People and nations across the world are celebrating the development of effective vaccines against coronavirus, heralding this as the triumph of academic science working in close collaboration with the biopharmaceutical industry. According to the Milken Institute Vaccine Tracker there are over 230 vaccines against Covid-19 in development, of which 38 are in human clinical trials. It is a remarkable achievement to develop such a large number of vaccines in such a compressed timescale and highlights what can be done in a moment of crisis. However, this massive effort is not just restricted to finding a vaccine but extends to a search for treatments for those already suffering from the disease. According to the US Food and Drugs Administration (FDA) 370 clinical trials for drugs against Covid have been approved, there are 270 in late stage clinical trials testing safety and efficacy, and 5 treatments have been approved for Emergency Use.
Of course, a major reason for the speed and scale of Covid vaccine and drug development has been massive government support either through direct research grants or manufacturing contracts guaranteeing a market for the products in the pipeline. For example, through the US Government’s Operation Warp Speed (OWS) Pfizer/BioNTech will receive $1.95 billion in Federal funding for delivering 100 million doses of their vaccine in the USA once approved. Similarly, the AstraZeneca/ Oxford vaccine received $1.2 billion to supply 300M doses to the USA. The total committed to OWS by October 2020 was in excess of $18 billion. This financial arrangement is one of the reasons that the Oxford/ AstraZeneca vaccine can be made available at low cost in order to be readily available in low income countries. This combination of tight public-private collaboration, a clear public health goal and targeted funding is a good example of what has been called innovation for public value or mission-oriented innovation. This approach is seen as distinct from the dominant market-based model which according to Mariana Mazzucato “…fails to meet critical medical needs, secure sufficient therapeutic advance, or offer good value for money.”
The systemic problems of the established model and the limitations of current public policy are clearly visible in the case of drugs for rare conditions. This is a field historically marked by a lack of investment and poor access to therapy. Over the last three decades governments across the world have provided financial incentives and additional market exclusivity to develop so called “orphan drugs” to address the lack of treatment for these very small patient populations. Such initiatives have been highly successful, so that by 2020 there were over 500 orphan drugs approved in the USA alone. However, this progress has come at a cost, with the average price of the top 100 US orphan drugs in 2018 being $150,900 per patient per year compared to $33,600 for the top 100 non-orphan products. These high prices are not restricted to the USA and have resulted in continued problems for many rare disease patients in getting access to often life-saving medicines. These problems are likely to be further exacerbated by a wave of new biological, cell and gene therapies that offer hope of breaking out of the decades long ‘productivity crisis’ in the sector. For example, the recently approved gene therapy, Zolgensma, for the treatment of SMA has a price tag of $2M.
These long-running problems of low productivity, lack of effective therapies, high prices and access difficulties have led to calls for the creation of new approaches to pharmaceutical innovation that attempt to change the systemic incentives within the sector. These would aim to shift the emphasis from ever increasing costs/ prices to a more sustainable and lower cost model. Proposals include novel forms of distributed and networked innovation that more fully engage patient groups and their experiences. Such approaches might be enabled by the increasing use of Big Data and artificial intelligence, as well as new forms of regulation and governance based on conditionality and “real world” evidence. One of the best examples of this involves so called drug repurposing, where an already licensed product is tested for a new therapeutic indication. This has the advantage of speeding-up development times as the broad safety profile of the drug is already known. This is far less commercially attractive as many of these products are cheap generic drugs and would only get patent protection for the new indication. However, it is notable that a significant number of the drugs in late stage clinical development against Covid-19 are repurposed products, highlighting the real potential of this approach to be used more widely.
The move towards alternative and more sustainable models of pharmaceutical innovation will almost certainly need to be accompanied by a new political economy that builds a mixed market of drug developers. In addition to the existing ecology of large biopharmaceutical companies and smaller specialist biotech firms, there is a place for not-for-profit companies, patient groups and charities, as well as social enterprises and for profit companies with a clear social mission, including B corporations such as the Italian pharmaceutical firm, Chiesi. These could co-exist alongside “Big Pharma”, serving different needs and addressing areas of market failure. Public policy should therefore go beyond just supporting basic biomedical research and incentivising the established industry in discovering new medicines and support the creation of alternative forms of collaboration, mission oriented innovation and drug developers that have a clear social purpose. The success in creating vaccines against coronavirus shows what can be done by adopting a different approach.
These ideas will be explored further by a new five year £1M Wellcome Trust funded grant that I was awarded earlier this year. The project “Orphan drugs: high prices, access to medicines and the transformation of biopharmaceutical innovation” will explore the commercial development of orphan drug products, some of the controversies around them and the policy response to high drug prices and the way in which patients are engaging with pharmaceutical innovation. In particular, a key strand of the research will examine the emergence of patient-led drug repurposing to make new medicines for very small patient groups. We are also exploring the creation of an international network of scholars looking at alternative and more sustainable models of pharmaceutical innovation. If you are interested in the project or the network please contact Paul Martin (paul.martin@sheffield.ac.uk).
