pharmaphorum deep dive opinion

SMEs: the next generation of pharma

  • The future beyond blockbusters: more precise, more patient-centred and more SME-led.
  • The higher-risk areas that big pharma has moved away from could provide the growth opportunity on which SMEs can capitalise.
  • The UK has infrastructure, insights and systems to help biotechs develop their products without having to reinvest or reinvent.

Chris Molloy, CEO of the Medicines Discovery Catapult, one of the latest members of the UK’s Catapult network, explains why SMEs are set to power future medicines development.

We are familiar with the industry noise around falling productivity and return on investment in pharma R&D. We are less familiar with hearing the call of the small and medium-sized enterprise (SME) community.

With help, I believe this group can quietly develop the greatest explosion in ‘new’ biology in 50 years. Traditionally the agile, risk-taking testers of new ideas, primed to be bought and incorporated into bigger firms or to see their products developed by others, SMEs now have a new opportunity: to grow and conquer.

The Medicines Discovery Catapult has been established in the UK as a collaborative national R&D centre to help SMEs overcome barriers to growth. Our sector analysis, The State of the Discovery Nation 2018 report, shows how this generation can be assisted to go beyond the next acquisition, and become the next generation of pharma.

A model in need of retooling

Every successful medicine has to perform the biological equivalent of threading the eye of a moving needle from 500 miles away. Today it still takes 13 years, hundreds of skills, thousands of people, millions of pounds and countless data points to turn 1% of good scientific ideas into an approved treatment.

So, if the blockbuster medicine is now a thing of the past, what does the future look like? In short: more precise, more patient-centred and more SME-led.

This is the view of the Medicines Discovery Catapult, a new member of the Catapult network focused on turning good science into new, high-value medicines and tools, reshaping the drug discovery model along the way. Our sister organisation, the Cell & Gene Therapy Catapult, has been blazing a trail in this emerging area for over five years.

Today, major pharma is faced with an R&D conundrum: focus on commercially-validated product development, or focus on the science and see where it leads. We may know in 13 years which of these strategies pays the long-term dividends demanded by their shareholders. However, both approaches leave valuable space for smaller companies to do more than simply start up, push to proof of concept, and look to sell.

Stratified disease approaches and new agile trial models lend themselves to faster patient studies with strong industrial footings. More complex biomarkers also lend themselves to studying earlier, more predictive endpoints. While many of these approaches are being proven in cancer, they can and will be used in other areas that discovery needs to focus on today: diseases of systems degradation, tissue ageing and those where multi-morbidities can even make the combination of approved treatments worse for the patient than any of the conditions.

New room to grow

These higher-risk areas that big pharma has moved away from could provide the growth opportunity on which SMEs can capitalise. Using the examples of many successful orphan drug companies (who have developed focused pipelines based on platforms of core science and excellent translation), today’s SMEs, supported by new tools and techniques, may now be positioned to prove principle, collaborate collectively on small cohort trials, and even reach the market by attracting more finance and bolting on outsourced, specialised sales capabilities.

To inhabit these spaces will certainly demand the adoption of new technologies, products and services. It may also require the combinations of companies with complementary products and a requirement for model-breaking in process. However, this is the home ground of the small company, and even translational research charities such as the Cystic Fibrosis Foundation in the US and Parkinson’s UK.

We have talked extensively to SMEs about what tools, techniques and support systems they need in these changing times. The State of the Discovery Nation 2018 report sets out clear calls to action for the sector, by the sector. Having conducted 200 online surveys and 100 face-to-face interviews, we identified two major themes that we, and other national stakeholders, must grasp to create the ecosystem that will secure this community’s future.

New approaches, tools and technologies

SMEs are asking for two major things to help them reduce the risks of failure and become more efficient in translating great science into pipelines of targeted products they can prove in patients more efficiently than is done today.

1. Faster validation and release of breakthrough academic disease models for industrial use.

Biotechs and technologists need faster access to industrially-validated models that better reflect human disease. Much of this remains locked in academia and is tough to fund through translation, in either the public or private sectors. Biotechs find that overly complex IP negotiations and insufficient industrial validation dissuade them from using their limited time and expensive venture capital to extract these potentially game-changing platforms. Validation of these new, data-rich models requires academic science to be coupled with industrial know-how. It then requires access to emerging technologies, such as ‘tissue’ mass spectrometry that can fingerprint disease at the tissue level and informatics that can interpret the torrent of data they produce. It also needs improved access to consented patient samples and data to validate its findings. Above all, it takes collaboration with clear purpose. All these come together within the Catapult.

2. Easier and orchestrated access to existing national R&D capabilities.

The UK has a wide range of robust infrastructure, insights and systems that help biotechs develop their products without having to reinvest or reinvent. The National Health Service, charities and academics, as well as the growing services sector, have great potential strength but can appear impenetrable, particularly to smaller companies, which, coupled to biotech’s natural fragmentation, means they often go unknown or are used inefficiently. Industrial insight on what experiments to run, ‘fail-fast’ R&D planning and smart signposting can provide SMEs with access to the right resource at the right time. This will allow biotechs, public and private sector providers to leverage each other to create national wealth.

A new generation?

These are not simple challenges to address and focus is needed to prioritise those efforts that will have the highest impact. The building blocks are in place, so the work now must use industrial know-how to validate emerging biology platforms and preclinical technologies.

We have to apply our industry experience of multi-party translation and new methods of development to support and enable a highly flexible, high-demand community. With this focused support, SMEs will develop the products that will attract the investment they need to grow, rather than sell, and the UK can firmly embed the next generation of this strategic industry sector.

About the Author

Chris Molloy Medicines Discovery Catapult

Chris Molloy is CEO of the Medicines Discovery Catapult and passionate about its vision for innovative, faster-to patient drug discovery and how this work is aligned with the government’s industrial strategy for life sciences – benefitting the British economy and society.

He has over 25 years’ international Executive and Board experience across multiple areas of medicines discovery, including roles as Chief Operating Officer of MerLion Pharmaceuticals in Singapore and VP Corporate Development and Marketing at IDBS, an international software company focused on improving R&D and healthcare organisations through better access to data.

About the Medicines Discovery Catapult

The Medicines Discovery Catapult is a national facility for collaborative R&D exploring and developing new approaches to the discovery and proof of well-targeted medicines, diagnostics and biomarkers.

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