Congress must encourage the 21st Century Cures Initiative
By Peter J. Pitts
Over 200,000 American heart attack survivors will have another heart attack this year. Fortunately, the FDA recently approved Zontivity -- a medicine that prevents blood clots in people who have already suffered a heart attack. In clinical trials, the new drug significantly reduced the rate at which survivors experience another heart attack.
But this breakthrough came too late for the nearly 5 million Americans who died of heart disease during the eight years between when Zontivity first proved effective in clinical trials and when the FDA granted approval.
Times have changed. The "gold standard" of large-scale, long-term, and expensive clinical trials requires reinvention. That's why Congress should encourage the 21st Century Cures Initiative. Though still a work in progress, the bipartisan congressional initiative has the potential to streamline the drug approval process -- delivering breakthrough treatments to patients sooner and eliminating wasteful spending.
More efficient regulations could free up billions for new research and development. That's why the 21st Century Cures Initiative is proposing measures that accelerate the approval process by reducing "regulatory duplication and unnecessary delays."
The initiative's proposals could help to revolutionize the current drug approval process. Today, pharmaceutical companies test thousands of compounds in the lab until they identify one that shows promising medical applications. Next, they conduct "phase I" trials on healthy adult males to ensure a drug is safe.
Phase II trials provide an initial assessment of a drug's effectiveness in sick patients. Phase III, which involves thousands of volunteer patients, helps confirm the results of phase II trials.
The draft legislation will allow FDA regulators to approve a groundbreaking drug if it proves effective after a phase II trial. That will deliver treatments to patients much sooner and eliminate the need for some phase III trials, which take years and account for 90 percent of the total development costs for drugs that eventually gain FDA approval.
Had the law been in effect in 2007 when Zontivity passed its phase II trials, millions of patients could potentially have accessed the new treatment. Instead, barely 10,000 people received the drug during years of phase III trials.
The 21st Century Cures Initiative will also spur new drug development by helping biotech companies "fail faster." Of all the potential medicines that enter phase I trials, only 8 percent ever receive FDA approval. The proposed legislation would expedite "adaptive clinical trials" which identify the failures -- the other 92 percent -- earlier. That prevents researchers from wasting time and money on drugs that ultimately don't pan out.
Identifying and scrapping those failures earlier in the research and development process will allow drug companies to redirect billions towards more promising treatments. If adaptive clinical trials can catch even 5 percent of eventual failures in phase I instead of phase III, pharmaceutical manufacturers will save at least $15 million per drug.
The proposed changes mark the beginning of a transition towards a true 21st century innovation system that eliminates traditional phase II and III trials altogether. In such a system, doctors, insurers, and drug makers could use the power of big data to chart a drug's effectiveness and patient outcomes as soon as it proves safe in phase I trials. Using crowd-sourced, real world data will deliver lifesaving treatments to patients years quicker than the current clinical trial process.
Congress should ensure that Americans have the best and latest drugs possible as quickly as possible. Passing the 21st Century Cures Initiative is the best way to do so.
Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.