“PhRMA’s Outlook on Personalized Medicines”
Dr. William W. Chin, M.D.
Executive Vice President, Science & Regulatory Affairs
The Pharmaceutical Research Manufacturers of America
9th Annual Personalized Medicine Conference
Wednesday, 6 November 2013
Thank you, Dick (Maas). Also, thank Raju and the other for inviting me to be part of this year’s program.
The last time I was here at this meeting I was speaking from the translational medicine perspective as the Executive Dean for Research at the Harvard Medical School but I am excited to be speaking today from the perspective of the biopharmaceutical industry, which is equally committed to healing and meeting the health needs of patients through advancing personalized medicine.
Each of today’s speakers and panelists, like every attendee at this conference, is excited by the power and potential of personalized medicine. It is the grand experiment in medicine that will, I think, define our age. It is the future of medicine, when diagnoses are more precise, and therapies are suited for the specific diagnoses. It is quintessential patient-centered.
Indeed, it is already changing everything we in do – from how we approach once seemingly intractable medical challenges to how we research and development new medicines and how we reach patients as well as their interactions with physician and our healthcare delivery system.
Every one of us also feels the urgency of realizing the potential and power of personalized medicine and making it a reality.
We’ve also heard that urgency from tens of thousands of patients. Patients like Adriana Jenkins and Stephanie Haney. Adriana -- a breast cancer patient whose life was prolonged by a personalized medicine, Herceptin – but ultimately she lost her battle against the disease. In a 2011 commentary in Forbes she said her dying wish was that biopharmaceutical research companies do more to bring forward the next generation of targeted therapies. Stephanie is a lung cancer patient who recently spoke on Capitol Hill on behalf of the Personalized Medicine Coalition.
She told legislators and staff that she is alive today because of personalized medicine, and called on policymakers to help accelerate targeted therapies to help others struggling with life threatening diseases.
And, we’re here this week because we know that realizing the potential of personalized medicine is as difficult as it is urgent. The science is complex – it seems the more we learn about the influence of genetics and other non-genetic factors on disease and treatment response, the more we realize how much there still is to learn. The technology is, in short, disruptive, posing new challenges to business models, regulation, and policy.
Today I want to offer PhRMA’s and the biopharmaceutical research sector’s perspective on how we can more fully realize the opportunities personalize medicine offers us as we seek new solutions for patients and strive to improve the nation’s health care system.
About personalized medicine, I will:
- Describe the industry’s profound and long-term commitment;
- Discuss the impact of industry’s work today,
- Show how the exciting ways it could transform both the practice of medicine and the health care system tomorrow – a topic well-known to you all.
- Talk about some of the barriers we face that are slowing progress in personalized medicine.
- And conclude with some thoughts on how we can all work together to remove or overcome those barriers
1. The Biopharmaceutical Research Sector is committed to Advancing Personalized Medicines.
I want to begin the discussion of the biopharmaceutical research industry’s commitment to personalized medicine with an observation.
There was once significant and even understandable skepticism about industry’s interest in personalized medicine. I lived it during my time at Lilly.
The tantalizing prospect of more personalized medicines butted-up against how industry went about its business and its very business model. In a word, the blockbuster was king.
That model for R&D was well understood in the laboratories, in board-rooms and among investors. It had proven its benefit to both patients and its power to generate investment and fuel new R&D.
It also fit well with the practice of medicine and the healthcare system delivery system.
But survival in an innovation and technology-based business, like other facets of life, requires evolution. Science necessarily drives what our industry can do and how we do it.
Science and technology evolve. It is constantly changing. Experimental successes and failures suggest new approaches and new solutions. Whether in academia, industry or government, we all must be open to change, be able to absorb change and move with it – even if the end direction and the impact is not fully understood.
For industry, this meant confronting big questions. Most importantly: what is the business model for more personalized medicines?
The answers to these questions are also evolving. But biopharmaceutical research companies unable to change with the science ultimately fail patients and their very reason for existing.
Because we are a science-based industry, we constantly embrace the unknown alongside of our academic and other colleagues. The science tells us what is possible. Patients’ need drives our research. The industry has needed to quickly and dramatically change as a result.
The Tufts Center for the Study of Drug Development industry survey a few years ago demonstrated that industry is adapting, adopting and embracing the science and the technology.
Enormous resources are now going into R&D into personalized medicines. While the transition has not been easy it has been moving apace, driven forward by promising results.
In particular, the Tufts survey found that biopharmaceutical sector companies increased their investment in personalized medicines by around 75 percent between 2005 and 2010, and expect to see an additional 53 percent increase from 2010 to 2015.
Of the companies surveyed, 94% said that they were investing in personalized medicine research and 100% said they are using biomarkers in the discovery stage to learn more about compounds. This research has required large investments in new tools and training.
Further, the commitment to researching and developing personalized medicines – highlighted by the survey – is bearing real therapeutic fruit.
