Gottlieb Promises to Accelerate Drug Approvals; Surrogate Endpoints Can Help

Gottlieb Promises to Accelerate Drug Approvals; Surrogate Endpoints Can Help
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President Trump's FDA appointee Scott Gottlieb wants to speed up the drug approval process to give patients quicker access to life-saving drugs.

Luckily for him, President Obama gave him the tools he'll need to do so. In the waning days of his presidency, President Obama signed into law the bipartisan 21st Century Cures Act. The legislation permits the FDA to approve more drugs based on "surrogate endpoints." This move will speed up the drug approval process and improve millions of Americans' chances of beating cancer.

Scott Gottlieb has long favored quicker drug approvals as a way to save lives. In 2012, he claimed the FDA's "hunger for extreme certainty about how drugs work" has done more harm than good. In other words, by demanding years of exhaustive clinical trials, the FDA prevents safe, effective drugs from entering the marketplace.

Surrogate endpoints are the answer Gottlieb is searching for. Surrogate endpoints sound complex, but the idea is relatively straightforward. Traditionally, FDA regulators don't approve a drug until a pharmaceutical company shows that the medicine achieves the "clinical endpoint" -- a metric such as increased three-year survival rates.

Hard endpoints, like overall survival, are considered the gold standard for measuring the efficacy of a cancer drug. But measuring overall survival endpoints takes years and requires large sample sizes of clinical trial patients. That's often infeasible for medicines that treat rare, deadly cancers that kill in months.

So instead, the FDA is sometimes willing to approve drugs based on surrogate endpoints -- how long the patient lives without the tumor spreading, for instance. Regulators believe that if a drug effectively delays the tumor, it also improves life expectancy. That probable cause-and-effect relationship is enough for the FDA to grant expedited approval to the drug and get it into dying patients' hands.

Substituting surrogate endpoints for clinical endpoints shaves three and a half years off the drug development and approval process, according to FDA data. That accelerated timeframe can mean the difference between life and death for patients. By approving treatments for serious diseases right away, the FDA gives patients a fighting chance to beat their diseases.

Take Stage IV breast cancer. The prognosis is grim: four out of five women won't live five years past their diagnoses.

But thanks to surrogate endpoints, one treatment, Afinitor, is making a difference. More than half of advanced breast cancer patients taking Afinitor in conjunction with another drug saw their tumors shrink or disappear, nearly twice as many as those taking the companion drug alone.

Overall, the treatment doubles the time patients live without the cancer worsening -- a surrogate endpoint called progression-free survival.

Some medicines approved based on surrogate endpoints improve patients' quality of life even for the rarest of diseases. Chronic lymphocytic leukemia is a rare type of cancer that afflicts 19,000 Americans each year.

Patients are seeing dramatic improvement with a drug called Imbruvica, which was approved based on surrogate endpoints. More than eight in ten patients saw their tumors shrink or stop growing, compared to 35 percent of patients treated with the current leading chemotherapy.

Because of the FDA's already considerable reliance on surrogate endpoints, the United States leads the world in healthcare innovation. From 2003 to 2010, the FDA took just six months, on average, to approve new cancer treatments.[9] The European Medicines Agency -- which waited until this past June to propose a significant use of surrogate endpoints-- took nearly twice as long.

Across the Atlantic, patients don't only wait longer for treatments; some medicines are unavailable altogether. The United Kingdom's National Health Service, which provides most Britons' healthcare, is reluctant to pay for drugs approved based on surrogate endpoints. In 2015, the NHS stopped covering 25 cancer drugs, including Afinitor. Most of those targeted rare or advanced cancers

Medicare, by contrast, covers 80 percent of FDA-approved drugs.

 Since it started approving drugs based on surrogate endpoints in 1992,  the FDA has shown it values innovation and is willing to cut through unnecessary red tape to quickly deliver breakthrough treatments to patients. The new legislation gives Gottlieb and his team authority to make even greater use of these surrogate endpoints. That's sure to give cancer patients more time with loved ones and a new lease on life.


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