Breakthrough! Why You Should Care About FDA HAE Designations

Recently, the FDA gave “breakthrough therapy” status to a potential new drug that may help prevent HAE attacks. But how does that affect you, the patient? What does “breakthrough therapy” really mean? And what are some of the other designations the FDA uses when reviewing possible new treatments?

When a drug shows the possibility of addressing unmet medical needs for a serious or potentially fatal condition, the FDA may grant one of four Expedited Program designations:

  • fast track
  • breakthrough therapy
  • accelerated approval
  • priority review

A drug’s designation is no guarantee of approval–the drug still must be proven to be safe and effective. So why are the designations important?

It’s all about getting treatment into the hands of patients as efficiently as possible.

Fast track – Intended to help in the development and review of drugs, the “fast track” designation allows for frequent interactions between the FDA review team and the drug developer or sponsor. Fast-tracked drugs can also be eligible for “priority review.”

Breakthrough therapy – If a drug has been granted the “breakthrough therapy” designation, the FDA helps the drug sponsor design and conduct their development program as efficiently as possible. This results in a considerably shorter development program than for other drugs intended to treat the same disease. For example, clinical trials may require less time to complete or the number of patients in a control group (using a potentially less effective therapy) could be reduced. The FDA review team and drug sponsor meet throughout the development process to help speed things along. Breakthrough therapies are also eligible for “rolling reviews,” when portions of the marketing application can be reviewed as they’re submitted.

Accelerated approval – Exactly as it sounds: Accelerated approval green-lights a treatment for market before it has undergone extensive clinical trials. In order to be granted accelerated approval, a drug for a serious or life-threatening disease must show a “reasonably likely clinical benefit” based on available data. This approval process has been used for drugs that treat various cancers and HIV. Because drugs that receive accelerated approval have not been studied in extensive clinical trials, there is generally less information about rare or delayed side effects. There is also a greater risk that the drugs ultimately do not provide their anticipated benefits. Accelerated approval can be revoked if post-marketing trials do no verify the anticipated effects.

Priority review – A new drug application can be granted “priority review” status if the drug’s approval would lead to “a significant improvement in safety or effectiveness.” More attention and resources are given to applications with “priority review” status, so that decisions can be reached within 6 months (rather than 10 months through standard review).

For more information on the FDA’s Expedited Programs for Serious Conditions, click here.

James Ernest Cassady

James Ernest Cassady

Though "Ernest" is a family name that's been passed down for generations, James truly earned his middle moniker when, at the age of five, he told his mother that "laughing is stupid unless EVERYBODY is happy." Since then, the serious little bastard has been on a mission to highlight the world's shortcomings (and hopefully correct them). In addition to his volunteer work at hospitals and animal shelters, James also enjoys documentaries and the work of William Faulkner. He is originally from Oklahoma.

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