Speeding up drug approvals could put patient health at risk
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During his first address to Congress in February, President Trump called for more rapid approval of drugs by the Food and Drug Administration (FDA), claiming that the agency has a “slow and burdensome approval process” that is keeping medicines from those in need.

He also called for slashing regulations at the FDA and his proposed fiscal year 2018 cuts the agency’s budget by 31 percent but aims to make up part of the loss by raising fees paid for drug and device makers to have their products reviewed. Just how would lowering funding help speed approvals?

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Drugmakers and their allies have been asking for speedier and less rigorous drug approvals through legislation such as the 21st Century Cures Act.

 

However, the drug approval system run by the FDA is already without a doubt the best in the world. A new drug is more likely to get approved in the US before it does in any other country. Last year, of the 22 new drugs that were approved, 19 were approved in the U.S. first. And all but one of them came in the first cycle of approval, meaning the FDA didn’t delay approval for additional information from a drugmaker.

The FDA also approves more drugs and does it faster than its European counterpart, the European Medicines Agency (EMA). Between 2011 and 2015, the FDA approved new 170 new drugs compared to just 144 by the EMA, according to an April analysis published in the New England Journal of Medicine. And the median review time was 303 days at the FDA compared to 383 days at EMA. The review also showed that over the same time period, the FDA approved 43.5 percent of applications for drugs to treat orphan or rare diseases, compared to just 25 percent at EMA.

It’s possible that the FDA is already approving drugs too rapidly. The FDA has 4 different expedited pathways in place: priority review (a 6-month FDA review rather than the 10-month standard), breakthrough therapy (for drugs that are an improvement over a current therapy), accelerated approval (approval based on a secondary outcome known as a surrogate endpoint that is thought to predict benefit) and fast track (for drugs where there is a serious unmet medical need). The FDA reported that nearly 73 percent of the 22 novel drugs it approved last year came through one or more of these rapid approval programs.

While this sounds great, the reality is there are risks to patients. Many of these drugs often are approved without strong proof of efficacy and have safety issues are always a concern. Shorter trials mean less time to study interactions with other medicines and until a drug is taken by thousands of people for months or years a full safety profile is impossible to create.

However, if studies are shortened and fewer people are in the studies that means that side effects won’t become apparent until well after they hit the market. In fact, a May Journal of the American Medical Association (JAMA) study found about one-third of the new drugs the FDA approved from 2001 and 2010 had side effects that caused so much harm that the FDA took action in form of a safety warning to healthcare providers, boxed warning on a drug’s label or even withdrawal from the market. And that doesn’t touch on the issue on not knowing minor side effects like nausea, dizziness or headaches.

One of the most common methods used to prove efficacy faster is the use of “surrogate endpoints.” A good example is the cholesterol drug Vytorin. It was shown to decrease LDL (bad) cholesterol and C-reactive protein, both considered “markers” of heart disease. The true endpoint is survival, and out in the real world it was found that patients using Vytorin did not live longer, even though their LDL (also known as the bad cholesterol) was lower.

These patients were harmed in three ways: 1) not receiving the benefit promised; 2) being exposed to a drug’s side effects; and 3) by not receiving effective care for their condition.

All of the expedited pathways to FDA approval should be limited severely to true breakthrough drugs that are needed urgently. Instead, too many drugs with only incremental benefits are being rushed through vetting creating an unnecessary risk for too small a benefit.

The public would be well-served by the FDA getting tougher on requiring post-marketing studies for new drugs. A study published in BMJ in May confirmed that post-market studies are relatively infrequent for new drugs that are approved based on limited evidence. Researchers looked at all the new drugs the FDA approved between 2005 and 2012 based on either a single pivotal trial or trials that used surrogate endpoints.

For 35 percent of the indications examined, there were no post-market studies conducted. And at an average of 5.5 years after approval, fewer than 10 percent of those indications had been studied in at least one controlled, double-blind, randomized study that showed clinical efficacy significantly better than placebo.

Those that want to streamline the drug approval process believe that the more medications that come to the market the more people benefit. But in order to do this, clinical trials will likely be curtailed, they will be conducted for less time and efficacy measures will be weakened.

We will have incomplete knowledge of a drug’s side effects and adverse events. Undoubtedly, drugs that will do more harm than good will be approved as a result. And that could only benefit pharmaceutical companies at the expense of patients’ health.

Suzanne Robotti founded MedShadow Foundation in 2012, which is a non-profit organization that explores side effects and long-term effects of medications that many people take.


The views expressed by contributors are their own and not the views of The Hill.