Can Personalized Medicine Ever Truly Become A Reality?
By Rob Wright, Chief Editor, Life Science Leader
Follow Me On Twitter @RfwrightLSL
On October 6, 2014, I received an invitation from Poppy MacDonald, president and publisher of the National Journal, for a private dinner conversation on the topic of personalized medicine with AstraZeneca’s Dave Fredrickson, VP of specialty care and William Mongan, VP of business development, new product planning and foundations portfolio. Leading the on-the-record discussion would be Marilyn Werber Serafini, VP for policy at the Alliance for Health Reform. Wanting to be well prepared, I thought it a good idea to seek some fresh personal medicine perspectives from a cross section of folks within my network. Sharing a list of exploratory questions, here is what they had to say.
Personalized Medicine – Views From The C-Suite
One of the questions on the agenda for discussion is, “What advantages will personalized medicine bring to patients, and what is its overall public health benefit?” to the chief medical officer of a top 50 pharmaceutical company. Their insights are, “Personalized (or precision) medicine ultimately leads to a decreased health burden to society. For patients, personalized medicine means improved personal outcomes to enable them to live longer lives with improved quality. Better compliance can be achieved with personalized medicine. If patients are given a therapy that is better matched for them, they will feel better and stay on therapy. Compliance enables better offset of healthcare costs incurred as a result of patients switching therapies, skipping doses or avoiding therapy.
Personalized medicine leads to an overall reduction in healthcare costs and improvement to society where patients can return to work or other activities. The reduction in healthcare costs is derived from: 1) less adverse events for patients, 2) more efficient means of determining treatment for patients and 3) improved response to therapies.”
I posed another planned discussion question, “How are the country’s healthcare stakeholders collaborating to provide more effective, precise treatments,” to the CEO of a clinical stage anti-infective company who shared, “Not very well. In fact, funders are on one side, developers are on the other. Aligned with funders are public health officials responsible for health of populations: they look for efficient delivery, not innovation, and cost is a major factor in applying healthcare to large populations. Aligned with the product developers are the individual healthcare providers who generally want to cure, not harm, their patients. In the middle are the health ethicists, who are concerned that too much genetic information about an individual will encourage insurance companies denying coverage to people with certain genetic signatures, regardless of whether their genetics has (or will) result in disease (there are always environmental factors that modify genetic control, whether for good or bad).
The chief medical officer shared multiple examples on how U.S. healthcare stakeholders are collaborating to provide more effective treatments, such as the Friends of Cancer Research (FOCR) partnering with the FDA to address key regulatory challenges. “Specifically, a consortium was formed to develop a white paper which outlined proposals to FDA on a regulatory pathway for Next Generation Sequencing (NGS)—a newer technology being broadly used by academic institutions to test patients for a broad set of genetic mutations.” The chief medical officer also attributes the concept of basket trials to successful collaboration. “These are trials which provide greater efficiency for developing targeted therapies,” they told me. “The concept was created through the partnership of FDA with academic institutions and NCI. The premise is to align patients who carry a specific therapeutic target with a specific drug since this results in higher response rates than have been previously seen.”
What Innovations Are Needed To Create Better Diagnostics?
Another planned discussion question, “What innovations are needed to create better diagnostics,” gained responses such as next-generation sequencing (i.e., high throughput sequencing of genes via a panel) and liquid biopsy (i.e., an emerging technology creating the means for less invasive sampling for patients). Although the CEO of the clinical stage anti-infective company feels there is a need for much more information on the factors that influence expression of genetic abnormalities before we will be able to create true “diagnostics” for disease, they reminded me of the importance of communicating the benefits of existing technologies. “Think of the huge advances we’ve made with vaccination against former infectious plagues, improving lives and life spans. And then think of the (up to 55 percent in some states) parents who opt out of vaccinating their children for ’personal reasons.’ We need to educate before we pontificate.”
A currently practicing OB/Gyn who also holds a Master of Science in Public Health (M.P.H.) seems to agree with the CEO. “One thing I wonder about with regard to targeted genes is that just because you carry a certain gene does not mean you will necessarily end up with a disease. Simple things like decreasing stress/increasing relaxation, diet and exercise have all been shown to turn certain ’bad’ genes off and ‘good’ genes on. We already know we can improve immunity, decrease many cancers, and prevent diabetes and heart disease through behavioral modifications such as not smoking, diet and exercise. So why dream of testing everyone when a long-term prevention program is better. When I discuss genetic testing with patients, let’s say their BRCA [a gene blood test that uses DNA analysis to identify harmful change in either one of the two breast cancer susceptibility genes — BRCA1 and BRCA2] comes back positive. What will you do with that information, lop off your breast or live knowing you have a high, but not a 100 percent risk of developing breast cancer?”
Thoughts On Personalize Medicine Reimbursement And Regulatory Roadblocks
“Can payers create reimbursement policies that support this new approach,” is another planned discussion question which I shared with a former SVP and president of a top 10 pharmaceutical company’s global R&D department, who shared a succinct response, “Absolutely! Payers are far more likely to reimburse when the drug has a very high chance of being effective.” However, the chief medical officer elaborated further sharing their belief for contemplating new business models that view the companion diagnostic and drug as a single entity, as both create value for the healthcare system when used together. Further, the CEO feels we need to think long-term when it comes to reimbursement policies. “In the long run, we can either pay for innovation now and get better cures, or later pay the astronomical end-of-life costs that result from no cure. What we don’t do today will come back to bite us in the future. Short-term gains are now the norm, and this is not likely to change until we correct Wall Street’s view of short-term profits and rewarding payers for short-term cost savings.”
The final discussion question shared with me by Poppy MacDonald is, “What are the regulatory roadblocks ahead?” The former global head of R&D thinks the FDA is more open to personalized medicine as an approach and thus why personalized medicine therapeutics are getting priority reviews, particularly in oncology. According to the chief medical officer, the industry needs greater clarity on a global basis for the support of codevelopment programs. Although companion diagnostics and their targeted therapies are available in markets outside of U.S., non-U.S. regulatory bodies are behind in providing clear regulatory guidance for codevelopment programs. “Newer technologies such as Next Generation Sequencing do not yet have a defined regulatory pathway for approval. Given the rapid broad use of NGS for research, clinical investigations and assignment of drug therapy, a regulatory approved pathway is needed to ensure accurate test results for patients.” To reward innovation and encourage the development of new companion diagnostic tests, the chief medical officer believes there needs to be a level playing field for the regulatory review of laboratory developed tests (LDT) versus tests developed by diagnostic manufacturers as companion diagnostics. “Today, higher hurdles are in place for companion diagnostic tests developed by diagnostic manufacturers. In contrast, there has been no regulatory review of laboratory-developed tests used for the purposes of a companion diagnostic. Regardless of the LDT or diagnostic test offered by diagnostic manufacturers, greater flexibility for test approval is needed while still ensuring patients have timely access to accurate tests.” One regulatory roadblock the CEO sees is skepticism. “Until we can definitively link a genetic test to an outcome, either a diagnosis or a prognosis, the regulators will be skeptical about benefit (rightly so) and reluctant to push the envelope on new regulations that are more supportive. The buck stops at the product developer’s doorstep. Make your case, but do it with excellent science.”
I am looking forward to learning more about personalized medicine at this upcoming event. Thanks to my industry colleagues for helping me prepare, and I look forward to sharing additional insights gained from my participation.