Personalized Medicine Education and Advocacy

Thought leadership in personalized medicine

Challenges and Opportunities in an Era of Personalized Medicine

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by Stephen J. Ubl, president & CEO, PhRMA

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Stephen J. Ubl

We are at a pivotal time in health care in America. Biomedical discovery and innovation have the potential to revolutionize the way we diagnose and treat patients. At the same time, rapidly changing regulatory and market dynamics are shifting the ways we develop and pay for new technologies. Personalized medicine stands right at the center of this revolution, with the science enabling greater precision that not only can improve the lives of patients, but can also create efficiencies within the health care system by delivering the right treatment to the right patient at the right time.

We are at a unique juncture in this drive toward personalized medicine. We applaud Congressional focus on biomedical innovation to accelerate new treatments and cures for patients, as seen in the 21st Century Cures Act and in the Senate Health, Education, Labor and Pensions Committee’s ongoing effort to spur medical innovation and drive new research paradigms. We are energized by both the Precision Medicine Initiative and the National Cancer Moonshot, where we see stakeholders from across the health care community coming together to hasten progress for patients.

The biopharmaceutical industry has long been committed to advancing personalized medicine. Last year, more than a quarter of novel new drugs approved by the U.S. Food and Drug Administration were personalized medicines, offering patients important new treatment options across a number of diseases. In cystic fibrosis, a new era of personalized treatment is enabling patients to target the underlying genetic cause of their disease, where previously they were only able to treat symptoms. Oncology is on the leading edge of personalized medicine, with cancer patients seeing a four-fold increase in their targeted treatment options in just 10 years. Five-year survival rates have tripled for patients with chronic myelogenous leukemia since the introduction of the first targeted therapy for this devastating blood cancer, transforming a deadly disease into one in which patients live close to normal life spans.

The great news for patients is that biopharmaceutical companies are just getting started. Today, a full 42 percent of medicines in development are personalized medicines and 73 percent of oncology medicines in development are potential personalized medicines.

Private and public sector collaborations and the ability to maintain an ecosystem that supports and encourages innovation are key to driving continued progress in personalized medicine. As the health care market continues to evolve, we need to make sure we have policies that deliver innovative treatments to patients.

I look forward to discussing both the challenges and opportunities we are facing in this new era of personalized medicine on May 25 at the Personalized Medicine Coalition’s 12th Annual State of Personalized Medicine Luncheon. I hope you’ll join me.

Register for the luncheon here.

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