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

What are the next steps after being accepted into a clinical trial?

Acceptance into a clinical trial begins with the consent process.  Risks and potential benefits, as well as other options are explained by your physician or research nurse and also presented to you in writing in the Consent Form – typically a lengthy document.  Once you have been fully informed and have consented to the treatment, the next step is to complete the pre-treatment evaluation.  This includes a visit with your doctor or nurse, blood tests, scans to determine the extent and location of your cancer, and potentially other tests mandated by the study.  At this stage, it is still possible to be “uninvited” from the study.  This happens if a test result shows that you don’t meet all the criteria to safely participate.  Once the pre-treatment evaluation is complete, the study treatment begins. 

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 is being in a clinical trial different from getting regular treatment?

In many ways, being in a clinical trial is similar to receiving standard treatment.  There are regular visits, blood tests, scans, and a cancer drug – or several – are given on a set schedule.  Side effects are monitored and adjustments are made to minimize them.  There are some differences.  As much as cancer treatment programs tend to be fairly rigid and structured, clinical trials are even more so.  Everything that happens follows a precise recipe.  There may be additional tests or surveys to fill out that would not be a part of standard care.  Some of these may include much more extensive monitoring – for example serial blood draws to measure drug levels in detail during a 24 or 48 hour period.  It is also likely that less is known about the drug or drug regimen that is being tested than standard care.  Outcomes in cancer treatment are never certain, but participating in a clinical trial involves a greater degree of the unknown.  

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 many patients typically participate in each phase of the clinical trial?

Clinical trials are often described as phase I, II, III, and sometimes phase IV.  The number of participants varies greatly depending on the phase of investigation, but also depending on the cancer type, situation, and expected outcome.  Phase I trials are principally charged with determining the safe and optimal dose of a new drug or drug combination.  Such trials may involve 20 to 40 patients.  Phase II trials are where the potential of a drug to be effective is first evaluated.  Typical phase II studies range in size from 50 to 250 participants.  The higher end of that range is more common in randomized phase II studies where 2 or more doses or drug combinations are being examined side by side.  Phase III trials are the ultimate test of a new treatment.  Participants are randomly assigned to the new treatment or the current standard.  These trials involve hundreds and sometimes thousands of participants.  The number of participants needed is determined by the question being asked, the expected magnitude of the difference between the two treatments, and the percentage of patients who are expected to experience the outcome being measured.  For example, trials that seek to reduce the risk of cancer recurrence are often very large in part because many of the participants (thankfully) don’t suffer a recurrence at all.  A large number of participants are therefore needed to make sure that enough of them have a recurrence so that a difference can be detected.

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

Hormonal Therapy for Prostate Cancer - Education Video

In an occasional departure from our focus on clinical trials, we thought we would share our latest patient education videos for prostate cancer patients.  Here Dr. Beer discusses hormonal therapy for prostate cancer - very much the way it would be discussed in a clinic visit. 


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

In the US, who decides what cancer research is funded and how much funding each project receives?

There is no single authority that allocates cancer research funding.  Each funder has a process.  The largest source of funding for research is the National Institutes of Health which uses a peer-review process.  Panels of researchers review and rank research proposals.  This is probably the best process we have.  It is independent and invites the best thinkers to drive it.  Sometimes this process does, however, end up emphasizing funding for ideas that fit well with today’s thinking.  Unconventional ideas may not always be appreciated by peer scientists.  The pharmaceutical industry is the other large source of funding.  It allocates funding largely based on it’s view of market opportunities for new drugs, although industry also provides funding for independent research.  Many private foundations, and government agencies other than the National Cancer Institute are also important.  They generally follow the peer-review process or some variation on it.

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

Chemotherapy for Prostate Cancer - Education Video

In an occasional departure from our focus on clinical trials, we thought we would share our latest patient education videos for prostate cancer patients.  Here Dr. Beer discusses chemotherapy for prostate cancer - very much the way it would be discussed in a clinic visit. 


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

Immunotherapy for prostate cancer - patient education video

In a bit of a temporary departure from our focus on clinical trials, we thought we would share our latest patient education videos for prostate cancer patients.  Here Dr. Beer discusses immunotherapy for prostate cancer - very much the way it would be discussed in a clinic visit.  In the coming weeks, we will share our videos on chemotherapy and hormonal therapy for prostate cancer patients.

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