What process does the FDA follow when determining whether to approve a drug?

FDA approval doesn’t actually take as long as most people think.  In a recent blog post, http://www.cancer-clinical-trials.com/2012/07/waiting-for-new-drug-to-be-approved.html we discussed the fact that FDA approval, at an average of 322 days, is actually a little faster than approval in Europe and Canada.  That’s not to say it’s quick, but it does not take years.  What takes a long time are all the studies necessary to collect the information the FDA requires to consider an application.  The FDA uses the “safe and effective” standard.  Effective means that cancer patients benefit in a meaningful way.  This often involves living longer, being cured of cancer, or experiencing quality of life improvements.  The FDA reviews all of the clinical trial data in detail to determine if these standards have been met.  This review takes several months.  While we all want cancer drugs to come to market as quickly as possible, it is also worth remembering that the FDA is held accountable when a drug is approved that later proves unsafe.  It’s a balancing act between moving quickly and dotting the i’s and crossing the t’s.

For more questions and answers about clinical trials, visit the Talk about Health website.
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

How does the FDA know about new side effects for a drug that has already been approved?

The FDA has surveillance programs in place to monitor the safety of drugs after approval.  These programs primarily rely on physicians reporting to the FDA the side effects they see.  While not all physicians routinely make such reports, enough do that unexpected side effects usually come to light.  For some drugs that are approved with some questions about safety, special requirements can be put into place.  These might include so called phase IV studies to gather more safety data or special prescribing programs for high-risk drugs.

For more questions and answers about clinical trials, visit the Talk about Health website.
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

What metrics are typically used to determine if the drug is successful?

The ultimate success of a new drug or drug combination is judged by the “safe and effective” standard.  Both of course are examined in context.  Safe often means safe enough – or no less safe than what we have today.  Some cancer treatment have severe side effects – but because their benefits are judged acceptable – they are approved.  Effective means that cancer patients benefit in a meaningful way.  This often involves living longer.  When survival is not impacted, meaningful quality of life improvements are required.  In some situations, cure rates are the measure of success.  Survival benefit, which is the most common measure of success, can take a year to several years to determine.

For more questions and answers about clinical trials, visit the Talk about Health website.
To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.

(c) 2012 Tom Beer and Larry Axmaker

Cancer Clinical Trials – minorities and the elderly missing in action

An article in the November 2011 issue of CancerDiscovery, the journal of the American Association for Cancer Research, defines the problems and outlines strategies for recruiting minority groups and seniors for Clinical Trials.

Although about one third of the US population belongs to a minority group, this group accounts for less than 1 percent of adults enrolled in clinical trials. And, while nearly half of those diagnosed with cancer and 65 or older, seniors make up only 25% of clinical trial participants.

Minority groups and the elderly are at higher risk of being diagnosed with cancer than the population as a whole. So when there is low participation in trials from any group of cancer patients, the trial results may not be as applicable to those groups. And the low participation can also delay final approval of beneficial drugs. In order for us to know how to best treat all Americans with cancer, we need all Americans to be represented in clinical trials.

Several groups are now focusing on providing basic clinical trial education using DVDs to these groups—in several languages when appropriate.

Another direction is to provide additional training to nurses and doctors to help them better understand the issues in recruiting and the possibilities of participation in clinical trials for their patients.

And in some locations patient ‘navigators’ are being trained to provide one on one information, answer questions, and help potential participants fill out application materials.

Waiting for the new drug to be approved…

Not all clinical trials end with a successful drug or treatment being approved by the FDA. But when the trial is successful we want the new drug to be available today—if not sooner. It seems like it takes forever for the FDA to review and finally approve a drug, especially if it’s one that might benefit us.

In a study recently published by the New England Journal of Medicine, two researchers from the Yale School of Medicine studied the average time from clinical trial to approval by the FDA in the U.S., the European Medical Agency (EMA), and Health Canada between 2001 and 2010.

Although the average approval time still may seem long, the FDA approval time was more than two months faster than the EMA and more than three months faster than Health Canada.

Average drug approval times:

FDA                            322 days

EMA                           366 days

Health Canada         393 days

Approval agencies around the world are always under pressure to approve potentially successful drugs as soon as possible and many approvals are done in less time than the averages shown here. It is worth noting also that a long approval time may mean that the study results are not as clear cut as it seems and the FDA needs to ask more questions. The other side of the coin, of course, is when a drug may be approved too quickly and then have to be recalled because of risks and problems not known at the time of approval.

To put a smile on your face see Larry's latest cartoon.
To learn more about clinical trials, take a look at our book.
(c) 2012 Tom Beer and Larry Axmaker