Patient groups need to be given a bigger role – after all, the entire point of the medicines development process is ultimately the benefit of patients.
European Federation of Pharmaceutical Industries and Associations (EFPIA) Director of Science Policy Magda Chlebus champions the collaborative efforts of partnerships like IMI in moving medicines research and development forward...
Collaboration is a word being heard ever more frequently in discussions about research and development in the pharmaceutical industry – and with good reason. Today’s scientific and technological tools are opening many new doors in medicines development. We have the possibility to develop targeted treatments in the form of personalised medicines. There is the potential offered by ‘big data’ – the enormous amount of healthcare information generated with tools like genomic sequencing machines and electronic health data. Such advances have already contributed to medical success stories, and can be of great help as the pharmaceutical industry takes aim at the healthcare challenges ahead, from addressing the problems posed by ageing populations, to filling treatment gaps in areas of unmet medical need. Research methods are evolving, and we are entering a promising new era of medicines development. However, great promises also present great challenges: tapping into the potential of such technologies in a way that ensures maximum benefit for patients around the world presents unique hurdles.
Personalised medicines provide a compelling example of the dichotomy in the current moment in medicines research and development. These medicines are developed based on a disease’s unique molecular make-up: the European Medicines Agency has already approved such medicines for certain types of breast cancer and skin cancer. In the future, we may be able to develop targeted treatments for thousands of different types of cancer. However, this demands a significant investment of time, energy, and money. The development process is complex: because tumour cells are heterogeneous and genetically unstable, a cancer sub-group may develop resistance to a pharmaceutical agent during treatment. This is one reason for the move towards adaptive methodologies and away from the randomised control clinical trial model, which is nearly 50 years old and outdated in many ways.
The role of 'big data'
Another challenge with personalised medicines comes from the role of ‘big data’. The 13 year long genome sequencing project concluded in 2003 and cost some $2bn. Today, an individual’s genome can be done within a day for less than €1,000. However, assembling that sequence into a complete genome remains more time consuming and costly than is practical. Known as secondary analysis, this process is necessary if scientists are to gain a comprehensive understanding of a person’s genetic variations, which is needed to identify potentially effective targeted treatments. The storage of genetic information is also problematic. A single person’s genotype is one thing; but storing the gene sequences of many patients in a way that researchers can easily access and search the data is difficult. Further, since personalised medicines address fewer patients for specific treatments, a structure for clinical registries to help assess a medicine’s clinical effectiveness – the actual outcome when a patient population is treated – would be necessary to evaluate the long-term benefits and risks of such medicines.
Such challenges are compounded by a difficult regulatory framework in Europe, which has not kept up with the advances made in science and technology. More flexible regulatory models and a supportive incentive system that accommodates adaptive methodologies are needed if we are to help control growing R&D; costs and ensure more efficient patient access to innovative medicines.
When you start to enumerate the hurdles standing in the way of research and development, a daunting scenario unfolds. These challenges are too significant for any one person, institution, company or discipline to tackle alone. Therefore a diversity of knowledge and resources is needed.
Researchers need to collaborate and share their knowledge, and companies need to come together and pool their resources and data. This is already being done, as companies – once strictly competitors – are banding together to take on demanding issues, such as diabetes treatment development.
The industry needs to enhance its working relationship with policymakers to address regulatory issues. Here, again, we are already seeing changes: a move towards more open dialogue is evident, and concrete relationships are being built in the form of public private partnerships such as Europe’s largest, the Innovative Medicines Initiative (IMI). Jointly funded by the European Commission and EFPIA, with a total budget of €2bn, IMI is the biggest biomedical research public-private partnership worldwide.
'Enhancing patient knowledge and participation'
Patient groups need to be given a bigger role – after all, the entire point of the medicines development process is ultimately the benefit of patients. The IMI-funded EUPATI project (European Patients’ Academy on Therapeutic Innovation) is a patient-led initiative aiming to develop educational material about the R&D; process, with the aim of enhancing patient knowledge of and participation in R&D.; Patients are also increasingly playing an active role in research thanks to improved self-monitoring devices – for instance by providing data through health monitoring smartphone apps.
Of course, this means even more stats to add to the ‘big data’ flood – another problem being addressed by collaborative solutions. In 2011, the Coriell Institute for Medical Research turned to IBM for help maintaining its vast collection of biomaterials. More recently, Harvard researchers teamed up with coding community TopCoder.com to launch a contest for computer programmers to write genetic analysis software.
These are just a few of the examples of collaborative efforts already being made in the interests of medicines research and development – and it’s a trend we must continue to push for if we want to see a positive progression of the current evolution. It’s essential to acknowledge that the challenges facing medicines development cannot be overcome by any one individual: teamwork, collaboration, and open dialogue are musts. All relevant stakeholders – researchers, patients, healthcare providers, decision-makers, and others – need to be at the table when discussing how to move research and innovation forward.
Director of Science Policy
European Federation of Pharmaceutical Industries and Associations (EFPIA)
[This article was originally published on 10th
June 2013 as part of Science Omega Review UK