Faster Drug Approval: Winners and Losers

By the time a drug is approved by the Federal Drug Administration, it’s been through extensive human clinical trials. There are so many obstacles to greenlighting any new medication or treatment, in fact, that it seems like it can take forever — with countless lives lost in the meantime.

“We should be moving at the speed of science, which is advancing at a breakneck pace, and no single agency —  or company —  can possibly be fast enough to do this alone,” said one researcher.

Recently, due to calls for novel treatments that move faster from the (lab) bench to the bedside, a legislative effort is attempting to speed up that process. The 21st Century Cures Act would allow companies to submit evidence of safety based on patient stories instead of actual clinical trials.

The House already passed its own version; the Senate is expected to review it in the coming weeks, STAT reported.

While the measure could speed up the process, some people fear it would also remove a lot of quality control. And when it comes to health, it’s good to know that the medications you take have been tested extensively and are backed by reliable data.

Another concern is any new fast-track process would enable pharmaceutical companies, for the most part, to dictate the efficacy of a drug instead of the FDA as a third party.

Patience for Patients
Paul Howard, a senior fellow at the Manhattan Institute, said the new act uses adaptive clinical trial designs to identify biomarkers that can match drug candidates with patients who are most likely to respond.

Time-consuming pre-market trials can now be done with greater confidence, he said.

“We should be moving at the speed of science, which is advancing at a breakneck pace, and no single agency — or company — can possibly be fast enough to do this alone,” he said.

Howard believes passing the act would make patients safer and allow the FDA to continue to ensure safety.

“Clinical trials won’t disappear but will become much more pragmatic, and be focused on real world evidence and outcomes.”

“Clinical trials won’t disappear, but will become much more pragmatic, and be focused on real world evidence and outcomes,” he explained.

Dr. David Gortler, a former FDA senior medical officer and current drug safety and FDA policy expert at FormerFDA.com, told LifeZette he doesn’t agree with speeding up the process because lawmakers typically don’t have an extensive pharmacology background. Circumventing the time-tested FDA process will not help Americans.

“They [lawmakers trying to speed up the process] don’t trust these people who have dedicated their lives to pharmacology and medicine,” Gortler said.

People think they know better, but they are really just putting their lives at risk, he added. “They don’t trust doctors or pharmacists. They trust GNC.”

In a Washington Post editorial, Susan F. Wood, an associate professor at George Washington University’s Milken Institute School of Public Health and a former FDA assistant commissioner, explained that the 21st Century Cures Act is based on an assumption that there will be more cures if drugs and devices are studied more quickly on fewer patients.

She, along with Diana Zuckerman, president of the National Center for Health Research, believes the studies would be too small to allow safety and effectiveness findings to be broken down into subgroups such as male and female.

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“This embrace of smaller, more preliminary studies could drastically lower scientific standards. When fewer people are studied, it is more likely that a drug will seem safe and effective even if it has dangerous side effects for many patients — who may not have been included in those small studies,” they wrote.

FDA Under Fire
Recently, the Government Accountability Office issued two reports critical of the FDA’s drug approval process. One of them said the agency was lacking on goals to properly evaluate the approvals process.

The FDA sped up 2012 pre-market approval application times — down to seven months from 14 months and three years earlier. Reviews on new drugs and biologics also saw improvements from 13 months in 2008 to 10 months in 2010, 2011 and 2012.

Still, while reports vary, it can take an average of eight to 12 years to get a drug from development to the market. The preclinical stage — before a drug goes through the FDA approvals process, which includes three phases — can be three to four years alone and cost upwards of $500 million to develop a new drug.

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There are a few existing FDA programs to speed up the process. These include fast track, which is for drugs to treat serious conditions and fill an unmet medical need; breakthrough therapy, which upgrades a current therapy with a better one; accelerated approval, which allows for drugs that treat serious conditions that filled an unmet medical to be approved based on a measure that is thought to predict clinical benefit; and priority review, which gives approval on an application within six months.

Purpose in the Process
Gortler believes the drug-approval process timeline should not be changed.

“The process can’t be sped up very much,” he said. The FDA has made some improvements to the process for drugs that treat terminal illnesses or offer a groundbreaking solution. Otherwise, the process is in place because it serves a purpose: to properly evaluate drugs and devices for safe and effective human use.

Of all the drugs that make it through the first phase, 98 percent will fail. Anywhere from 80 to 85 percent of drugs don’t make it past phase two, and only half of drugs make it through phase three. These fail for safety reasons, because a drug can be proven effective but it also must be safe.

The FDA has to take into account different populations of people including various ethnicities, and test drugs on those with genetic differences. That all takes time — hence the long process, he said.

“They really put their best foot forward,” Gortler said of the FDA. “The drug really has to thoroughly be researched.”

“The FDA doesn’t want something to be released … if it’s going to be hurting people,” he added.