The importance of health technology assessment to ensure an efficient and equitable access to high-cost therapies

Resumen

According to the World Health Organization, the key goal of health systems is to improve the average level of the population health and to reduce health inequalities in the population. In order to realise this goal, health system decision-makers need to decide which health technologies to invest in and which not to. Health technology assessment (HTA) provides a framework fer decision-makers to make resource allocation and priority setting decisions based on the existing evidence. Considering the increasingly tight healthcare budgets and the rich pipeline of high-cost, innovative drugs very likely coming to market in the next few years, it is crucial that a robust and transparent HTA process be undertaken to assess these drugs, evaluating all aspects of the disease and treatment and involving all stakeholders affected. We conducted three standalone projects analysing different aspects of recently launched innovative drugs. In our first study, we combined high-quality sources of evidence, both from the real-world and randomised controlled tria Is, to evaluate the cost-effectiveness of carfilzomib fer treating multiple myeloma patients. By harnessing the power of these data sources, we demonstrated that the reimbursement of carfilzomib is likely to representan efficient allocation of existing resources. Despite the availability of good sources of evidence, the real-world distribution and use of innovative drugs may not be efficient nor fair, and this is what we demonstrated with our two other studies. Firstly, we showed that significant inequalities exist in the distribution of anti-osteoporosis drugs in primary care in England. The most striking case was that of denosumab, a high-cost innovative treatment, with prescriptions disproportionately concentrated among the least deprived. Substantial inequalities also exist in the use of insulin glargine biosimilars in primary care in England, even though guidelines and initiatives to promete the use of biosimilars have been put in place. In this study we observed that the real-world savings realised from the use of insulin glargine biosimilars represents a small proportion compared with what could have been achieved should their uptake had been higher. The results of these two studies, therefere, show that resource allocation may not be efficient nor fair in the real world, and similar situations are likely to exist in other disease areas. In summary, even though in many cases ample evidence exists to assist healthcare authorities making resource allocation decisions, we have demonstrated that resource allocation in the real world may not be optimal. We hope that our results provide useful insights so that healthcare authorities, in England but also in other countries, adopt measures that ensure the key objective of our healthcare systems is achieved: to improve the average level of the population health and to reduce health inequalities in the population.