An Rx for making wonder drugs
Boston is to life-sciences innovation what Florence was to the Renaissance: a fertile hub for the blending of new ideas and technologies, revolutionizing our understanding of human biology. Boston’s ecosystem of cutting-edge hospitals, startups, venture capital and large companies is unraveling the molecular roots of complex, chronic diseases at astonishing speed. The goal is to drive ever-more-personalized treatments and prevention efforts, saving lives and reducing costs.
Novel technologies, however, are increasingly running into roadblocks in a regulatory and health care reimbursement system built for the mid-20th century, not the 21st. The enormous cost and time required to bring new medicines to patients strains America’s drug discovery ecosystem to the breaking point.
Today, bringing a single new medicine to FDA approval costs $1.3 billion and takes 10 to 15 years, according to the Tufts Center for the Study of Drug Development. A 2012 Nature Reviews Drug Discovery article estimated the number of drugs commercialized per $1 billion in investment falls 50 percent every nine years. Cautious regulators have also increased hurdles for companies bringing new treatments to market — requiring larger, longer and more expensive trials for drugs treating serious illnesses.
Health care reform also creates a more uncertain reimbursement environment for innovative drugs and diagnostics, leading investors and companies to worry about recouping large sunk costs even if they receive regulatory approval.
Experts worry that patient access to new technologies could be jeopardized, even as the potential health benefits have never been greater. As “ground zero” for health care reform, the state is also the right place to explore better ways to reward innovator companies for delivering greater value to patients.
Bay State companies, researchers, and policymakers thus have a critical role to play in developing a sustainable national model for bio-innovation, delivering better jobs and faster economic growth for the U.S., creating greater value for our health care system, and helping patients live longer, more productive lives.
We don’t have an answer for every challenge facing innovators, but we know, broadly, what must be done.
• Create a better model for drug development. U.S. Food and Drug Administration regulations should reflect state-of-the-art science, while policymakers need to encourage closer collaboration between regulators and other stakeholders, including National Institutes of Health and patient-led disease organizations. Not every question about a drug’s safety or efficacy can, or should, be answered through one-size-fits all clinical trials.
• Encourage broad data sharing, while protecting privacy. Federal privacy regulations, such as the Health Insurance Portability and Accountability Act, make it difficult for providers to share the large data-sets researchers need to discover underlying causes of complex chronic diseases. Data sharing should be a two-way street: patients dictate how, when and with whom their data is shared; in return, they become true partners in advancing medical research.
• Ensure innovation remains rewarded. Reimbursement systems must measure the full cost of treating an illness across the entire continuum of the disease, including side effects, hospital and physician use and the economic impact on lost productivity (including caregivers).
Boston’s life sciences leaders are justifiably proud of their achievements. Yet resting on their laurels isn’t an option. Sustaining and expanding the state’s leadership in this vital sector will only be possible if the country reforms how it discovers, develops, and delivers better treatments to patients — creating 21st century cures with 21st century tools.
Dr. Andrew von Eschenbach, chairman of the Manhattan Institute’s Project FDA, is a former commissioner of the U.S. Food and Drug Administration. Dr. Phillip A. Sharp is a Nobel Prize winner and Institute Professor at MIT. Dr. Kenneth I. Kaitin is director and professor at the Tufts Center for the Study of Drug Development. The authors will be participating in a panel on this topic at Tufts Medical School Monday.