastes valuable resources within healthcare.
3. Waste reduction
Reducing medicine waste saves valuable resources and minimises environmental impact. It is suggested that patients be scheduled for identical treatments on the same day, so that prepared intravenous therapy that would otherwise be discarded can be used for another patient.
4. Challenges of combination therapies
Assessing the cost-effectiveness of combination therapies is often more complex than for single technologies, due to uncertainties around synergy and the financial burden of multiple drugs. Among other things, this complicates negotiations with different pharmaceutical companies and sometimes leads to unfavourable costs per QALY, even in rich countries.
5. In-house development and manufacturing
Academic hospitals often have the expertise to develop or manufacture drugs to address unmet medical needs. Several Advanced Therapy Medicinal Products (ATMPs), for example, were originally developed in academic hospitals.
6. Repurposing precision medicine in oncology
Repurposing approved drugs for new indications, as happens within the Dutch Drug Rediscovery Protocol (DRUP), significantly reduces R&D costs. This offers alternative treatments for patients with advanced or metastatic cancer.

The publication provides an overview of current efforts to ensure access to innovative medicines and improve cost-effectiveness. There is often a knowledge gap after the introduction of new drugs, and this needs to be addressed systematically to optimise treatments. This special topic shows some specific examples that may be useful to achieve sustainable solutions. These are essential to both improve access to medicines and drive innovation. In doing so, it also shows the importance of continuing to look for opportunities that can contribute to this with specific examples.

Read on to discover more about the insights and practical solutions in this critical field: Frontiers | Editorial: Towards continued and affordable accessibility of innovative drugs: sustainable development and efficient use of medicines (frontiersin.org)