Clinical Research - Competitive advantage through translational research

Competitive advantage through translational research

Gregory Venters looks at a future model for the UK that could help industry give the translational – or ‘bench to bedside’ – aspect of the pipeline the full attention it deserves

During the 20th century, medical research has seen enormous advances in basic science discovery, as typified by the sequencing of the human genome, yet we have not witnessed a corresponding success in the widespread application of these advances into medical practice. While the UK continues to be a recognised world leader in medical science research, bridging the gap between the discoveries achieved at the ‘bench’, and interventions and therapies at the ‘bedside’, remains a challenge. This gap has an unwelcome effect not only on patient care but, more broadly, on the UK’s economic development.

The ongoing challenge for the industry to deliver innovative medicines highlights the persistence of a drug development model that has not adapted to changes in science, public perception of the pharmaceutical industry, or the marketplace. Until now, high profit margins have shielded drug development from the kind of pressures that would prompt reform. The spate of mergers in the past decade among many of the largest drug companies has exacerbated the problem by adding the complexity that mergers bring to an already inefficient and outmoded process. As a result, the industry has not given the translational or ‘bench to bedside’ aspect of the pipeline the full attention it deserves.

Industry’s approach

Over the years, industry collaboration with academia has figured prominently in numerous translational successes, including angioplasty, recombinant growth hormone, stenting for coronary artery disease, and many new drug treatments and medical devices. However, the drug discovery and development process pursued by the biotechnology and pharmaceutical industries has come under criticism. The main objections have focused on the time required to develop products fit for human use, inefficiencies in the process, and expense. Current conventional wisdom states that up to 15 years is required to take an original idea to product launch, that about one billion US dollars will be invested, and that only one in every 5,000 to 10,000 compounds synthesised will actually become a commercial product. It has been proposed, however, that seven years is a reasonable target timeframe and a success rate of 1 in 250 compounds is feasible. Attacking these challenges forms a core objective of translational medicine.

Universities have become a familiar component in the early stages of research, identifying and characterising biological targets. Pharmaceutical companies largely divested early stage drug discovery activities, with the resulting gap filled by the universities, and (a shrinking pool of) biotechnology companies. However, some recognise there are opportunities for still deeper industrial collaborations, with greater sharing of research goals and direction. Some of the best collaborations are found not in the ‘core’ of the traditional pathway, such as drug discovery, but in supporting and enabling activities, in areas such as bio-marker identification and development, and imaging science.

A model for the UK

One example that may prove to be a future model for the UK is the Drug Discovery and Development Centre (DDC) in Germany. With the help of the pharmaceutical industry, the Max Planck Institute and Max Planck Innovation set up the DDC, an incubator in two parts: the Lead Discovery Centre and the Development Company.

The Lead Discovery Centre focuses on taking hits along the development pathway to the point of a well-proven lead. Because the Max Planck Institute is itself only allowed under German law to perform basic research activities, the Lead Discovery Centre fulfils the translational gap by moving basic science to a more developed, commercial point. The second part of the incubator, the Development Company, which launched in 2009, will take a lead and develop it to the point of Phase II clinical trials. The Development Company is intended to be a technology transfer entity with responsibility for managing the licensing of results to an established firm, engineering the spin-out as a stand-alone enterprise, or providing co-investment of a viable project result.

For the UK, while infrastructure sharing for translation can be actively encouraged, it may also prove beneficial (especially for the less commercially viable disease areas) to place resources like the DDC partly outside of the usual higher education institution (HEI) structures. The DDC presents a good basis for the UK pharmaceutical sector and HEIs to establish together a similar ‘neutral’ forum for the development of collaboration focused on translation.

Opportunities for industry

Academia’s transforming outlook toward its role in translational research represents one of the most promising areas of opportunity for industry. Until now, the relationship between academia and industry has been clouded by the fact that industry tended to take the lion’s share of profits by investing in later-stage research. One example of this has been to buy IP outright and develop it for human (commercial) applicability.

What has begun to change is academia’s view toward the IP it develops alone. Increasingly, the universities strive to maintain control of their IP for as far along the development pathway as possible. If a university is able to carry forward a project as far as pre-clinical trials, the value of the IP is significantly enhanced, and their bargaining position with industry equally so. The result is a stake in the commercial outcome of the research. With the two sectors participating more as equals with both sharing in the outcome. This degree of competitiveness between academia bringing IP further forward to enhance its bargaining power and industry seeking to balance investment and risk will be a principle driver in shaping the future relationship between the two parties.

