demystifying-clinical-trials

Clinical Trials Demystified

Copyright © 2010 TheClinicalTrialsGuru.com

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By Dan Sfera, Clinical Trials Guru

Table of Contents

1.) Introduction 3

2.) Are We Really Gurus? 4

3.) What Is A Clinical Trial? 5

4.) Are Clinical Trials Safe? 8

5.) Your Rights As A Clinical Trial Participant 9

6.) Your Privacy 12

7.) Informed Consent & Your First Study Visit 12

8.) IRB’s Protect Safety and Rights of All Trial Partcipants 14

9.) You are More Valuable Than You May Think 15

10.) Therapeutic Misconception 16

11.) Money, Money, Money 19

12.) Know Thy Expectations 20

13.) Conclusion 21



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1.) Introduction First of all, both Don and myself believe it’s important to tell you a little about us and what we hope this e-book and our website, The Clinical Trials Guru will accomplish, both in terms of helping the clinical trials industry, as well as society as a whole (I’m reaching just a little). My name is Dan Sfera and I have worked in the clinical trials industry as a Research Site Director for the past 6 years. Prior to that, I was involved in clinical trials as a Research Study Coordinator while I was an undergraduate at The University of Arizona. I currently am a majority owner of South Coast Clinical Trials, Inc., a dedicated psychiatric research clinic located in Southern California, and am responsible for Business Development and Marketing for our three clinics: Anaheim, San Bernardino and Norwalk, CA. My colleague and co-guru, Don Walters, has been involved in the clinical trials industry for over 10 years, primarily as a Recruitment Specialist. He currently serves as the Director of Community Outreach for South Coast Clinical Trials. As a recruitment guru, Don understands the issues that research clinics have to deal with in order to meet their enrollment numbers. If you ever have any questions about recruitment and clinical trials, send Don an email. The purpose of our website and this e-book are basically to help demystify the clinical trials industry by talking about it in plain English, and doing so EVERYDAY. Our intended audience are people who may be considering a clinical trial for themselves, or for a family member or a friend. Our intentions are NOT to enroll you into a study, or to convince you that clinical trials are your best options in seeking better medical treatments (although sometimes that is indeed true). An interesting thing that occurred as our website evolved, is the fact that research professionals and clinical trial industry types have also started interacting with us through Twitter, Facebook, LinkedIn, and our company Fan Page on Facebook, and many have even contributed their ideas to our audience by allowing us to interview them (in most cases even live and unscripted!), and welcoming the idea of being featured in our videos. For an industry that is notoriously so mysterious and secretive, we found that to be a welcome occurrence, and we’ve got



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plenty more “guest gurus” (more on the whole guru thing later on in the e-book), lined up for future interviews and videos. Discussing clinical trials in plain language everyday, providing excellent and authentic content, engaging with study participants (some who regularly appear in our videos), former study participants, industry leaders, and industry insiders, are just some of the things our audience expects from us everyday on our site. We understand that trust is a hard thing to earn in this industry, and we too are frustrated by the lack of educational and useful content that the clinical trials space apparently suffers from (both online and offline). The day we stop providing authentic and useful information on clinical trials is the day someone should pull the plug on our blog. Our goal is to empower people who are interested, to know more about clinical trials than they already do. We hope you get a chance to participate with us in seeing our goal become a reality. 2.) Are We Really Gurus? No. Not at all. However, with that being said, we do have A LOT of industry experience, we know the ins and outs of both, participating in a clinical trial as well as running an actual clinical trial from the Research Clinic’s perspective. Furthermore, we have the desire to discuss clinical trials and share our thoughts as we continue to run our company, South Coast Clinical Trials. The website, www.theclinicaltrialsguru.com was built to be the actual “guru”. We are not physicians, and will never give any medical advice. At no point in time will we ever attempt to sell you anything (unless it’s to other industry professionals) or convince you to join a trial. That is not the purpose of this website. This website is a side project of ours and was created as a by-product of our passion for this subject. We want those people who may be considering joining a clinical trial to be empowered and informed enough to make their own decisions about clinical trials, and to never blindly follow anyone else's advice.



