Pressures to increase productivity and maintain investment returns has led to Pharmaceutical companies filling their early stage research and development (R&D) pipelines with more targeted niche products that address high unmet need. It is because of this more targeted approach with smaller patient populations that a new challenge is emerging: how to ensure all patients who can substantially benefit from these new treatments have access.
In an ideal world effective and innovative medicines would displace established less effective medicines as they slot seamlessly into health systems offering a significant and easily measurable clinical and financial benefit. Goals of industry and Health Technology Assessment (HTA) agencies would be in perfect alignment as appropriate access is granted, providing companies with sufficient returns to feed future R&D, and health systems with cost savings and improved outcomes to help manage spend on an ageing population with complex needs.
The reality is far from this ideal and the common goal of improving patient outcomes is currently overshadowed by short term financial pressures on both sides. Today's health system is facing well documented challenge, expenditure is increasing faster than GDP - indeed across Europe it's projected to reach between 12 and 15 per cent of GDP by 2030. Innovative new drugs are increasing expenditure and adding tension to the already challenged health system as they:
- are often additive rather than replacing existing drugs. The increasing use of combination therapies for cancer is a good example
- launch with a higher price tag. With a decreasing patient population, a higher price tag may be necessary to generate the required revenues to cover R&D expenditure (Alexion's Soliris is priced at $490,000 per year per patient in the US!)
- more frequently offer improvement in outcomes than provide a demonstrable cost saving
The net result when governments and HTA agencies are considering approval of new treatments is a requirement for pharmaceutical companies to:
- 'prove that it works'
AND - 'prove that it's worth it' (i.e. deliver a cost saving)
This mentality has led to slower uptake of innovation as industry struggles with the 'prove that it's worth it' in more targeted patient populations at higher price points. With the increasing financial pressure, gaining access for orphan and ultra-orphan drugs across Europe is becoming increasingly difficult. For example it is estimated that up to 30 per cent of rare disease patients in Europe are unable to access regulatory approved therapies. So the key question is whose responsibility is it to ensure access? It could be argued that industries priority is to invest in good science with the greatest potential to address unmet need and to build a strong enough evidence base for HTA agency approval. However, the interdependency of challenges facing industry and health systems suggests shared ownership of this challenge and an amended approach to the new R&D pipeline is needed.
One example of shared ownership is the UK's new Pharmaceutical Price Regulation Scheme in which the Department of Health and industry have committed to control spending while improving access to and uptake of effective innovation. However, industry is looking for an environment that encourages shared ownership before the HTA takes place, an environment that may be created by early access schemes currently in development. These schemes have the potential to enable early and controlled adoption of drugs to small identifiable groups of patients to increase the patent period, facilitate evidence development and improve outcomes for patients.
Success relies on a number of complex factors. Firstly, better tools e.g. electronic health records and companion diagnostics are required to identify target patients which HTA agencies are willing to grant early access to. Secondly, once in the real world of early launch, there is a need to establish fair and transparent evidence requirements for assessment and better tools to collect and interpret data, for example through patient reported outcomes (PROMs) so that a richer understanding of value (clinical and economic) can be achieved.
Successful implementation of early access schemes and the required analytical capabilities (on both sides) could create a more collaborate access model and help to ensure the maximum appropriate use of innovate medicines to improve patient outcomes.
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