OHE Involved in Developing New AMS Report on Stratified Medicines

Prof Adrian Towse, OHE’s Director, joined Professor Sir John Bell and Professor Andrew Morris at the Science Media Centre last week to launch Realising the Potential of Stratified Medicine, a new report by the UK’s Academy of Medical Sciences. Adrian and Prof Lou Garrison, OHE Visiting Senior Research Fellow, were two of a dozen experts who oversaw the preparation of the report. Towse and Garrison chaired the two working groups examining pricing and reimbursement issues.

Stratified medicine, according to the report, “is the grouping of patients based on risk of disease or response to therapy by using diagnostic tests or techniques”. Its benefit for patients and health care providers is more targeted and effective treatments. The biopharmaceutical industry benefits from both a potentially more efficient development process as well as markets for these new treatments.

The importance of stratified medicine is clear from the case studies included in the report. For example, it can identify the 2–9% of patients who have a genetic biomarker for hypersensitivity to Ziagen® (abacavir), an HIV medication, thereby avoiding severe reactions. Similarly, Xalkori® (crizotinib) is markedly more effective than alternative treatments for a subset of patients with a particular strain of lung cancer, identified by genetic testing.

The report finds, however, that existing regulatory and pricing systems are not designed to enable the effective, widespread adoption of a stratified, and increasingly personalised, approach to medicine—and instead are imposing serious barriers. Under current pricing systems, for example, the price of a medicine remains the same or decreases after it reaches the market. Raising the price in response to the development of a new, targeted use is not possible, missing an important incentive for developing increasingly personalised medicines. Similarly, new incentives are essential for the development of the diagnostic tests that are the backbone of stratified and personalised medicine.

The pricing and reimbursement recommendations included in the report include the following.

  • Introducing flexible pricing for drugs and prices reflective of value for diagnostics. This is to address scenarios in which the drug and diagnostic are launched together, as co-dependent technologies, and when the potential for better targeting of the medicine only becomes clearer after marketing.
  • Developling approaches whereby health technology assessment of diagnostics can appropriately value platform technologies, such as human genome sequencing, that can test for a number of different ways of stratifying populations in different disease areas.
  • Apportioning value between the drug and the diagnostic appropriately. For economists the value is jointly created and any attribution is arbitrary. However, the way that drugs and diagnostics are priced to reflect this value can have significant implications for the rate of future innovation.
  • Recognising that evidence requirements for companion diagnostics need to be flexible to limit the complexities added to drug development.  More accurate tests are crucial to improving health, yet the resources available to diagnostic manufacturers to undertake independent clinical studies are limited. Coverage with evidence development may be one route forwards to earlier access to important diagnostics.
  • Exploring how diagnostic manufacturers can receive better intellectual property or data exclusivity protection to create incentives for generating better evidence of clinical value. One approach would be NHS use of diagnostic kits with an evidence base in preference to in-house tests with no such evidence base.

The report also emphasises the critical need to harmonise and link clinical databases and biobanks, nationally and worldwide, and to follow proposed “Good Genomic Practice” guidelines in collecting such data to maximise its research potential.

For the use of stratified and personalised medicines to become an everyday practice in health care, the report notes, a wide range of groups must become involved in implementing the report’s recommendations. This includes thought leaders in academia, policy makers in health care systems, the private sector, research funders, licensing authorities and regulators, health technology assessment bodies and patients’ groups. The report calls for the expansion of the UK Stratified Medicine Innovation Platform to provide the framework for bringing all stakeholders together, unifying existing initiatives and co-ordinating future activities.

Copies of the report are available for download from The Academy of Medical Sciences. OHE has been involved in exploring many of these issues recently including the following, for example.

Posted in Drug Development/R&D, Health Care Systems, Health Technology Assessment, Innovation, Precision Medicine | Tagged