Posts tagged phase 4

Are Clinical Trials Safe?

Dec 28, 2012 Leave a Comment ~ Written by Dan Sfera

One of the first things people will ask themselves when considering joining a clinical trial: “is this safe for me?” This is a very good question to ask yourself, but in all honesty, no one can claim that any clinical trial will be 100% risk free. If anyone does tell you otherwise, then they must be pretty desperate to recruit you, and are probably trying to meet their enrollment goals! Also, if you ever hear someone claim that there are no risks associated with joining a clinical trial, RUN away, and tell me all about it here. After all, there wouldn’t be a need for conducting clinical trials if the drugs they are testing were risk free. Therefore, no clinical trial is truly “safe”. So if you are looking for an answer to your question that is absolutely, without a doubt, 100% yes, then a clinical trial is just not right for you. Once you reach the decision to continue exploring the possibility of joining a study, you have to ask yourself what degree of risk am I willing to take?

Phase 1 studies have the most risk involved. As explained in a previous section, this is the first time a study is being conducted in humans, so the side effects of the drug are nowhere near being understood or discovered. Up until this point, the drug was tested in animals, but the effects on humans is virtually unknown outside of the theories established by scientists. However, these types of studies pay the most money to its participants, and many people are perfectly ok with the risks in order to get their compensation. The possibility of getting placebo also plays a role here. It is from these studies that the terms “human lab rat” and “professional guinea pig” originated.

Phase 2 and 3 studies have relatively similar degrees of risks. The toxicity of the drug as well as its major side effects have most likely been determined. Now the drug is being tested on the actual targeted population that the drug was designed for. For example, a Phase 1 trial may test a diabetes drug on a healthy person, but in a Phase 2 and 3 trial, a person with diabetes will test the study drug. The risks here are still side effects that may be unknown, but now we also throw in the possibility of your disease or disorder worsening rather than improving. With this added risk of a worsening condition due to getting a placebo or perhaps the fact that the drug doesn’t work, you must calculate whether you should consider joining the trial if you are actually doing well with your current treatment. These studies also pay significantly less than the phase 1 studies due to less relative risks, and the possibility of an actual purpose for the drug in treating your current condition. The risks are calculated differently if you do not respond well to your current, pre-study treatment and are willing to try the study since you may have less to “lose”. In this case, if the drug doesn’t work for you, you are now in the same position as you were before.

With all these different possibilities and combinations of risks versus your expectations, you can see that the subject of clinical trials truly varies on a case by case basis, and almost no blanketed statements can be made except for perhaps the fact that no matter what the outcome for your particular case, data was collected and science was advanced, if even a little.

Posted in Featured, Study Participants - Tagged adverse events, informed consent, phase 1, phase 2, phase 3, safety

Do People Participate In Clinical Trials Just For The Money?

Dec 18, 2012 Leave a Comment ~ Written by Dan Sfera

Why choose to participate in clinical trials…is it REALLY about the money?
A word about paid studies. Let’s face it, many people that I’ve run across, not only at my own clinic, but also at industry conventions and other such events, choose to do studies for the payments. This especially becomes evident in phase 1, healthy volunteer studies, where the study participants jokingly refer to themselves as “professional guinea pigs”.

I would just like to say that while there is nothing necessarily wrong with doing studies primarily for the payment, many people, especially those in phase 2-4 studies, don’t consider payment the primary reason for doing studies. For many people that I’ve come across, traditional therapies that are already available on the market do not work as well as previously anticipated, and these people seek newer treatments, not yet available to the public, via clinical trials. In cases such as these, payment becomes a mere afterthought, and the treatment of the disorder or disease become the primary motivator. Furthermore, many patients feel as though they actually receive better medical attention when they participate in studies. This may be true in many cases, as some outpatient study visits may take anywhere from 1-4 hours, and all bases are pretty much covered in terms of a clinical perspective (vital signs, labwork, ECG’s, MRI’s, CT Scans, quality of life surveys, etc).

Many of these procedures, while often important, will never get recommended in a non-study setting due to ever increasing pressure from insurance companies to keep costs down, however in a study, these procedures would never be overlooked. This may be a sign of the times, and evidence of how broken our medical system actually is.

Posted in Featured, Study Participants - Tagged compensation, ethics, phase 1, phase 2, phase 3, safety, study participants

More CRO’s Gaining A Firm Foothold In China. Charles River Labs Acquires Chinese Pre-Clinical Company

Nov 05, 2012 Leave a Comment ~ Written by Dan Sfera

A few days ago, according to an official press release from publicly traded Contract Research Organization, Charles River Laboratories, has acquired a 75% stake in Chinese pre-clinical development company Vital River. According to Vital River’s website, “Vital River Laboratories is a leading company in commercial production of laboratory animals in China, dedicated to offer integrated laboratory animals and related services in support of biopharmaceutical research.” This deal not only gives Charles River Labs a greater presence in China, but also will allow it to provide more of an end to end or phase to phase clinical trial service for its Pharmaceutical clients. A few weeks ago I wrote about US companies looking to China for more of their outsourcing needs and how this is paving the way for Chinese CRO’s to gain a good amount of market share. Even more recently I wrote about smaller niche CRO’s joining forces to compete with the larger CRO’s in order to offer end to end clinical trial development services. What this actually means is the ability for a CRO to conduct the ENTIRE clinical trial process, from drug discovery to pre-clinical development (which is what Charles River Labs gained in this purchase today) as well as phase 1 to 4. What this deal does for Charles River Labs is allows itself to better establish itself as a full-spectrum CRO in China, especially in the pre-clinical and “research modeling” areas. Keep your eyes peeled for many similar deals to occur ofver the next several months. For more info on pre-clinical trials, I did an interview with a guest guru on this although it is a bit hard to hear. I will try to redo this interview sometime for you.

