For this week's 'Thoughts from the Centre' we're delighted to share an article written by a US colleague: Terri Cooper - PhD, Principal, Federal Health Sector Leader, Deloitte Consulting LLP. Terri's 'My Take' discusses the challenges surrounding precision medicine and how recent trends are helping to unlock its potential.
Twenty-six years ago, researchers announced they had discovered the gene that causes cystic fibrosis (CF).1 Just three years ago, the US Food and Drug Administration (FDA) approved Ivacaftor (Kalydeco"), the first drug to target the underlying cause of CF: a faulty gene and its protein product. KalydecoTM is now approved for use in about 10 percent of patients who have particular genetic mutations. Though there is more work to be done to develop targeted therapies for patients with CF, the discovery of the gene and the decades-long quest for a targeted, effective treatment is a precision medicine success story.
Some diseases like CF and Huntington's disease stem from an error in a single gene. Unfortunately, many other, more common conditions (e.g., depression, autism and schizophrenia) likely result from the interaction of several genes and other factors. These can take years to identify. And even in the case of CF, multiple therapies may need to be developed to target different mutations. As another example, because cancers are caused by multiple mutations, it has long been recognized that it is not a disease that can be treated with a single therapy. Even cancers of the same tissue or organ vary significantly in their molecular details and have different responses to treatments.
Precision medicine offers the potential for more targeted therapies - targeting treatment to positively responding patients - and reducing adverse events. A recent report from the Tufts Center for the Study of Drug Development shows that investment in precision medicine has nearly doubled in the last five years.2Despite this growth in investment, we would be remiss to forget that targeted treatments still have to go through the rigorous and costly research and development process. And because the treatments may only be used for a small subset of patients, the return on investment is potentially reduced. It is both in spite of and because of these challenges that momentum around precision medicine is gathering like never before.
The President announced the Precision Medicine initiative in January, and funding for it is included in the...