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Marc M. Boutin, JD
EVP, Chief Operating Officer, National Health Council
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As we all know, there are hundreds of thousands of people in this country with unmet medical needs. They are desperately seeking new treatments, let alone cures, for their conditions. I represent an organization that addresses systemic issues that impact the lives of all people living with chronic diseases and disabilities, so I struggle with identifying any one new treatment for any specific condition that we want to emerge from the biopharmaceutical pipeline.
What I am hopeful for – and the patient community is already seeing emerging signs of it becoming a reality – is a concerted effort to instill greater engagement by people with chronic conditions in the drug development process. Companies are beginning to involve patients at the front end of the continuum. Manufacturers are eliciting the patient perspective in new ways and seeking the means to incorporate those views broadly. By defining what product characteristic patients’ value and what outcomes are most meaningful to people with chronic condition, we will see higher-value products.
On the regulatory side we are already seeing a concerted effort to better understand the patient perspective through the FDA’s Patient-Focused Drug Development program. This series of meetings with people in specific disease categories is designed to embed the patient experience into the FDA's decision-making process, and private industry is already taking an interest in the tools created by the National Health Council to help systematically catalogue patient comments, concerns, and desires as part of the development program.
No one player in the biopharmaceutical pipeline has the clout to bring about the cultural shift that will result in honest, open patient engagement. But we are seeing an evolvement in thinking and action that gives me hope we will achieve a new awareness and concrete action in the coming years.
Paul Howard, Ph.D
Senior Fellow, Manhattan Institute
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It looked like 7-year-old Emily Whitehead had run out of hope. Ravaged by drug resistant leukemia, her doctors had exhausted all of the available FDA approved treatment options. So her family enrolled her in a small clinical trial at the Children’s Hospital of Philadelphia testing a new approach to fighting cancer – an immunotherapy that would genetically modify her body’s own T cells to hunt down and kill her cancer cells. In April 2012, she received her first treatment.
The results were stunning. Her body responded with an all-out attack on her cancer that melted her tumors away. But it also threatened to kill her, since the inflammation produced by her infection led to a raging fever. Her doctors diagnosed the source of the problem as cytokine release syndrome and prescribed another drug, used to treat rheumatoid arthritis, to bring her immune system under control. In August 2012, she started second grade, and remains healthy today.
Emily’s story shows biopharmaceutical innovation at its best. The development of cancer immunotherapies has taken decades, and faced numerous failures along the way. But those risks have paid off, and we’re now seeing more successful immunotherapies reach patients that, many experts think, will redefine the battle against many different types of cancer.
For all of its fearsome reputation – still much deserved – oncology treatment is surging ahead scientifically, benefitting from decades of robust investment, relatively flexible regulatory tools, and advanced diagnostics (like whole genome sequencing) that are helping us to unravel the underlying mechanisms driving the disease. Even more powerful prevention and detection tools should help us catch the disease in its earliest, most vulnerable stages.
Other disease areas, however, are much farther behind oncology. In neurology, for instance, we still don’t have a good understanding of the underlying mechanisms driving severe depression, schizophrenia, and autism. Collectively, these ailments strike Americans when they are young, robbing them of full potential and requiring years of expensive and intensive treatment. At the other end of the spectrum, Alzheimer’s threatens a tsunami of human and financial costs for an already overburdened health care system.
We need to shift our imagery of the “pipeline.” It’s a useful metaphor for thinking of late stage products, but doesn’t do justice to the complex “architecture” driving biomedical innovation. If you turn on your faucet, you expect a flow of water, and don’t think about the sophisticated architecture – the dams, aquifers, purification and treatment plants, and massive array of pipes stringing hundreds of miles across your state and city and into your home. If one of those pieces of invisible architecture breaks down, the water flow can flow to a trickle.
Biopharmaceutical innovation – in the form of new medicines – is also the result of a complex architecture that is largely invisible to the patients and frontline physicians who utilize it. Investment in the NIH, training next generations of scientists in our schools and universities, and a financial environment that encourages and rewards investment into cutting-edge therapies, are all necessary for a pipeline to produce new cures.
That architecture is threatened today by many forces. A stagnant NIH budget, the potential of price controls for Medicare Part D, threats to the exclusivity granted to biotech medicines, and a decades-old regulatory system endanger a pipeline that holds more promise now than ever before.
So I would conclude with that thought. How many Emily Whiteheads are there in America today? And tomorrow you or someone you know and love can become the next Emily, desperate for hope.
While we’re applauding the pipeline, and everything it is capable of, let’s not forget the pipeline is part of a vast and complex architecture driving medical innovation forward. So whatever we want from the pipeline tomorrow, we have to lay the investments for today. The lives of millions of Emily’s depends on it.
Dr. Bill Chin
EVP, Scientific & Regulatory Affairs, PhRMA
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On the surface this seems like a fairly straight-forward question, but when you dig a bit deeper it’s a difficult one. Why? Because there are a lot of very exciting potential scientific advances in the pipeline. Only a handful will result in a new, innovative treatment approved for patient use, but nearly all will lead to new and better lines of research.
Innovative medicines to treat or even cure a disease or condition are critically important to patients. Yet, for me, it is the possibility of out-right preventing disease that is a game-changer. The power of prevention is that it stops disease from transforming people into patients altogether.
The foundation already exists. As reflected in our Research & Hope Awards program held last week, vaccines play a tremendous role in halting numerous infectious and viral diseases. The once dreaded killer smallpox, for example, was eradicated globally because of vaccines – a first in human history. The overwhelmingly majority of people vaccinated never become flu, measles, polio or rubella patients; they never need a hospital bed or nursing care to treat those conditions; they don’t lose or miss their jobs to fight a preventable disease. In short, they live lives free of fear of these once-deadly conditions.
Better still, continuing vaccine and medical research provides new paths to preventing other diseases. Cervical cancer is now largely preventable by vaccinating against the human papillomavirus – an advance that opens new research doors into better ways to prevent other viral-induced cancers and other conditions. Beyond vaccines, biopharmaceutical researchers are working on new approaches to prevent cardiovascular disease, building on the transformational successes of cholesterol-lowering statins and anti-hypertensive medicines in addition to important lifestyle changes.
However, there are unique hurdles in the development of preventative agents. The enormous number of patients needed to participate in clinical trials over an extended period of time can discourage a company from pursuing a potential preventative compound because it may leave a limited patent term during which to recoup the sizeable investment in research and development. Unfortunately, business decisions and the desire to pursue promising science sometimes collide.
Beyond the scientific and drug development challenges, our health care system also maybe ill-prepared for a shift to preventative medicine.
As the science continues to provide more prevention possibilities, we must find new ways to promote compliance to medicines. This is particularly challenging when a person (note – a person, not a patient) is asymptomatic. We must work with the provider community to ensure that physicians and others on the front line recognize the potential of emerging preventative medicines, along with behavioral and lifestyle changes. We also must assure broad access to medicines and better recognition among payers (public and private), employers and policymakers about the value of preventive medicines versus long-term costs (both economic and quality of life).
It’s a very different way of approaching medicines and health care. As a physician, I think that it is the next step in the exciting evolution of medicine. It is a scientific revolution that started by focusing on the sick and providing patients with treatments and cures, but its full fruition will be realized in the complete prevention of many once-deadly disease threats.