The Issue

What is Personalized Medicine?

The goal of personalized medicine (PM) is to tailor medical treatments and services to the biological makeup of individual patients. Healthcare provides can select therapies or clinical management strategies based on a diagnostic test using biomarkers. Such tests provide the molecular or genetic profile to identify what makes a person susceptible to certain diseases or conditions and whether they are likely to respond to a specific therapy.

PM is believed to have the potential to improve patient care at the same time as saving money for healthcare systems through increased efficiencies in patient care. The strategy may minimize adverse effects from therapies that do not work or that may be harmful in a particular patient. PM technologies are being developed for many diseases and conditions, such as cancer and heart disease.

The key to PM technologies is that they comprise a therapy, service or clinical management decision based on a molecular diagnostic test. The test and the treatment are sometimes developed together, but often, are not. Sometimes the diagnostic test is developed after the therapy, for example, the diagnostic test to determine the appropriate of dose of warfarin, a common blood-thinner. Sometimes the diagnostic test is developed first, for example, for a rare genetic disease, in the hope that a therapy will become available in the future.

The issue

One key issue for the development and clinical use of PM technologies is lack of coordination in research and development, which leads to problems along the translational pathway as research moves from the bench to the bedside. There is an urgent need to improve inter-technology co-ordination of evidence development, regulation, and appraisal during clinical translation of PM technologies. Failure in co-ordination has significant implications for the cost of bringing a PM technology to market, the likelihood of a positive reimbursement decision, and the duration of post-market patent life. All of which reduce the return on public and private sector investments.

To become a healthcare product available for patients, a new technology must follow a non-linear and iterative, innovation pathway, where a series of institutional, structural, regulatory, behavioural, financial, and political factors contribute to the probability of success. Currently, the clinical translational pathway is made up of a series of distinct translational steps comprised of siloed blocks of research and research evaluation activity. These are characterized by poor integration and communication between stakeholders in each block.

Whilst meeting the evaluative targets for each step of the translational pathway is necessary for a new technology to advance, it does not determine whether the next stage of translation represents a good investment, from a commercial, health system or social perspective. Products that progress to the final stages of development but are not adopted by healthcare systems represent a valuable loss of time, money and effort.

There are multiple points along the translation pathway where PM stakeholders – researchers, developers, investors, health and safety regulators, Health Technology Assessment (HTA) bodies, and healthcare systems – must make crucial decisions about which technologies to support, invest in, adopt and reimburse. Decisions to proceed should be based, as far as possible, on a realistic appraisal of whether a piece of new knowledge or technological development can be translated into a marketable health care product. Most importantly, this requires an assessment of whether a health care payer will reimburse the technology at a price sufficient to generate a competitive return. Developers can no longer assume that technologies that make it through regulatory approval will be adopted and paid for; rather, they must respond to the needs of cash-strapped health systems, the largest market for PM technologies.

Read more about the PACEOMICS approach to addressing these issues in an integrated manner.