IP Review Autumn 2017

Patenting precision medicines Precision Medicines 14 IP review autumn 2017 Introduction Traditionally, pharmaceutical products have been designed as “one-size-fits- all”, working across as wide a patient population as possible. Medications that produced no effect in a large number of patients, or even produced severe side effects in some, often would not make it to market or would not survive once on the market. One unfortunate example is rofecoxib (Vioxx ® ). This was a leading osteoarthritis treatment up until a high-profile withdrawal due to allegedly inducing heart attacks in a small number of patients. Even where there are no side effects, if a patient does not respond to a medication, valuable time can be lost before switching to an alternative treatment, as can occur when prescribing cancer therapies. Precision medicine aims to predict how individual patients will respond to a medication, and thereby provide medications that are more tailored to the patient. This prediction is often done by identifying specific biomarkers that are linked to the patient’s condition and to the way the drug works. There are many biomarkers that can be used to make such a prediction, such as the presence of specific gene sequences, proteins or metabolites, or even a multiple biomarker signature. Once identified, these biomarkers can allow for a faster and more cost-effective precision medicine treatment approach. One well-known example of precision medicine at work is the use of the drug trastuzumab (Herceptin ® ) for treating breast cancer. The drug is only effective in patients where the HER2 gene is overexpressed, while in other breast cancer patients there is no effect or sometimes even a harmful effect. By testing for overexpression of HER2 in breast cancer patients, it will be known in advance if trastuzumab is likely to provide any benefit. More recently, the FDA approved Merck’s pembrolizumab (Keytruda ® ) for treating solid tumours that have a specific biomarker referred to as MSI-H or dMMR. This appears to be the first time a drug has been approved for a disease characterised only by a biomarker. Some commentators predict that the pharmaceutical industry will move further in this direction, particularly with the recent recommendation by England’s Chief Medical Officer that cancer patients should be routinely offered DNA tests to help with selecting the most appropriate treatments.