Panel, Conference Presentation
2014 London Summit - Collaborating to Fund Bioscience
Milken InstituteMargaret Anne Craig, Steve Ford, David Loew, Mark Treherne, John Reed, Margaret Anderson
Investment Landscape & Trends
- The UK government ring-fenced its science budget at £4.6 billion for basic science and manages a £128 billion NHS budget for 62 million patients.
- UK Trade & Investment attracted approximately £1 billion in foreign direct investment last year, primarily through new company setups.
- European biotech investment is shifting from linear to exponential growth, driven by the emergence of three major hubs: Cambridge, Munich, and Basel/Zurich.
- Startups such as Octetion and Speedel have scaled significantly, signaling a maturing ecosystem that allows talent to move between biotech and big pharma without high career risk.
- The number of global research consortia has grown from 360 to over 400 within six months, indicating a rapid shift toward pre-competitive collaboration involving regulators like the EMA and FDA.
- Clinical trial attrition remains a critical friction point, with 40% of drugs failing in Phase III specifically due to cardiac toxicity.
Challenges in Neurodegeneration & CNS
- Current Parkinson's treatments are 45 years old, focus on symptom management, and suffer from severe side effects and waning efficacy.
- Roche faces high hesitation in neurodegeneration trials due to lengthy timelines (5–7 years for Phase II readouts) compared to the 6-month timelines possible in oncology.
- Alzheimer's disease trials are projected to require 15–20 years, and polycystic kidney disease trials could extend to 30–40 years without new predictive tools.
- Patient organizations are pivoting from passive funding to strategic "venture philanthropy" aimed at de-risking early-stage assets to incentivize commercial investment.
- Parkinson's UK has recruited a former pharma executive as Research Director to inject commercial discipline and IP strategy into charitable funding.
- A key barrier to investment is the lack of biomarkers and imaging agents needed to quantify disease heterogeneity and predict progression.
Academic & Entrepreneurial Ecosystems
- Margaret Ann Craig's University of Glasgow spin-out developed a technology selected as a new regulatory asset by the FDA, set to be implemented by summer of the following year.
- University spin-outs face significant hurdles in accessing finance due to a lack of internal business experience and advisors within academic institutions.
- Scotland is considering removing IP controls from university Research Council funding to mandate a percentage of funds be used for commercial company interactions.
- A cultural shift is required to move academia from a "publish or perish" model to one incentivized by translational impact and commercialization.
- The US benefits from a robust network of non-profit groups (e.g., Biocom, Connect) that actively mentor new biotech CEOs and shape public policy for entrepreneurship.
- Europe lags in philanthropy's catalytic role; while the US has groups like the Michael J. Fox Foundation with massive critical mass, European charities are smaller and less coordinated.
- California's $3 billion stem cell initiative successfully combined patient advocacy with economic development arguments to secure state bonds and drive private investment.
Pharma & Regulatory Perspectives
- Roche and Sanofi are engaging in pre-competitive collaborations to pool clinical trial data and build models that could reduce required sample sizes by 30–40%.
- Roche is developing a new depression treatment with a novel mechanism that lacks the side effects of current antidepressants, though European reimbursement models currently struggle to value innovation over cheap generics.
- The establishment of EU-HTA Net is beginning to harmonize payer discussions regarding endpoints across Europe, a process that previously took years to centralize under the EMA.
- Sanofi observes that patient-centricity is now a business imperative, with payers and regulators increasingly requiring evidence of what endpoints actually matter to patient quality of life.
- There is a growing recognition that "tools research" (biomarkers, trial models) is as critical as product research for de-risking long-term development programs.
- Roche is funding projects like CAMDI to aggregate genomic data from cohorts, aiming to create shared resources that make CNS trials more feasible.
Patient Advocacy & Future Strategy
- Patient organizations are urging a shift toward "tools" development rather than just single-drug funding to address fundamental data gaps.
- Patients are increasingly educated via social media, bypassing traditional channels to demand compassionate use and influence FDA trial designs directly.
- Faster Cures data indicates that medical philanthropy remains a small, static slice of the total funding pie, necessitating better models to leverage it for the entire system.
- There is a consensus that European charities must collaborate to create funds with the critical mass of US counterparts (e.g., Michael J. Fox Foundation) to drive disease-modifying product development.
- New educational models, such as joint MD-PhD and MBA-MD programs in the US, are cited as successful templates for creating leaders who understand both science and business.
- The UK government has appointed George Freeman as the first Minister for Life Sciences with a joint appointment between the Department of Health and Department for Business to drive innovation uptake.
- Panelists predict that the integration of mobile health technologies and data from systems like the UK's 100,000 Genomes Project will significantly de-risk clinical trials by improving patient selection and cohort speeds.
- A proposed "Consortia Pedia" report highlights the rapid rise of consortia, though the industry now seeks clearer metrics and ROI from these collaborative efforts.