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Abstract
Medicines have made an appreciable contribution to improving health. However, even high-income countries are struggling to fund new premium-priced medicines. This will grow necessitating the development of new models to optimize their use. The objective is to review case histories among health authorities to improve the utilization and expenditure on new medicines. Subsequently, use these to develop exemplar models and outline their implications. A number of issues and challenges were identified from the case histories. These included the low number of new medicines seen as innovative alongside increasing requested prices for their reimbursement, especially for oncology, orphan diseases, diabetes and HCV. Proposed models center on the three pillars of pre-, peri- and post-launch including critical drug evaluation, as well as multi-criteria models for valuing medicines for orphan diseases alongside potentially capping pharmaceutical expenditure. In conclusion, the proposed models involving all key stakeholder groups are critical for the sustainability of healthcare systems or enhancing universal access. The models should help stimulate debate as well as restore trust between key stakeholder groups.
Financial & competing interests disclosure
There are no conflicts of interest from any author. However, the majority of authors are employed by ministries of health, health authorities and health insurance companies or are advisers to them. The content of the paper and the conclusions though are those of each author and may not necessarily reflect those of the organization that employs them. This work was in part supported by grants from the Karolinska Institutet, Sweden. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
No writing assistance was utilized in the production of this manuscript.
Medicines have made an appreciable contribution to improving healthcare. However, even high-income countries are now struggling to fund new premium-priced medicines.
The number of struggling countries will grow unless addressed driven by factors including changing demographics and the continual launch of new premium-priced medicines often with limited health gain versus current standards.
High prices are a particular issue for new medicines for cancer and orphan diseases. This includes trastuzumab emtansine and ivacaftor.
Another challenge health authorities are facing is the launch of new effective but high-priced medicines for hepatitis C as well as new premium-priced medicines for patients with type 2 diabetes mellitus, given the prevalence of both diseases.
Professional bodies including physicians and health authorities have instigated measures including educational activities, prescribing restrictions and registries where they have been concerned with the effectiveness and/or safety of new medicines in clinical practice, especially when the population is older with greater co-morbidities than patients enrolled into Phase III clinical trials.
Health authorities have also instigated a number of measures to address these challenges including developing new models to optimize the managed entry of new medicines. These start pre-launch with horizon scanning and budgeting. Peri-launch activities include the critical evaluation of the role, value and place in therapy of new medicines with post-launch activities including evaluating prescribing against guidelines and quality indicators as well as addressing concerns with interface management where these exist. Other activities to optimize pharmaceutical budgets include expenditure caps with trade-offs between prices of new and established medicines as well as the development of multi-criteria matrices to standardize the valuation of new medicines for orphan diseases.
Clinical pharmacologists, clinical pharmacists, health economists, biostatisticians and other healthcare professionals will need to play a key role in improving the critical drug evaluation skills of physicians to optimize the use of new medicines in the future. Health systems will also need to invest in continuous professional development for their long-term sustainability.
Notes
Adapted from Citation[139,141,159,160].