How to solve the puzzle of personalised healthcare

Carl Johan Sundberg, Associate Professor at the Karolinska, who is directing the research with Science|Business

The reality has been different than the promise. Roll-out has been slower, investment difficult. Regulatory clearance has been fragmented. While there has been progress and hints of the benefits to come, many barriers still stand in the way of deployment.

Why, and what are the barriers? This is the topic of a European research project and series of conferences being organised during 2010 by Science|Business, in collaboration with leading medical university Karolinska Institutet of Stockholm, and a consortium of industrial partners including Novartis, Pfizer, Microsoft and Genzyme. The research project, to be released this spring, analyses expert attitudes towards the technology in four European Union countries. The first of the conferences to discuss the results was held in Brussels on February 4.

The focus was on the barriers to development and deployment of the technology – and on ways to overcome them in Europe.  One challenge to overcome is the research, itself: For example, while a handful or drugs now come with companion diagnostics, and there are early moves to give those suffering from chronic diseases the means to monitor their disease state, much more remains to be done to create an integrated picture, in which personalisation begins at the start of drug discovery and is carried through to every aspect of healthcare. At the level of the tools that will make personalised medicine a reality there is a need to develop more biomarkers, diagnostics and point-of-care monitoring devices.

But there are other barriers that have nothing to do with the lab, and everything to do with the way we organise and regulate our healthcare systems:

• Insufficient Investment – As yet there are no overarching research programmes. How will the costs and benefits of personalised medicine be demonstrated, how will it be paid for, and what will be the attitude for reimbursement? This uncertainty deters many investors.

• Regulatory Vacuum – The European Medicines Agency, Europe’s national regulators, and the US Food and Drug Administration have yet to lay down rules for integrating biomarkers into drug development, or for the simultaneous approval of drugs and diagnostics. In Europe, diagnostics are approved by a separate regulatory system from drugs.

• Policy Vacuum – For the cost/benefit equation of personalised healthcare to be demonstrated, the current budget silos must be broken down. Monitoring patients with chronic diseases at home will increase costs for primary medicine, but reduce the cost of acute care; drugs that come with a diagnostic should be more effective, but will cost more too.

• Health Promotion - EU citizens are not provided with sufficient information on personalised healthcare products and services. One aspect of this should be to explain the prospects for greater efficacy and the lower risks of side effects in personalised medicine, in a bid to improve compliance Carl Johan Sundberg, Associate Professor at the Karolinska, who is directing the research with Science|Business, painted an overview of some practical measures to start to dismantle some of these barriers. First, he noted, the European Union overall has very good potential for studying populations, including tools such as national biobanks and cohorts in studies of particular diseases or populations from particular areas, that may go back as long as 50 years.

“But often they are not maintained. New knowledge will emerge when the people included in such studies become ill and eventually die. But we have to fund the infrastructure to allow such investigations to happen,” Sundberg said.

Secondly, it is important to broaden the focus from personalised medicine, to healthcare as a whole, factoring in all the variables to build a single, integrated structure for research into, and the delivery of, personalised healthcare, believes Sundberg. Thirdly, healthcare systems should pay for performance and reward innovation.

In another move, orphan drug legislation should be extended beyond its current focus on rare diseases – where it has had a very positive effect, to recognise that in personalised medicine, drugs will be effective in much smaller populations. As Sundberg noted: “Giving intellectual property protection for a longer time tends to change behaviour.”

And finally, comprehensive personalised healthcare cannot be realised without accompanying information technology systems, where information about tests, and so on, is available (with an individual’s consent), anytime and anywhere, said Sundberg.

Emmanuel Chantelot, Executive Director of the industry body European Biopharmaceutical Enterprises, agreed that biobanks are central to the development and deployment of personalised medicine, and he called for a “European Reflection Process” to consider how to plot a path to harmonisation of the 27 different national systems they are operating under currently. “On the legal side there is no regulatory harmonisation. There should be an effort to do this – to set rules on the use of samples and so on.”

Magdalena Radwanska, Science Officer for Strategic Activities at COST, reinforced this view, noting that in addition, “There is a requirement for standardised protocols on how to collect samples and store and analyse them, and for informatics and systems to be standardised too.”

As a representative of patients, Alastair Kent, Director of the Genetic Interest Group, also believes data re-use should be possible. One of the fastest routes to making that happen is to talk to patients and give them a sense of the potential of personalised medicine and personalised healthcare to enable them to make informed choices about their own care.

“We need to have public confidence and that means a greater effort to engage with patients and the public. We tend to protect patients’ interests through proxies such as ethics committees, but I suspect patients would care more about loss of opportunities to intervene and direct their treatment.”

As Adam Heathfield, Director of Science Policy Europe for Pfizer noted, this also exposes the need to reform health systems, to make them more patient-centric. “For a revolution in personalised medicine to occur, a number of elements in health systems will need to change. Personalised medicines are already having a significant impact, but this approach will only be able to show its maximum value and achieve widespread adoption in systems where informatics, diagnostics and reimbursement are all optimised around individual patient care.”

Werner Kroll, Head of Innovation and Research Alliances, Novartis, suggested this underlines the need to move on from traditional cost-benefit analyses. If innovative molecular diagnostics require more investment than routine laboratory tests, this should be acceptable and appropriately rewarded if it means a saving is made elsewhere. “In other words, we need to progress from cost-based to value-based assessments, and get away from silo budgeting,” Kroll said.

In terms of how things are shaping up at a European level Ruxandra Draghia-Akli, Director, Directorate Health, DG Research, European Commission, said the science is moving very quickly, leaving researchers and clinicians with a lot of information to appraise and organise in moving personalised medicine forward. “Creating the means to enable EU researchers to focus on important themes [in personalised medicine] is clearly a preoccupation”, said Draghia-Akli.

“We recognise that personalised medicine and personalised healthcare is what the medicine of the future will be. We are focussing on personalised medicine and will have a series of actions to advance it.”

Irene Norstedt, Head of Sector, Medicines for the future, DG Research, European Commission, noted there has already been significant investment research that is relevant to personalised medicine. In future the aim is to have a more focused approach and a series of workshops will be held this year, with the aim of “taking stock of the –omics” to see how the data they are generating can be translated across to personalised approaches. These deliberations contribute to a conference that will be organised next year in view of identifying the needs to progress this area from a research point of view”, she concluded.