Change in approach to IP helps facilitate the opportunity for research and technology management to move even further towards an open innovation model. Historically, pharmaceutical companies have expanded their R&D efforts outside their closed model by contracting with academia and, more recently, with smaller biotechnology companies. This has been done on a contract-by-contract basis and has been utilised to support the company’s existing business model; that is, targets may be sought from a university researcher to supplement the pipeline in a focus disease category.

Bayer and Eli Lilly have been early movers: Bayer Schering by supporting a collaborative approach to discovering and translating ideas from basic research into new drugs through its ‘Targets to Novel Drugs’ initiative. What an increasingly open innovation model can deliver in future is the possibility to move beyond existing research focus and business models and create entirely new business models. As part of its goal to become part of a Fully Integrated Pharmaceutical Network (FIPNet), Lilly, for example, not only seeks to collaborate on innovations to fill its existing pipelines but also to expand outside its core areas.

The increasing demand to be commercially astute will also steer universities toward more effective project management. Project managers provide the tools to accurately capture data and information acquired during project work, liaise between team members and stakeholders, and maintain all records, among other things. All of these activities are essential when working with a team of people from different disciplines and cultures who may have very specific backgrounds and skills and who do not otherwise work in tight project team environments. This approach to project management in medical research generally is quite foreign to most academic researchers, but it is essential for translational research to be successful.

Companies must be aggressive

Although academia is proving itself to be capable of moving further into the translational aspects of the development path, industry and academia will need each other to successfully bridge the gap. The opportunity for industry is to recognise and pursue the possibility of establishing a new ‘front-end’ to the early discovery phase of drug development where, by working closely with academia, new therapeutic entities can be developed and taken to market. The most nimble and aggressive players in the industry can achieve competitive advantage by developing the collaborative business model that establishes that ‘front-end.’

That model would need to address the challenges and weaknesses that have existed until now in past partnerships between academia and industry. The best business model would take into account the necessity for strict, industry-level project management capability within academia, implementation of standard operating guidelines and procedures, development of robust assay systems, and the provision for data reporting and control at the highest standard. Industry, for its part, must allow academia the freedom to pursue innovative research without applying its normal control mechanisms too rigidly. Importantly, the model must stipulate how all participants in the research will be rewarded for effort and success.

Advantage may be achieved for those within industry who define the business model most appropriate for collaboration with academia. Late-stage entry into the development process and investment into proven science are no longer valid alternatives for the large biotechnology and pharmaceutical companies. Instead, they now have the opportunity to identify the appropriate level of risk to assume in development and define the approach to collaboration that rewards both parties fairly.

As the discipline of translational medicine evolves and matures, academia and industry must increase the pace with which they pursue collaboration that delivers meaningful results. In our discussions with researchers in both academia and industry, we identified a number of factors that should improve the chances for successful collaboration (see Table 1).

These five factors constitute the most critical issues highlighted by those we interviewed. Industry players would find competitive advantage by responding to the opportunities translational research offers, especially within the evolving role of academic medical research. Collaboration has been shown to be the key theme in surmounting the challenge, and the five factors we’ve outlined form the core of what future collaborations should look like. Above all, by establishing mutual respect among all researchers engaged in collaboration between academia and industry, the path is prepared for identifying and pursuing potentially exciting new medical challenges in translational research.

Table 1: Five pointers to successful collaboration

1. With respect to drug discovery and similar core research, pharma will need to adapt itself culturally to take on a much more direct (and open) collaboration with academia, especially with respect to how academic research activity is perceived and how the different skill sets of the two can best compliment each other.
2. Opportunities abound for pharma to participate in research that is ancillary to core developmental research but which creates or improves the tools required to promote translation (eg bio-marker identification).
3. As academia pursues development further down the pathway, pharma will need to reassess its position on how it manages risk and supports research at earlier stages than traditionally preferred, which will require companies to consider new ways of sharing commercial rewards.
4. In particular, as the lines along the developmental pathway become crossed between academia and industry, the issue of intellectual property ownership will become critical, offering an opportunity to those companies that prove most flexible and innovative in their approach to collaboration with academia.
5. Collaboration must include the capability to bring together the right parties to form optimal project-specific partnerships, and the overall approach must incorporate a disciplined process for managing the portfolio of opportunities to the potentially divergent benefit of both parties.

Gregory Venters is a principal in Arthur D Little’s Technology & Innovation Management Practice venters.gregory@adlittle.com