Breakthrough Therapies Do Not Lead to Unsafe Drugs.
[Posted on: Thursday, July 19, 2018]

In a review of 46 drugs approved under the breakthrough therapy (BTD) program, it was found, not surprisingly, that most of the approvals were based on smaller, uncontrolled clinical trials, implying that the BTD programs leads to approval of unsafe drugs. However, this analysis ignores the core premise of this program, namely faster approval for treatments for life-threatening diseases for which either there are no treatments, or the available treatments are grossly insufficient compared to the new drug being developed. In such cases any delays in making the new drug available to the patients, who may otherwise die, must be avoided. The BTD program was specifically created to give FDA a way to approve drugs with significant promise based on preliminary clinical evidence, without requiring the full burden of proof. It allows FDA to project the data from smaller studies to the general population so that drugs can be approved faster. Most of these drugs are approved with significant post-market commitments. It is important to note that there are no reports of any of the drugs and biologics approved under the BTD program to have increased the risk to patients, while the benefits to the patients has been well documented. This program requires additional resources from the FDA due to the pressure to make quick decisions. This is not the first time that the BTD program has been questioned, but so far the program has yielded great promise and holds strong support both at the FDA and public. While the published analysis documents the facts, it would have been more useful if it had also reviewed the post-approval experience with these 46 products. The conclusions of the review would likely have been very different.