Sequencing a genome is similar to having all the pages of a manual needed to make the human body. Less than a decade ago, it cost millions and took many years to sequence the complete genome of one individual. Now it takes a day and costs less than €1000. This biological revolution makes it easier to identify abnormalities in the genome that can cause medical problems. Based on a deeper understanding of disease at the molecular level, pharmaceutical innovation is leading to a whole new generation of targeted medicines, many of which will be much more effective and cause fewer side effects than those available today.
The journey towards personalised medicines has already begun: A personalised medicine for advanced skin cancer was recently approved by the European Medicines Agency. It was developed after scientists learned that the mutation of a particular gene drives tumour growth in about 50% of patients with malignant melanoma, the most deadly form of skin cancer.
Innovations like these are significant achievements and worth celebrating. What’s more, we need to ensure the potential for further success stories like these with a supportive regulatory environment. A regulatory framework tailored to adaptive or progressive models can help guarantee timely delivery –for instance, one involving iterative phases of data gathering and evaluation.
To ease the regulatory environment for personalised medicines, it’s necessary to take a broad look at all areas that might be relevant. Regulation on everything from marketing authorisation application processes to companion diagnostics can be relevant. Companion diagnostics, for instance, are used by healthcare professionals in their decision-making when tailoring treatment to patients. Any regulation regarding companion diagnostics must thus take into account the impact it can have on personalised medicines. Regulation relating to personalised reimbursements and delivery is another essential issue. Market access conditions for personalised medicines could be improved, for instance through some harmonisation of Health Technology Assessment (HTA) processes: HTAs provide evidence on the effectiveness of diagnostic and treatment methods, medical equipment, pharmaceuticals, rehabilitation and prevention methods, as well as organisational and support systems used to deliver healthcare. They are used by policymakers to guide healthcare decision-making on issues such as pricing and reimbursement. Efficient HTA systems can help ensure efficient delivery of innovations like personalised medicines to patients.
Translating the new scientific paradigms into real healthcare solutions using personalised medicines is a challenge that requires massive investment at EU level and coordination across a wide range of research fields. The Innovative Medicines Initiative (IMI) is Europe’s largest public-private partnership and is one initiative working towards this end. One project funded by the IMI is the DIRECT (DIabetes REsearCh for patient straTification), a 45 million Euro project that focuses on the stratification of patients with diabetes. Scientists and clinicians working in academia have joined forces with the pharmaceutical industry to tackle the current bottlenecks in diabetes drug development and to develop a personalised medicines approach to treatment of ‘type 2 diabetes’ (T2D) with either existing or novel therapies. Leading European experts from 21 academic institutions and four pharmaceutical research organisations officially launched the DIRECT project on February 1 2012. The close collaboration of academic teams and pharmaceutical companies is providing unique levels of expertise and forms a strong basis to reach the DIRECT project goals.