The rapid delivery of Covid-19 vaccines has increased everyone’s appreciation for biotech research and development, prompting some governments to embark on a quest for “life science sovereignty.” But to strengthen their capacity for medical innovation, policymakers will need to heed lessons from past success stories.
CAMBRIDGE – The Covid-19 pandemic changed attitudes towards public health, fiscal policy and the role of the state in the economy. Demands for greater resilience of supply chains and strategic autonomy in drug development and production gave rise to the concept of “biotechnological sovereignty”.
French President Emmanuel Macron, for example, announced an ambitious plan for France to produce at least 20 new biotherapies by 2030. With funding from France’s Public Investment Bank (BIS), his government’s La French Care initiative aims to support the local biotech ecosystem and make France a “pioneer nation in mRNA technology”. Similarly, many other governments (the Netherlands, the UK and others) are betting on their biotech sector.
This interest is welcome, but will it be enough? As the covid experience has shown, getting a handful of vaccines and therapies licensed requires hundreds of clinical trials, for new and existing compounds, and many of those trials fail. Medical innovation is expensive, and the costs and risks that it entails are often a matter of misunderstanding, both on the part of officials and citizens.
Just think of the history of RNAi (ribonucleic acid interference) therapies, a new category of drugs that target the genetic causes of disease, by using small interfering RNA (siRNA) to “turn off” harmful proteins in the source. These treatments have virtually unlimited potential, but for patients, the road between scientific possibility and real opportunity has been a long one.
The discovery of the structure and function of DNA in the 1950s was the beginning of a sustained research effort to understand the biological mechanisms underlying the process of gene expression. Building on these advances, Andrew Fire and Craig Mello discovered RNAi, or “gene silencing,” in 1998, earning them the Nobel Prize in 2006.
Fire and Mello’s discovery generated a lot of excitement about the possible use of siRNA as a new type of therapy. Pharmaceutical companies invested heavily in this new area of research, but as they tried to create therapies based on RNAi technology, they encountered a number of technical challenges. The main problem was how to get the siRNA to the right place in the human body to be effective (ie, to the organ where the disease-causing gene is expressed). The difficulties encountered in navigating this uncharted territory led many researchers and companies to lose hope.
By the early 2010s, most big pharma had completely stopped investing in this technology. Only a handful of companies (including Alnylam) stuck to their guns and ultimately managed to solve the problem of getting to the right place, using “lipid nanoparticles” as RNAi carriers. There are already four RNAi therapies approved by the US Food and Drug Administration and the European Medicines Agency, and mRNA vaccines against Covid-19 use lipid nanoparticles. But it’s important to remember that it took Alnylam 20 years and nearly $7.5 billion to get to this point.
The RNAi story offers important lessons regarding “biotechnological sovereignty”. First, success does not depend solely on scientific excellence and public support. Today the Boston metropolitan area is a world-class biotech node, home to more than a thousand biotech-related companies. But creating that ecosystem took fifty years, from Biogen’s founding in the 1970s.
Boston’s biotech ecosystem owes its growth to the interplay of several forces. One undoubted factor was the presence of top-tier biomedical expertise within Harvard University and MIT, but the availability of interdisciplinary skills in engineering, business, finance, computing, and data science was also essential. So was the young industry’s proximity to some of the world’s largest research hospitals. Collaboration between the scientific community and the medical community was crucial for clinical development. Finally, capitalists and investors in Boston and New York provided the necessary initial financing.
A second lesson has to do with “sovereignty”, a concept that can be problematic, since it implies a nationalist orientation. Indeed, for a biotech ecosystem to work, it has to be open and internationally oriented, in order to harness scientific expertise, talent and capital from around the world. It is no accident that many of the largest European and Japanese pharmaceutical companies (Sanofi, Novartis, Takeda and Ipsen) have invested in facilities in Boston.
To facilitate the international growth of local companies, governments have to adopt policies favorable to attracting human and financial capital from abroad. The UK seems to be aware of this: through the UK Biobank, a leading biorepository for biomedical research, the country uses data from its National Health Service (NHS) to establish partnerships with companies and researchers around the world, with the ultimate goal of developing new drugs.
Third, medical innovation demands a considerable provision of funds from the public and private sectors. In this, Europe continues to lag behind the United States. Much more funding is needed for Europe to catch up and (perhaps more importantly) for China not to overtake it in the global biotech race.
Finally, to ensure financial sustainability and a continuous investment cycle, market incentives and public incentives must be aligned, so as to reward innovation. In this, too, Europe is far behind the United States. The great fragmentation of the European market delays the diffusion of innovations and therefore reduces the profitability of investments. The combination of limited growth opportunities and the commercial risks associated with unfavorable access to markets creates many disincentives against investing in research capabilities and clinical trials.
The creation of a more unified European market, in which new innovations follow an agile and predictable evaluation process, can correct these problems and create a virtuous circle of investment and growth. But for that, a change of mentality will be necessary. Decision-makers have to start seeing biotechnological innovation as a strategic investment instead of a health cost.
They also need to improve access to new innovations, as the NHS has done with its population health management initiative, in which patient records are used to provide early and broad access to new treatments. Many other innovative solutions exist, but guiding them to adoption requires more dialogue and a new social pact between the biotech sector, governments and the public.
Philip A Sharp
Professor of Biology at MIT, he is the director of the scientific advisory committee of Alnylam Pharmaceuticals and a Nobel Prize winner in Physiology and Medicine.
Julien Patris
He is Policy Director for International Markets and Country Manager for Belgium and Luxembourg at Alnylam Pharmaceuticals.
Translation: Esteban Flamini
Copyright: Project Syndicate 1995 – 2022