FDA approval of innovative therapies for lung-cancer, cystic fibrosis and melanoma—just to name a few – underscore industry’s growing commitment to personalized medicine. Indeed, according the Personalized Medicine Coalition, there were 13 prominent examples of personalized medicine products in 2006. By 2011 there were 72. And, while not all were drugs, the trend is obvious.
In fact, looking at the entire pipeline, PhRMA member companies report that 12-50% of the compounds they are researching are potential personalized medicines.
Needless to say, this research fuels continued medical progress and the hopes of millions of patients.
2. Biopharmaceutical Research Companies Realizing Personalized Medicines
This strong involvement in personalized medicine should come as no surprise. PhRMA companies are driven by innovation and committed to providing patients with the highest quality care available, ensuring they have access to the medicines and treatments they need. Breakthroughs in personalized medicine are playing an increasingly important role in what we do.
To be sure, thirteen years after the sequencing of the human genome, personalized medicine has not yet transformed patient care to the extent all of us had hoped. But the investments that the industry and our colleagues in the ecosystem continue to make are yielding significant progress. You’ve heard about that progress from today’s speakers and panelists, and you will hear more tomorrow.
As we all know, the first personalized medicine for HIV was approved in 2007, and the HIV/AIDS and personalized medicine stories converged. Using a diagnostic test, health care providers can identify which patients will benefit from this drug, thus choosing the best treatment options for patients who may be resistant to or intolerant of other available therapies.
In another example, Gleevec has completely transformed the outlook for many patients with chronic myelogenous leukemia. 88% of patients on the medicine survived after six years of treatment. It is important to note that these benefits were not fully known when Gleevec was first introduced. FDA approval was based on evidence showing the drug had an effect at the cellular level. It wasn’t until six years later that studies confirmed the treatment’s stunning survival benefit.
Similarly, Herceptin was approved for breast cancer patients with HER2 positive tumors in 1998 and further research in 2005 showed that it reduced recurrence by 52% in combination with chemotherapy.
In addition, in just the last few years, an explosion in knowledge about the genetic mutations that cause tumors is offering real promise for the development of drugs for the treatment of lung cancer, one of the most pervasive and deadly of all cancers. By decoding the DNA in tumors, researchers are discovering that lung cancer is actually many diseases. In fact, they have identified many lung-cancer variations, opening the door to new, personalized drug regimens that might target each one.
And lung cancer is just the beginning. As the Wall Street Journal recently reported, researchers have used DNA analyses to discover scores of variations in breast, colon, kidney, skin and other cancers in recent years. As a result, of the nearly 1000 cancer drugs in clinical development last year, many are targeted therapies, aimed at genetic mutations.
3. Growing Impact of Personalized Medicines on Patients and Healthcare
As exciting as these breakthroughs have been, they truly are just the tip of the iceberg. A decade from now, I’m convinced personalized medicine could be viewed as one of the great turning points in the history of medicine. As I’ve said, it likely represents the future of medicine.
Ironically, it will be a turning point that takes us back …to the very origins of medicine. Oft quoted, Hippocrates said, “It's far more important to know what person the disease has than what disease the person has.” His vision was to provide care directed to the specific needs… of specific individuals… at a particular time and place.
Personalized medicines are helping us realize Hippocrates’ ideal as well as embodies the reasons the biopharmaceutical research sector exists: to search for solutions that help meet the healthcare needs of each patient, improve their lives, prevent disease, help them get healthy, stay healthy and live a longer, better life.
But in addition to the promise of personalized medicine for patients, it holds significant promise to make our health care system more efficient and effective and, overall, helping control and lower long-term healthcare costs.
Further, following the science and developing personalized medicines gives us all a better chance of meeting the enormous health care challenges we face – such as the human and economic toll of conditions like Alzheimer’s disease, cancers and diabetes.
For me, one of the most important changes this personalized paradigm has fostered is a shift from reactive to proactive medicine. Genomic science can provide us tools to identify peoples’ risk for disease before symptoms even develop, boosting prevention. Genomic tools can also transform diagnosis, allowing physicians to be much more precise, much more early on about whether a patient will benefit from a specific diagnosis and a specific treatment.
Let’s look at the impacts of these shifts.
- For the biopharmaceutical industry, the combined effect of the key elements of personalized medicine could very well be a profound transformation. In the future, I believe the industry is not going to be about developing new pills as merely chemicals or biologics. Rather, we will provide information about new medicines and treatments—information about who is the right person for the right medicine, what is the best way to deliver it, what does a particular diagnosis reveal and how the medicine can be used most effectively.
- For patients, access not only to new medicines but also to this new personalized information will bring new confidence that treatments will be safe and effective in protecting their health.
- And for the nation’s health care system, proactive medicine, combined with better safety and effectiveness, can help us meet the challenge of rising costs. As noted by Dr. Ralph Snyderman at Duke, “health care costs are less when appropriate interventions are taken before a catastrophic event occurs.”
4. Barriers to Personalized Medicine Reaching its Potential
At this conference, you’ve heard many other exciting opportunities for change that personalized medicine has opened up --- in research, in patient care, and in the health care system.
