New Report: UK's Cell and Gene Therapy Sector Growing at a (very) Healthy Rate

The Alliance for Regenerative Medicine and the Bioindustry Association of the UK have released a report (Report) concerning development and growth of the cell and gene therapy field in the United Kingdom.  The Report notes “four key takeaways”: 

[1] The UK is a leading source of innovation in the research and development of advanced therapy medicinal products (ATMPs) in Europe.  [2] There is strong government support for scientific innovation, capital formation, and patient access to cell and gene therapies in the UK. [3] There is significant investment in the UK   to support the development of these life-changing therapies. [and 4] The clinical pipeline in the UK, both in terms of UK-based companies and other companies interested in clinical development in the UK, is robust and growing.

The Report states that 24% of ATMP companies in Europe are headquartered in the United Kingdom, additionally there was over a billion US dollars in funding.  The funding in 2019 is on track to meet or exceed 2018’s funding.  There are detailed stats on funding by type in the Report.  Moreover, since 2012, there has been a substantial uptick in ATMP activity—from 22 companies to over 70 companies, some of which is attributed to significant government support and organization.  The Report also contains data regarding current and past clinical trials and case studies concerning relevant companies and their technology.  


The Report concludes with the following recommendations: 

Support scientific research to develop and advance both cell and gene therapies and ancillary processes, including manufacturing and scale up.

Foster economic development and the creation of a skilled workforce to promote the continued growth of this industry in the UK.

Cultivate a positive regulatory environment for the research and development of cell and gene therapies, including fostering accelerated pathways to ensure that patients are able to access safe and effective therapies in a timely manner.

Develop the necessary infrastructures within NICE and its counterparts in Scotland, Wales, and Northern Ireland to ensure health technology assessments are able to address the long-term value provided by cell and gene therapies.

Collaborate with the NHS and other public and private payers in the UK to develop innovative financing models to ensure patients can access approved therapies in an efficient manner.

The development of a skilled workforce (and attraction of one) and the related issue of international collaboration is important, but not expressly tied together in the recommendations. The full Report is available, here. 

Trump Proposes to Cut Government Funding for Research

CNN recently reported on President Trump’s proposed budget and noted serious cuts to government funding for research.  Specifically, the article states:

The National Institutes of Health budget would be cut by $5.8 billion, meaning it would lose about 20%. The Environmental Protection Agency would face $2.6 billion in cuts, that's 31% of the agency's budget. The Department of Energy would lose $900 million, or about 20% of its budget. Health and Human Services would see a $15.1 billion or 18% budget cut; as part of that, it shifts costs to industry from the Food and Drug Administration budget. The National Oceanic and Atmospheric Administration would face an 18% budget cut.

As the article describes, a number of groups have criticized the proposed budget.  A recent Denver Post opinion piece by Noah Smith, a Bloomberg commentator, notes that the U.S. innovation system works well because we actually have a “pipeline” of new discoveries running to commercialized inventions.  Smith states that Trump is cutting off the new discoveries and essentially putting the U.S. at a disadvantage.  As this blog has noted, the Obama Administration basically kept funding for research almost level, but the budget was decreasing in terms of real dollars.  This was considered bad.  If we actually cut at the levels Trump wants to cut, we will be in a much worse position.  As Smith notes, the rust belt has somewhat been revitalized by biotech and cutting the NIH budget will retard the growth of one of the United States’ most promising industries. 

Importantly, Congress must pass the budget.  And, if my memory serves me correctly, President George W. Bush also proposed to cut government funding for research at the outset of his presidency and later retracted that proposal.  I imagine that someone explained to the Bush Administration how important government funding for research is for industry and the U.S. economy—hopefully that will happen for the Trump Administration. 

Measuring the Success of Technology Transfer Offices (and the field)

There is a glaring critique of the university technology transfer enterprise and perhaps the underlying Bayh-Dole Act in the United States.  That critique is based upon the fact that many university technology transfer offices fail to bring in enough funding through licensing or other activities to cover their own costs let alone make money for the university.  Indeed, only a handful of U.S. universities appear to make substantial amounts of revenue.  An additional criticism of the university technology transfer field generally has been that technology transfer offices (and really, the administrators above them) have been too focused on using revenue generated as a metric for success.  This focus arguably can distort the universities’ general mission directed to the public good, including skewing the incentives for academics.  For example, academics can be pushed to adopt research agendas focused on solving practical problems instead of engaging in basic science, which may ultimately have broader public benefits.  At the confluence of these two critiques is the issue of what should be the proper metric(s)for judging success for the technology transfer office and the field in general.  For sure, U.S. universities are feeling the “pinch” of less government monies for research and are looking for alternative funding sources, such as crowdfunding for academic research.  However, even with that pressure, adminstrators and faculty should judge the success of their technology transfer office based on criteria that flow from the mission of the university and that are aligned with its objectives.  So, when is it a success or not?  What are the right metrics?

Valerie Landrio McDevitt, Joelle Mendez-Hinds, David Winwood, Vinit Nijhawan,Todd Sherer, John F. Ritter, and Paul R. Sanberg, have authored a paper titled, “More than Money: The Exponential Impact of Academic Technology Transfer.”  The paper sets forth the benefits of technology transfer beyond revenue alone and perhaps provides the starting point for the development of additional metrics to judge the success of technology transfer offices.  Here are the benefits described by the authors:

Revenue generation

Unrestricted funds to institution from license income

Direct personal financial benefit to inventors and authors

Increased opportunities for funding

Eligibility for funding by compliance with federal regulations requiring a technology transfer program

Increased opportunities for interinstitutional and interdisciplinary grants

Outreach, licensing, and facilitation of new startups yield new funding partnerships

Increased opportunities for funding sources requiring a commercial partner, for example, SBIR and STTR

Facilitates establishment of international research relationships

Promotes a culture of entrepreneurship and innovation

Successes increase university brand and prestige

Enhances university fundraising efforts

Opportunities to strengthen donor ties by engagement with startups

Positively factors into high level recruitment efforts

Positively affects retention of high-producing and high-potential faculty

Student success

Provides opportunities to participate in real world translational research

Provides exposure to the process of obtaining intellectual property protection

Strengthens prospects of finding jobs and being successful

Public benefit

Fulfills the university’s larger missions to address social, medical, environmental, or technical problems

Improves the quality of life

Economic development

Revenue from university licensing positively affects the US economy

Brings money into the state or region

Aids in the retention of local talent

New university startups create high-wage jobs

It may be difficult to measure some of these “benefits.”  But, what do you think of some of these as potential metrics?  For sure, some of the most beneficial programs often bring to the table attributes that are difficult to measure.  And, surely, metrics such as revenue generation, invention disclosures, patents granted, patents applied for, patents licensed, number of start-ups and other traditional metrics still have some place in the game.  (Hat tip to Technology Transfer Tactics for a lead to the paper.)