The Irish Government has been engaged in a Research Prioritisation Exercise over the last year seeking to develop a strategy to best deploy resources in publicly funded research and development. Whilst I’m not part of this exercise, I suspect that there has been discussion in relation to how the state would support new therapeutics. What follows are my views in respect of broad international trends in therapeutic discovery and early development and how the Irish university sector might respond.
Large pharma has had a difficult decade. Despite launching many blockbuster drugs in the 90s and investing hugely in R&D, the industry has largely failed to replace these high earners as they rapidly reach the end of their patent life and replaced by generics. R&D expenditure peaked at around $37Bn in 2008, yet the number of New Molecular Entities (NME) has, at best, remained constant.
The trend in the response of large pharma companies over the last three years has been to abandon early stage R&D and focus on acquiring or partnering therapeutics from small biotech & pharma companies. The strong preference for pharma companies is to focus on those therapeutics that have shown efficacy in patients (Phase IIa or later) and the percentage of deals occurring for Phase I and earlier NMEs is low at 5% or less of total deal volume at present. However, the level of deal activity seen for these earlier stage therapeutics is growing for two reasons. Firstly, the available pool of unpartnered/non-acquired Phase IIa and later NMEs is diminishing. Secondly, as pharma has divested itself of internal discovery and early development capability, it is increasingly driven to source this externally.
The Growing Role of the University in NME Development
In a recent review of the source of 252 NMEs approved by the FDA from 1998-2007 (Kneller, Nat. Rev. Drug Disc. 9:867), the author observed that “of the 252 drugs studied …. universities that transferred their discoveries to biotechnology companies (U→b) were attributed 40.3 (16%), and universities that transferred their discoveries to pharmaceutical companies (U→P) were attributed 20.4 (8%)”. Furthermore, the author observed that “The data indicate that drugs initially discovered in biotechnology companies or universities accounted for approximately half of the scientifically innovative drugs approved, as well as half of those that responded to unmet medical needs”.
Large pharma have recognised this dynamic. In a move to capitalise on the fountain of new therapeutics knowledge, teaching hospital/clinical infrastructure and associated spinouts and small companies, pharma has begun to move to formalise large, multi-year alliances with institutions (Scudellari, Nat. Med. 17:3). For example, Pfizer announced a $85M, 5 year alliance with UCSF in November. AstraZeneca has a 2 year deal with U Penn and J&J have a 5 year deal with MIT – all to pursue the discovery, development and early clinical evaluation of NMEs. This trend demonstrates a desire on the part of pharma MNCs to create longer term collaborations with universities to gain from their drug discovery capability.
How Irish Universities Might Align With International Trends
My view is that there is a wealth of capability and technology platforms available across the University sector in Ireland after successive rounds of PRTLI, SFI and other state funding initiatives. What is lacking is an overarching network that allows consolidation of these resources and simplifies access by pharma and biotech companies. One model might be that of Fraunhofer in Germany. The benefits of such a shared services framework are:
i) a formalised and facilitated R&D network to support indigenous therapeutic spinouts and existing companies in the space
ii) a critical adjacency to encourage backwards integration of earlier stage R&D efforts from pharma/biotech MNCs and associated FDI
iii) a compelling base that can be leveraged to obtain philanthropic/charity/FP7 and other non-exchequer therapeutic discovery/development investment
iv) an engine to grow wealth and job creation in the therapeutics space
The specifics of how such an initiative would be structured would have to be negotiated between relevant stakeholders, of course. However, the time may be ripe to consider it.