By MaryAnne Rizk, PhD., Vice President, Global CRO Partnerships, Oracle Health Sciences
One of the great challenges for the effective use of pertinent data from multiple sources is to be able to integrate, standardize and normalize those data, which include clinical, outcomes, financial, administrative and genomic data into a single repository upon which one can query and analyze to create actionable information. What’s needed to advance personalized or individualized medicine is the effective utilization of all available data to develop the optimal treatment plan for an individual patient for improved outcomes. Genomic biomarkers are becoming the Holy Grail for Pharma and Biotech companies to find these right treatments and for clinical care providers to improve patient care.
In many diseases, there is a direct link between certain genes and the disease itself. These genes, as well as the proteins that are derived from them, are collectively referred to as “biomarkers” and often are indicative of the presence of a disease, the potential to develop a disease or indicators for targeted drug treatments. The costs for whole genome analysis, targeted DNA sequencing and other molecular diagnostic tests are dropping, the sophistication of the tests is increasing and more tests are being developed, all of which are having a positive effect on R&D and clinical care. With biomarkers being the first step in personalized medicine, Pharma and Biotech companies are aggressively incorporating biomarkers into their R&D efforts and clinical research programs.
The Center for Drug Evaluation and Research (CDER) at the FDA recently boasted of the Department’s strides in precision medicine, demonstrating that since 2012, CDER has approved thirty targeted (biomarker –related) therapies, eight of which were approved in 2014. Personalized medicine is rapidly coming of age, with 42% of all compounds and 73% of oncology compounds in the Pharma development pipeline have the potential to be personalized medicines. In addition, Pharma has nearly doubled their R&D investment in personalized medicines over the past five years, and expect to increase their investment by an additional one-third in the next five years. Also, Biopharmaceutical researchers are predicting a 69% increase in the number of personalized medicines in development over the next five years.
The importance of biomarkers in personalized medicine is to define which patients would benefit most from which therapies, at the right dose with the lowest risk. During clinical development, Pharma and Biotech companies are now using the presence or absence of biomarkers as key inclusion/exclusion criteria for their clinical trials. Taking this approach, along with greater use of adaptive study designs, are being employed to improve the chances of including the right patients that have the best chance of demonstrating a beneficial effect of the experimental drug being tested, thereby reducing the time, cost and risk of development and accelerating the availability of effective treatments sooner.
As clinical development proceeds, Pharma, Biotech and CRO’s are expanding their data needs to include clinical outcomes, comparative efficacy and cost effectiveness. Historically, these needs could only be partially met, since the technology to effectively integrate these data from multiple disparate sources didn’t exist, or was too cumbersome.
Personalized drug safety, the practice of determining which patients are more likely to experience a serious adverse event, is another area of focus for Pharma, Biotech and CRO’s. Patients who experienced a serious adverse reaction to an experimental drug in a clinical trial are evaluated in an attempt to identify biomarkers that may be correlated to that event/reaction. Once a biomarker has been shown to be correlated with an untoward event, that biomarker can be used to exclude patients from being treated with a given drug or prevented from being enrolled in a clinical trial. The effective use of biomarkers as a risk/safety mitigation tool can be extremely beneficial by excluding patients that may be at risk for an untoward event from being exposed to the drug, thereby avoiding adverse events that could be very costly.
In any clinical research project for any Pharma or Biotech company, mitigating risk, reducing time, minimizing cost and improving the probability that the right patients will be enrolled to optimize beneficial effects are critical to the overall success of the project. For the scientific and healthcare community, it’s exciting to see precision medicine and the use of biomarkers “going mainstream” since improving patient care, optimizing outcomes and reducing costs are common goals. Cracking the code on personalized medicine takes a true collaboration between Pharma/Biotech and Healthcare organizations. Creating a technology platform that fosters the collaboration of all key stakeholders, Pharma/Biotech, Healthcare and CROs provides the basis for the growth of personalized medicine.