By: Katherine Bonter
CLINICAL TRIALS
CLINICAL RESEARCH AND PATIENT ENGAGEMENT IN
PERSONALIZED MEDICINE
It is largely accepted now that we are in the midst of a major transformation in drug discovery, clinical development and clinical care, one that has the potential to deliver substantial health, social and economic benefits.
14 BIOTECHNOLOGY FOCUS May 2013
By integrating molecular information with an individual’s medical or personal information, personalized medicine (PM) is a key factor in this transformation. Innovations like Pfizer’s Xalkori, Roche’s Zelboraf, and Vertex’s Kalydeco demonstrate the types of impacts that this transformation provides. PM can shorten approval times and speed drugs to market. For example, Zelboraf and the companion BRAF V600E test were approved ahead of their goal dates, and the FDA PMA review was less than four months compared to the standard of nine to 18 months. Xalkori was on the market only four years after the discovery of its target and companion diagnostic (ALK) and reviewed and approved by the FDA in less than five months. Furthermore, these drugs have been welcomed by patients, physicians and payors, despite their high cost. They have also provided unprecedented advances in treating two deadly cancers, metastatic melanoma (Zelboraf) and non-small cell lung cancer (Xalkori). Developing drugs that target small populations in partnership with patient organizations can be a recipe for success in PM. Kalydeco,
launched late in 2012, proved effective in individuals who carry a G551D mutation in cystic fibrosis (CF) gene, which accounts for only four to five per cent of CF cases. Although the CF gene was discovered in 1989, by a team that included Lap-Chee Tsui from Toronto’s Hospital for Sick Children and Francis Collins (now director of the NHI and a prominent advocate for PM), Kalydeco is the first drug to target the defects caused by mutations in the CF gene. The early development of the cystic fibrosis drug Kalydeco by Vertex was funded and supported by the CF Foundation in exchange for a royalty on future sales.1 This was critical for the success of Kalydeco and has set a model for similar partnerships between other foundations/patient groups and drug developers. In all of this, what is the role of patients, or even the general public? How can they participate in this transformation? What contributions can patients make to accelerate progress? Co-creating value with patients, healthcare consumers, tapping into their knowledge of disease and healthcare, is increasingly a potential driver of clinical innovation. In
