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Category: Selected writings

Three key barriers to the effective translation of bioscience breakthroughs into patient benefit

In April 2012, speaking from Japan, a very symbolic location, the UK Minister for Universities and Science David Willetts announced the opening of a £180 million Biomedical Catalyst scheme to provide grant funding for innovative small and medium sized enterprises (SMEs) and academics to develop solutions to healthcare challenges. Prime Minister Cameron, and before him Blair , had emphasised that “Invention is one of the things we do really well in the UK”. Without a doubt, the emphasis is for translational research to be at the heart of science policy in the UK and to ensure that scientific innovation gets translated into applied uses in business.

The NIHR, through the Biomedical Research Centres (BRCs) and Units (BRUs), the Comprehensive Local Research Networks (CLRN), the NIHR Office for Clinical Research Infrastructure (NOCRI), and the Technology Strategy Board (TSB), to name only a few, have all been established to facilitate the process of translating bioscience breakthroughs into patient benefit by providing clinical infrastructures and personnel, funding, access to patients and to businesses.

However, numerous obstacles remain and this paper will outline three of the most important barriers to effective translational breakthroughs. These have been identified based on my 17-year experience working in the UK, from the bench to the clinic and beyond, in areas as various as basic sciences trying to understand the mechanism of cancer progression to the conduct of international phase III HIV prevention trials.

Translational medicine aims to bridge the gap between basic science and its application to human diseases, from bench to bedside, and beyond. It is a long and complex process too often inappropriately conceptualised as a stepped progression in which one stage of product development leads to another. In fact, it is much more appropriate and accurate to consider translational medicine as a whole, as a system designed and managed for the purpose of bringing medical innovation to the public.

The process is fraught with pitfalls and drawbacks and the risk for failures are numerous and high at every stage. The following three areas represent what I believe are the most important barriers to effective translation.

Intellectual Property (IP)

This is an area often overlooked at the early stage of research and development. However, existing IP can restrict access to products and technologies on which some innovations are built. Combining different IP is often difficult. Owners of background IP see a potential danger in mixing IP from different products and technology, in terms of ownership as much as because they fear potential damages from unexpected outcomes of the research.

At a later stage of development, unsettled or unexplored IP issues can threaten the development and roll-out of scientific innovation. Working with the industry and trying to understand and agree IP management and exploitation requires highly qualified professionals who are not always available in an academic environment where the translational process starts.

Art search and the patenting process are long, time-consuming, and costly. They require a non-negligible effort and contribution from researchers to provide background information about their products which they don’t necessarily have. Nowadays, several grant schemes have stressed this aspect of product development (such as the MRC DPFS/DCS scheme), but they have also created further difficulty by tying funding to their own IP terms. Gates and EU grants require favourable pre-agreed terms for access in developing countries whilst projects are still at an early stage. This is unwarranted as commercial success is unpredictable in the early days of development but creates barrier to access.

Regulatory frameworks

There is a giant step between making and testing a product arising from a bioscientific breakthrough in a research lab or in pre-clinical settings, and then manufacturing and trialling the same product for the purpose of using it in humans. Rules and guidance for product manufacturing (cGMP) and the conduct of trials (ICH-GCP), decided at EU level and implemented nationally, are complex and constraining. Regulations in the US and elsewhere are also different.

Overall, such regulations create different levels of requirements which limit and complicate opportunities for product manufacture and development. Further, regulations often fail to distinguish between products for use in early stage human studies and products for late phase III trials leading to increased cost and delay in manufacture and development.

The constraining frameworks created by these regulations have been recognized. In 2011 the EU conducted a Public consultation on a concept paper on the revision of the ‘Clinical Trials Directive’ (2001/20/EC) to assess its functioning. Much work needs to be done in order to simplify and streamline regulations and construct them so that they address the specific risk associated with new discoveries.

Management and coordination

Translational Medicine Management is about filling the gaps, joining the parts and building bridges between numerous infrastructures, people, interests and concerns. Skilled and efficient management from start to finish, from idea to end user is critical and pivotal in overcoming the many obstacles that thwart translational medicine.

The knowledge required is vast in scope (from basic science to trial regulations and public health policy) and necessitates skills in administration, finances, business and IP, as well as a sound knowledge of institutional practice. Several organisations have been set up to provide some support to researchers and clinicians wanting to engage in the process, but the breadth of the task ahead remains daunting.

Innovators have to juggle many other tasks in parallel and are not necessarily trained to manage a seamless transition from the bench to the bedside. Universities rarely provide the skilled managers and operational support required and inventors are left to deal with a high level of complexity on their own, not always knowing where to start and where to go for advice and support/assistance. It would be useful to create a roadmap from breakthrough to patient as there is one for the conduct of clinical trials .

Navigating the rapids, in what David Willetts called a “valley of death” towards commercial successes that will benefit patients remains a slow, expensive, and failure-prone endeavour . There is help at hand but the multiplicity of frameworks and stakeholders creates barriers to the translation of innovation into practical applications.

With the current emphasis on conducting research that benefits patients and contributes to the UK economy, the cost remains an issue at all stages but projects that are worth developing are able to raise and attract funding when they receive appropriate institutional and business support. Funding, now often milestones-based, decreases the risk for the funder to invest in a product for which development remains uncertain.

