Personalised Medicine: Putting promise into practice
This month Oxbridge Biotech Roundtable (OBR) brought a key debate to the University of Manchester. With recent huge developments in personalised medicine, the question remains whether these advances are being put into practice and translated into healthcare benefits. An in-depth discussion was led by OBR, bringing together a panel of Professor Simon Hollingsworth, Executive director of Personalised Healthcare and Biomarkers at AstraZeneca, Professor Katherine Payne from the University of Manchester who holds a chair in Health Economics, Dr Steven Little, CEO of Premaitha Health, Dr Bill Newman, Senior Clinical Lecturer in Genetic Medicine at the University of Manchester, and Dr Mark Saunders Consultant Oncologist at Christie Hospital.
Personalised medicine is a model that encompasses all aspects of medical decisions, practices and products, to be tailored to the individual needs of the patient. With the current ‘one size fits all’ approach to medicine, doctors often prescribe medicine that is ineffective or leads to bad side effects, since treatments are tested on broad populations and are prescribed using statistical averages. For example anti-depressants are effective in only 62 per cent of patients, and ineffectiveness of cancer drugs ranges from to up to 75 per cent.
Thanks to the Human Genome Project, which celebrated its 10th anniversary this year, we can use genetic sequencing technology to target treatments to patient subpopulations that are more likely to respond to it. Biomarkers and the presence or absence of drug receptors can indicate how patients should be treated, enabling preventative as well as therapeutic interventions. In addition to this, personalised medicine can reduce the time, cost and failure rate of clinical trials, and eliminate trial-and-error inefficiencies that inflate healthcare costs.
So despite the tremendous potential, what is stopping it from being incorporated into everyday healthcare? OBR led the debate using three main questions:
Has the blockbuster drug model hindered new approaches to tailored medicines?
No, according to Prof. Hollingsworth, arguing that the ‘reality’ is that we need to invest money to research into new drugs, and that blockbuster drugs have actually driven us into this new personalised medicine era. Dr Newman expressed that pharmaceutical companies are charging far too much for drugs, which in turn Prof. Hollingsworth responded with the need for pharmaceutical companies to demonstrate ‘clinical utility’ of the drug for FDA approval. This requires running global studies, which cost millions, and if the National Institute of Clinical Excellence (NICE) think the NHS cannot afford the drugs then the company itself loses out too. This then led to him expressing that people have ‘lost their faith’ in NICE. The UK is unique in having the NICE infrastructure, having a cost-effective analysis in place is important for fair deployment of treatments, and as Prof. Payne pointed out the NICE is necessary for looking at the system as a whole. The NHS and pharmaceutical industry have different objectives that often cause tension between the two, and so guidelines set by NICE are vital to deliver the best healthcare.
How is personalised medicine developing beyond diagnosis into prevention and prediction of disease?
Since 2007, the FDA has recommended genotyping for all patients receiving therapy involving Warfarin. Prescribed over 20 million times a year, a very dangerous implication for many was blood clotting, and thus genotyping allows the prescribing of drugs at the correct dosage only to those who would benefit from it. ‘We can now split patients up and target the ones who need it’, said Dr Saunders, and this is being applied in more and more areas of medicine. The development of Oncotype DX, a breast cancer assay, helps specialist teams determine the likelihood of disease reoccurrence and whether patients would benefit from certain types of chemotherapy. With techniques such as genetic profiling and immunoassays increasingly being used, personalised medicine is helping to predict susceptibility of disease, and preempt disease progression.
Is there a market for personalized medicine?
According to PwC’s estimates, the total US market for personalised medicine is approximately $232 billion and set to grow 11 per cent annually, nearly doubling in size by 2015. There is a market for this new healthcare paradigm, although it is in its early stages, we are beginning to see its initial benefits.
So although personalised medicine offers tremendous potential, many aspects hinder its wider application. From improved understanding of the molecular basis of disease, to adaption of the technology and structure within healthcare systems, there are many challenges. To fully integrate personalised medicine, there will need to be advances in screening and diagnostics, as well as education and training. Furthermore, regulatory issues, as stressed by Prof. Hollingsworth, are extremely costly. Despite enabling more targeted clinical studies to demonstrate efficacy, regulators’ demands to demonstrate the safety of new drugs, along with diagnostic tests and a clinical algorithm guide, can impose huge development costs. As a result, it may be decades before we see the full effects of personalised medicine. A shift of focus from therapies to diagnostics would speed up innovation in this new model, as well as a new health technology assessment body, as introduced by the EU.
Image source: http://www.bigbrotherwatch.org.uk/wp-content/uploads/2012/02/dna-3.jpg