Speech by FG Deputy, Jerry Buttimer on Thursday, 21st March 2013
European Alliance for Personalised Medicine
Innovation and Patient Access to Personalised Medicine
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The Challenge of Personalised Medicine
If it were not for the great variability among individuals, medicine might as well be a science and not an art. –Sir William Osler 1892
That may have been true in 1892 and it may have been in true in 1992 but today medicine has become a science. Over the last twenty years our increased knowledge of biology, genetics and pharmacology has given us great insights into the operation and manipulation of the human body. At the same time technology has advanced, devices have become smaller, new materials have been developed. The convergence of these advances has created the concept of personalised medicine; matching drugs, dosage and timing to maximise clinical efficacy and to minimise toxicity.
The challenge of personalised medicine requires all stakeholders, clinicians, industry, regulators, Governments and policy makers, to set out a plan for personalised medicine, to deliver funding models which maximise the reach and benefit of personalised medicine.
From a policy perspective the increasing personalisation of medicine raises some very serious questions. While the possibilities seem great it raises practical challenges for the funding models used in healthcare systems.
Funding challenges for Personalised Medicine
Although personalised medicine has the potential to deliver very significant health benefits it also has the potential to increase costs. Drugs are now being developed to target smaller groups of patients, often with the same, or even greater, outlay required in the development stage as for drugs targeted at larger groups. The consequence of this is that the cost of the drug or treatment for the patient is significantly higher.
Recently in Ireland these very issues have arisen. The very high cost of Kalydeco, a drug for the treatment of a specific Cystic Fibrosis mutation, costing about €234,000 a year for each patient, has been a political issue. It eventually took direct Ministerial intervention to approve the drug, a procedure usually left to administrators.
As drugs become more specific it is likely that these kinds of problems will become increasingly familiar. Smaller numbers of patients benefitting from new drugs reduces the economic return and consequently puts at risk the financial investment from industry.
Before we advance much further down the road of personalised medicines we must, as a society, address some ethical and social considerations. The questions that need to be answered include how to we determine the cost-to-benefit ratio and what changes need to be made to our systems of reimbursement. These questions raise political issues similar to those posed in the Kalydeco example.
If we are to maximise the benefits of personalised medicine our reimbursement systems must be adjusted. We must ensure thatthere is a sufficient return for those who invest in product development. Without sufficient returns will there be financial incentives to encourage the continued advancement of personalised medicine?
Money Follows the Patient
Here in Ireland the funding of public hospital care is moving away from a system of inefficient block grant budgets to a new system where hospitals are paid for the actual level of activity undertaken. It is Government policy to deliver a Money Follows the Patient system, where hospitals will be funded for the services actually delivered to patients.
The idea behind the change is to encourage greater efficiency and productivity. Under the new system providers will be paid for the needs they address, the quantity and quality of the services they provide and the outcomes they deliver. This change is seen as a building block for universal health insurance which is the ultimate aim of Government policy.
Stage one of this policy is setting national prices for services and determining the overall budget. It is this stage of the new policy which has the greatest potential for personalised medicine. The Money Follows the Patient model will be a case-based funding model using Diagnosis Related Groups. Internationally there is a strong convergence toward the implementation of such a system as it provides incentives for greater efficiency and more fairly and transparently allocated resources.
But what about high cost cases, outliers? What about the use of innovative personalised medicines?
The policy proposal will take account of this; it will recognise that high cost cases occur and that high cost drugs and treatments are sometimes required. Supplementary funding for certain high cost drugs will also need to be considered, while taking account of existing initiatives and the increasing role of technology.
The Minister is currently in the middle of a consultation on this Money Follows the Patient policy. I hope that this process will further expand on the issues that need to be considered, in particular the increased potential of personalised medicine.
Personalised Medicine and Technology
The issue of funding is not only relevant to the delivery of personalised medicine to the patient. At the early stages of research and development funding is also an issue. Should it be left to research institutes and commercial bodies to dictate the allocation of funding or should Departments of Health play an active role. For many Departments of Health this would be a huge change, having mainly being involved in the delivery of healthcare rather than the development of medicine and technology.
Only last week I visited Tyndall National Institute, one of Europe’s leading centres for information and communications technology research, in my own constituency of Cork South Central. The purpose of the meeting was to meet the new CEO, Kieran Drain, and to discuss the issues are facing Tyndall. During the course of that meeting discussion turned to one of the large fields of research being undertaken there, the convergence of technology into healthcare applications.
One of the key benefits of this convergence is that it will enable the personalised and targeted treatment of illness, at Tyndall they are continuing to advance personalised medicine.
Researchers in Tyndall, and in many other similar institutes across Europe and the globe, are working to provide new solutions for cancer, cardiovascular disease, neurodegenerative diseases, diabetes and various other diseases. This work is being carried out against a backdrop of an aging population and increasing costs of providing healthcare. Its purpose is to use technological advances to deliver affordable healthcare solutions which are targeted, more effective and accessible to the wider population.
This research is not being conducted in isolation; it is being done in collaboration with other research groups and clinicians based in the Royal College of Surgeons in Ireland. Already it has had some clear benefits for personalised medicine, enabling the choice of treatment and dosage to be customised for each patient to ensure optimal effectiveness.
One innovation at Tyndall is a radiation field effect transistor which has been adapted for healthcare application and is already commercialised. This device can be placed in a body or as an implant to accurately control the radiation dosage delivered at the target site.
In the area of pharmacogenetics Tyndall has developed a prototype system for near-patient genetic testing, enabling a patient’s genetic profile to be determined from a finger-stick blood sample. This new technology will facilitate the customisation of a prescribed therapy programme. An advantage of developing ‘companion diagnostics’ will be that it will allow regulatory agencies to licence drugs which have no effect or adverse effects on portions of the population knowing that they will only be used on those whom will benefit.
Where to next?
Increasingly we are moving away from blockbuster drugs, where one-size-fits-all, the focus of medicine is shifting from the general to the personal. As these changes are occurring we must move towards a system where the increased presence of personalised medicine can be matched by appropriate funding models. Our reimbursement systems must facilitate this transition to personalised medicine.
In the last century the major breakthroughs were in the areas of mass vaccination, today’s advances are often targeted to specific groups of patients, to specific mutations. The same funding models which were appropriate for large scale programmes may not be as effective in delivering access to personalised medicine.