Posted in Featured, Research Professionals - Tagged China, cro, outsourcing, phase 1, phase 2, phase 3, preclinical

Know Your Expectations Before Joining A Clinical Trial

Oct 22, 2012 Leave a Comment ~ Written by Dan Sfera

Way back in 2010 I wrote a blog post regarding expectations and clinical trials. I basically made the argument that one cannot truly make an informed decision about a clinical trial until they understand what they want to get out of it. In this video I discuss the reasons for this, but it basically boils down to the fact that there are so many different types of clinical trials and it may be very easy for someone to feel overwhelmed about clinical trial study participation. For example, phase 1 studies are for healthy volunteers and the benefits are mainly making a lot of money for the trial participant. Phase 2-4 studies are studies on the targeted patient population but even there you have options such as placebo-controlled trials, as well as randomization and double-blind studies. To learn more about these trials check out this post of mine. Whatever your reasons for joining a clinical trial may be, and there are indeed many, as long as you are sure of what you are seeking to find, it will then become easier to ask more specific questions when speaking to the various research clinics out there. As always, feel free to contact me with any other questions.

Posted in Featured, Study Participants - Tagged compensation, ethics, expectations, phase 1, phase 2, phase 3, placebo, questions, randomized, side effects, study participants

Phase 3 Clinical Trials Just May Be Your Best Option!

Oct 17, 2012 Leave a Comment ~ Written by Dan Sfera

I got asked a great question by a viewer who is considering to participate in a clinical trial. He asked me what phase of trial would be best for him. For those that want to learn more about the various phases of clinical trials, I recently wrote about it here.
My answer is addressed in the video, but basically if a clinical trial gets to Phase 3, it is often called a pivotal trial. These studies usually recruit a large number of study participants, usually between 500 and 2500 total participants. The dose and the majority of side effects are believed to have been determined by this point, so the main objective is to determine whether the drug works in comparison to a placebo (sugar pill). Safety and side effects are still closely observed as well. These studies usually occur in outpatient settings and involve many study visits (between 10-30 at times). If the drug proves to have efficacy (does it actually work?) over placebo, or a comparable drug that is already approved and on the market, the drug company will file an NDA (New Drug Application) for FDA review. The FDA may take as long as 1 year to make a decision on whether the drug will be approved, or whether even more trials are needed to determine safety or efficacy. At this point, labeling issues (what information appears on the bottle, and what the drug company can claim regarding the drug) are addressed.

Posted in Featured, Study Participants - Tagged efficacy, free treatment, good experience, phase 1, phase 2, phase 3, safety, side effects, study participant, treatments

Clinical Trials Explained From Phase 1 to Phase 4

Oct 14, 2012 Leave a Comment ~ Written by Dan Sfera

There are four phases of clinical trials, and due to the lack of adequate information on this out there, I decided to write this article to help you in your general understanding of clinical trials. I also did several videos on this, my favorite one here which wraps up clinical trials in a few minutes.

Phase 1 trials are first in human studies. The first step of an investigational product’s path to commercialization commences with phase 1 healthy volunteer studies. According to a recent article in Fierce CRO,# one of the biggest criterion that pharmaceutical companies look for when selecting a contract research organization, hereinafter “CRO,” to conduct their phase 1 clinical trials is the ability of a CRO to quickly enroll appropriate study participants into the study. Most phase 1 trials involve healthy human participants, but there are two indications, oncology and psychiatry, where Phase 1 clinical trials are actually conducted on the targeted population. These trials help researchers and ultimately the drug companies identify the dose range for the investigation products, more specifically, at what dosage do the drugs cause toxicity. These data are crucial to understand in order to get to the Phase 2 trials. The Phase 1 trials are generally conducted at roughly one dozen research clinics per study and typically enroll about 100 study participants worldwide. Once these studies are done, and the toxicity levels are found, phase 2 clinical trials are conducted.

Phase 2 clinical trials, with the exception of some psychiatry and oncology compounds, are the first time that the investigational products are being tested on the targeted population. In other words, if we were to develop an asthma drug, we would test the toxicity levels on healthy volunteers in the Phase 1 trials. Then, an appropriate dosage (non-toxic) would be used in the phase 2 trials to see if the drug is, first of all safe, and also to see whether it actually works, or is efficacious. This is the point at which these trials become extremely costly. The Phase 1 trials are typically limited to under 100 participants at only a dozen centers, and these studies typically only last two to four weeks. The Phase 2 trials are conducted on several hundred patients across the world at several dozen different research clinics, and these studies may last several months in length.

Phase 3 trials, also known as “pivotal trials,” are where the bulk of the clinical trial budgets are spent. These trials are usually the final trials needed to prove to the FDA that the drug is not only safe but also effective. These types of studies typically enroll several thousand study participants (3,000 to 5,000) worldwide and contract several hundred research clinics from around the globe. Furthermore, phase 3 clinical trials can last anywhere from one to five years depending on the data that the pharmaceutical company is trying to extract.

Phase 4 clinical trials are also referred to as “post marketing trials.” Often times, when an investigational product has been approved by the FDA following a phase 3 trial, the FDA requests additional safety data. What is being analyzed in these studies is the “fine print” that often goes onto the medication/intervention packaging. These medical claims must be approved by the FDA before they are allowed to be used as promotional information by pharmaceutical representatives. Phase 4 clinical trials, in the opinion of co-author Dan Sfera, may indeed offer the most benefit to study participants due to the fact that placebos are rarely, if ever, used. Furthermore, an exorbitant amount of data have already been gathered on the medication/intervention in the phase 4 trial; enough data to convince the FDA that the drug is safe and effective enough to be prescribed by physicians.