It is especially exciting for researchers investigating diseases where there was once little hope. In the fight against cancers, for example, we’ve learned more about changes within genes in cells and how they can lead to abnormal cell growth.
This opens the door to potential therapies that can attack defects and slow growth.
It gives us a glimpse of a possible pathway to a cure. But cancer is one thing, but vexing diseases such diabetes mellitus, cardiovascular disease, etc, are less tractable. Still others such as depression and schizophrenia are yet even more challenging as we know much less about causal factors.
Validating biomarkers and their related diagnostics, for instance, may be a key to future success. But realizing this potential requires we evolve how we in industry, government and academia work together.
The Biomarkers Consortium, among other efforts in this field, is an example of an effort that PhRMA supports to combine resources to scale up and speed up research to enable personalized medicine. This public/private partnership among the Foundation for NIH, PhRMA, FDA, and others is working to discover and validate biomarkers and has already made important findings, such as the discovery of a predictive biomarker for Type 2 diabetes.
But, unfortunately, as we meet here today, scientists, researchers, patients and practitioners continue to face barriers to seizing those opportunities and turning them into real progress. In particular, progress is often hampered because scientists and physicians are trying to do 21st Century research and development on personalized medicine in a healthcare system that in many respects is 40 years out of date.
In addition, despite the nice progress that Dr. Elizabeth Mansfield just reported, federal regulation of diagnostic and laboratory testing can be made clearer. This would create less uncertainty for personalized innovators and providers. Also, efficient pathways for premarket review of drug/diagnostic combinations need to better defined by FDA. In another area, will random control trials remain as the key evidentiary basis for drug approvals? Or will observational data be allowed to complement these more traditional studies?
Finally, we need a policy environment aligned with personalized medicine.
For example, as the field of comparative effectiveness research moves forward, it is important for it to align with and support the emergence of personalized medicine. Done well, CER can close evidence gaps involving personalized medicine, and can help illuminate the role of genetics and other factors that cause patients to respond differently to treatments. Done badly, CER will rely on broad population averages that do not incorporate genetic information and obscure patient differences.
To quote Francis Collins, "We need to be mindful of the goal of comparative effectiveness research and not lose all that we have gained in understanding how individuals differ and how that could be factored into better diagnostics and preventive strategies."
Also, as policy makers define new performance measures and provider incentives to improve health care quality and efficiency, it is important for them to facilitate adoption of novel, promising advances emerging from personalized medicine. If they focus on incentives that seek to hold down costs based on patient averages, they will slow the progress of patient-centered care and the science of personalized medicine.
Tomorrow, you’ll be hearing a great panel on the impact of big data on personalized medicine. One of our concerns at PhRMA is that outdated regulations on how our members communicate with health care professionals could limit the ability to use big data to move personalized medicine forward. As the healthcare system migrates to electronic record keeping, data collected by healthcare providers, insurers and others provide a rich source of information that can be mined and analyzed. The results can help researchers understand better why some medicines work with certain patients and are ineffective with others. That’s possible only if the different components of the healthcare delivery system communicate with each other. Unfortunately, current regulations rule that out.
5. Overcoming the Barriers
Like everyone here, PhRMA companies want patients to experience the benefits of personalized medicine. We want the barriers to progress taken down. But we know those barriers can only be overcome if every element of the healthcare system represented at this conference works together – we need the knowledge and strength of biopharmaceutical companies, academic, academic, hospital and government laboratories, practitioners, advocates, patients and their families. We need enlightened government leaders and regulators.
Speaking now about PhRMA the organization, where I have been leading the scientific and regulatory affairs function for about four months now, we’re committed to promoting the kind of cooperation that moves research forward, and ensures policy makers the right thing. PhRMA has always been at the forefront of advocating for a better policy environment that supports and fosters innovation but we are also actively supporting scientific progress and partnerships. We are convening more conversations about how we can better cooperate, share information, harness big data, and move science forward.
Just this year we established our new Scientific Leadership Advisory Council. The Council is comprised of heads of R&D, chief medical officers and chief scientific officers from our member companies. One of its most important goals is to deepen scientific engagement with our partners in the medical innovation ecosystem to spur advances such as personalized medicine.
Of course, it is also essential to the continued advance of personalized medicines for all of here today to work more closely together to cooperate and share wherever and whenever possible. As a recent example, this week we announced a collaboration with the Genetic Alliance to implement a web-based crowdsourcing effort to enhance patient input that is complementary to other information available to regulators on matters related to patient perception of severity of disease and unmet medical needs.
I began by saying that we are all here because we are excited about the power and potential of personalized medicine, and we know how urgent it is to make that potential real. The patients we serve and who wait and hope for better treatments and even cures deserve our best efforts. But hurdles still remain. As a result, the future of medicine could be kept waiting. So let’s work together all across the health care ecosystem to move the science forward and create an environment that embraces change and opens the door to personalized medicine.
To close, I say that by working together we can not only imagine the future but shape it. Thank you for your attention.