Bob Damms and Hakim Yadi discuss the potential of stratified medicine, which is where researchers look, in detail, at the cells of cancer patients in order to find ways to improve treatments. This is one step towards personalised medicines. In this article, Bob and Hakim look specifically at Cancer Research UK’s new stratified medicine programme.
The potential of stratified medicine has long been talked about, but the pace of development has increased dramatically in recent years. We have most of the science and technology in place to make it a reality, but we now need the regulatory and reimbursement regimes to catch up.
Good progress is being made in developing the systems and infrastructure that will make personalised treatment available to patients. In oncology, Cancer Research UK has been working in partnership with the NHS, industry and researchers to establish a system for large-scale genetic testing for cancer. The first phase of this project, which will run until July 2013, is demonstrating, on a small scale, how the NHS could provide a routine molecular diagnosis for all cancer types in the future.
The project focuses on six tumour types: breast, bowel, lung, prostate, ovarian and melanoma, with patients asked to give permission for surplus tissue from tumour biopsies to be analysed by genetic testing labs. This information is then being used to explore the links between the presence and absence of particular genes and gene mutations and the impact of using different therapeutic treatment regimens. The data will be stored in a NHS data repository that can be accessed by researchers and clinicians to help them identify patients that will benefit from genetically tailored treatments. This should also facilitate the identification of patients who can take part in clinical trials of new treatments based on genetics.
We already have the ability to identify common genetic mutations in breast, lung and colorectal cancers. The next step is to ensure that the development of diagnostic products keeps up with the research. That means focusing on interoperability between products; providing diagnostics at the point of care, and supplying results rapidly in ways that are easy for clinicians to use.
However, perhaps the biggest challenge is in the regulation of stratified medicine. It is going to change the way new drugs are developed and offers the potential to reduce development times, lower failure rates and decrease costs. In particular, the design of clinical trials for personalised solutions is very different to that for conventional medicines. If developers know the drug will only work in a small number of people then the large scale, and consequently highly expensive, Phase 3 clinical trials may not be needed, reducing development time and cost significantly. However, at the moment, in general even if a drug is only going to be suitable for a few thousand people, the regulatory requirements can be as onerous for one that is going to be used by the general population. Regulators need to respond to the smarter science by creating a smarter process that is appropriate for a smaller number of people, but still ensures patient safety.
The next challenge is reimbursement. Those who pay for medicines are still focused on drugs that work for large numbers of people. New models have emerged that are based on ‘payment by results’, but these have been driven by cost concerns over high cost oncologics rather than by the sector embracing a future of stratified medicine. The reimbursement bodies are yet to industrialise the process of approving stratified medicines, it still remains a cottage based industry driven by individual submissions. The reimbursement bodies will need to develop payment systems that respond to the much smaller numbers who can be treated by stratified medicine.
So there is a real need to develop truly outcomes-based payment systems that reflect a targeted approach. To deliver on this targeted approach requires much richer engagement and access with the clinical and economic data that supports the evaluation of health care outcomes. Historically, health economics has never been considered fashionable, but now it has the potential to take the headline. There is much more work to be done to install the processes and tools to collect the evidence that will support such a system and provide assurances about value for money.
Stratified medicine is also going to change business models of pharmaceutical companies. They are already facing huge challenges from global competition; from patent expiry, and the failure to produce new block buster drugs. Stratified medicine offers the opportunity to do something different. It is hard at this stage to predict whether this will lead to consolidation as big companies try to get control of these developments or whether we will see smaller more nimble companies take the opportunities to develop smaller scale drugs, but it is clear the commercial environment will evolve.
All these factors means that for the potential of stratified medicine to be realised we will need a revolution. We will need a revolution in the attitudes of regulators, and this will have to be at a global level, to provide a level playing field across the world. We will need a revolution in reimbursement systems and business models, and in attitudes to create a system that funds new medicines which, by definition, will not be used for the majority. All this will have to be supported by the research-based pharmaceutical industry revolutionising what it does and the way it gathers evidence on the effectiveness of its products.
The potential is huge and could make a real difference to patients. The science is well on the way to making this a reality, but that is only the first step. For the full potential of stratified medicine to be realised, all those across the health sector will need to be committed to changing the way they work to make it happen. Importantly, the role of governments should not be overlooked; policy decisions on the clinical trial and regulatory environment will play an integral role in moving this sector from a concept to reality.
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