There is a need for building and understanding the big picture that would help identify roadblocks and leverage points to fasten and simplify the translation research process in line with government aspiration.

HIV Prevention: towards the medicalisation of sex?

2010 will be a year to remember for the field of HIV prevention. After decades of interventions with limited results (with the exception of circumcision and the prevention of mother to child HIV transmission), two clinical studies are raising the hope that the HIV epidemic can be tamed.

In July, the CAPRISA team (based in South Africa) reported that a vaginal gel containing the anti HIV drug tenofovir could reduce the risk of HIV infection by 39%. This was the first proof of concept that a microbicide could potentially reduce the risk of HIV infection whilst offering women an HIV prevention tool that they could control.

In November of the same year, the iPrEx study conducted on a population at high-risk of infection showed that taking the anti HIV Drug Truvada reduced the risk of contracting the virus by an average of 44 percent.

Both studies are hailed as a milestone and landmark in the history of HIV prevention and expectations are high that HIV prevention will finally mean more than the ABC of ‘Abstinence, condom and faithfulness’. But despite the hope, neither approach will immediately translate into marketable products as there are a number of questions that needs answering before microbicide and PrEP are available to the public. Read more »

A funding struggle for an HIV prevention in women’s hands

Attendees at the 18th International AIDS conference held in Vienna in July 2010 felt a tremor of hope when Prof. Salim Abdool Karim received a standing ovation following the announcement that a vaginal gel containing the anti-HIV drug tenofovir could reduce the risk of HIV infection by 39%. The groundbreaking results came out of the CAPRISA clinical trial conducted amongst 900 women in rural Vulindela district (KwaZulu-Natal) and urban Durban, South Africa. Euphoria followed in the audience, online and later in the printed media. After 30 years of limited success, the field of HIV prevention could potentially add a new powerful tool to circumcision, condoms, and the prevention of mother to child transmission. Most remarkably, that tool is in women’s hands and the story could unfold with even more good news if it weren’t for a small setback: funding the next clinical studies. Read more »

Microbicides: Efficacy and Effectiveness

What is good enough? Who decides?

This presentation was prepared for an IRMA global teleconference on December 4, 2009.


A Sustainable Globalisation?

washington vs Geneva ConsensusGlobalisation has been the buzzword of the roaring nineties and with the fall of the Berlin’s wall, the end of the cold war and the victory of capitalism over socialism it has opened a new era in human history. Rightly or not, globalisation has become synonymous with market economy, capitalism and development. Much discussions, books and movies have placed it at the centre of the debate about the future of development with a “New Deal” or a “New Barbarism” as two possible scenarios. As the Washington Consensus is being challenged by the Geneva Consensus, the possibility of a sustainable globalisation, conducive to social justice, human security and environmental protection, being an unrealisable goal is a question of great contemporary interest. To address this question we will examine how globalisation affects social justice, human security and the environment. We will then introduce different views and responses to the globalisation process, which when integrated altogether will provide a framework to answer the question of realizing a sustainable globalisation. Read more »

Challenges facing the UK Research Base

The academic year 2007 started with a “Time Bomb” uncovered by the Higher Education Policy Institute (HEPI): student in England are having it the easy way with an average of 26 hours a week spent studying compared to 30 in Ireland, 35 in France and 41 in Portugal. Variations are wide depending on the subject but with less than 15 hours of tuition a week, fears and concerns are raised that Higher Education is going down the drain and will have serious academic consequences for the future of Science in the UK. Read more »

Gendering the Fight against Aids

Two strong messages have emerged from the 16th International Aids Conference in Toronto, Canada. The first is that with drug treatment now being rolled out in developing countries, prevention should return to centre stage in future policies and strategies. The second is that women’s lives and status need to be improved and that women need to be given power to prevent HIV infection.

Both messages were embodied in Bill Gates’s keynote speech:

“We need to put the power to prevent HIV in the hands of women. This is true whether the woman is a faithful married mother of small children or a sex worker trying to scrape out a living in a slum. No matter where she lives or what she does, a woman should never need her partner’s permission to save her own life.” Read more »

French Fries and Fat Kids – Asia’s next Epidemic

Popular belief has it that obesity only affects wealthier societies where food is plentiful: the curse of the developed world epitomized by hulking Americans that struggle to order their king-size Big Mac, French Fries and Coke without breaking sweat.

Obesity is no longer exclusive to the developed world

The reality is a very different. Obesity and its associated diseases – diabetes, hypertension and kidney diseases – respect neither wealth nor class and strike instead into the heart of every society where there is easy access to convenience food, low physical activity and ubiquitous advertisements for sugar-fat-salt-rich food. Read more »

Wealth versus Health – the Thai Frontier

In the battle against HIV and AIDS, Thailand has been exemplary: since 2001, the AIDS death rate there has fallen by 79 percent, thanks to the supply of low-priced locally produced generic drugs and the 30-Baht universal health care scheme. But this success story is about to be challenged by the United States-Thai Free Trade Agreement (FTA) currently under negotiation, which includes restrictive intellectual property rights, and will put at risk the survival of hundreds of thousands of Thai people living with HIV, and beyond Thailand, the survival of millions who will be affected by the Thai precedent. Read more »