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While many people have had great experiences with clinical trials, many have had terrible experiences, and it has therefore become very challenging for someone who has never volunteered for a trial to form an opinion of his or her own. We have searched the Web for some time and were unable to locate any sites that are purely informative outside of www.clinicaltrials.gov ,the FDA’s official site. Most of the clinical trial websites out there have an agenda, and their agenda is basically getting you into one of their studies. Rest assured, this is not our intention with this site. The research clinics that we manage serve very niche therapeutic indications and chances are that less than 2% of the population would even qualify for one of our studies. The main objective of this site is to inform you about the clinical trials process and allow you to become more educated about this industry. In a nutshell, this is clinical trials in plain English, and the real “guru” is every one's collective thoughts and experiences about clinical trials and their particular knowledge of the subject. For example, we’ve had cancer gurus talk about their experiences with trials, study coordinators share their thoughts on the industry, current and former trial participants share their feelings, and even clinical trial professionals from developing nations check in with us on the status of clinical trials overseas. Basically, we are all gurus in our own right. Our website is a platform designed to showcase all of the real gurus’ ideas out there. Now, enough about us and our website, and on to demystifying clinical trials. 3.) What Is A Clinical Trial? When a pharmaceutical company creates a new drug, they have to invest millions (and sometimes billions) of dollars into the entire research process of bringing the drug from an idea to reality. This entire process can take up to 15 years or more. The first step is to create a compound that their scientists think will work on a given indication, or a group of similar indications. These compounds are then tested on animals, and the compound eventually moves on to phase 1 studies in humans. Phase 1 studies usually seek healthy normal volunteers. “Normal” here means that these individuals do not actually have the disease or disorder that the drug proposes



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to treat. The only exception to this rule are sometimes for oncology (cancer) or psychiatric trials, although that is not always the case in these indications either. In this step of the trials process, the safety and tolerability (how the body tolerates it) of the drug is examined. These trials usually pay the most to the study participant, ranging from 1500 to 5000 (or more) U.S. dollars. These Phase 1 studies require the study volunteer to remain in an inpatient setting where they can be closely observed for any side effects and tolerability/safety issues. These studies usually last between 4-8 weeks (sometimes longer). Usually the compound is tested on less than 100 people in this phase. Phase 2 studies are a little larger in terms of the number of total study participants, and here, the major side effects are believed to have been determined from the Phase 1 trials. Phase 2 studies usually target the patient population for the indication that the drug proposes to treat. The optimum dose of the compound is attempted to be determined, however,the main objective is to look at safety of the drug, and any signs of efficacy (whether the drug works) in this patient population. Basically, a Phase 1 trial determines the safe dose ranges of a particular drug, and a Phase 2 trial determines whether the drug actually works within that dose range. If a 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.



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Phase 4 studies are usually post marketing studies. These studies are conducted after a drug has been approved by the FDA, but labeling issues still need to be addressed. Oftentimes, certain side effect issues need to be cleared up so a drug company can legally change what they can claim and advertise about their drug. These studies usually involve a large amount of study volunteers worldwide. Clinical trials are carried out according to standards enforced by the FDA (Food and Drug Administration) and the results of clinical trials have allowed many people with some diseases such as cancer to live longer. Such testing can also point the way to future research. However, many more drugs fail to make it through the entire approval process than the ones that do get approved. Your chances of an adverse event or side effect from the study drug significantly decrease as the trials move from Phase 1 to Phase 4, however all side effects are never completely discovered even after a drug is approved and on the market. Researchers will attempt to match a study participant’s medical history and the study inclusion/exclusion criteria to see which people can participate. The controversy here is that often the inclusion/exclusion criteria are so strict that one never truly knows how the drug would affect an individual who would not qualify for the study, but does have the indication that the drug proposes to treat. There are potential benefits and risks associated with participating in any trial. The potential study participant should research as much as he/she can before deciding to join a trial, and should ask the study doctor and study staff as many questions as possible. Once in a study, YOU MAY WITHDRAW AT ANY TIME FOR ANY REASON WHATSOEVER. The bottom line is that without clinical trials, new medications may never be developed to help find treatments and cures for a variety of diseases and disorders. Please never, ever, ever, ever, ever, ever, BLINDLY follow someone else’s advice on whether you should or should not join a particular trial. It is imperative that you do as much research as you possibly can about the clinical trial you are



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considering. If you need me to, I’ll put you in touch with a few “gurus” who may be able to offer some extra advice, but ultimately, the decision will be up to you and your family. 4.) Are Clinical Trials Safe? 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 myself and Don 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



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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. 5.) Your Rights As A Clinical Trial Participant If you are ever considering joining a clinical trial, there are a few major things you will need to know that are study-specific. These are risks and benefits of the trial, possible side effects, and nature of the study and procedures involved. However, no matter what study you may end up participating in, you need to know that you have many rights that protect you as a study volunteer. If you feel that any of these rights have been violated or not followed appropriately, you should contact the IRB (Institutional Review Board). They are the committee whose sole responsibility is to ensure the safety of trial participants, and their number will be on the Informed Consent that you have to sign before or on your first study visit. (More on Informed Consents later).



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Here are your rights: -You must be FULLY informed of the risks of the trial -You must give FULLY informed consent -You can stop participating at any time for any (or no) reason -You can refuse any research procedure done to you These are your basic rights in any study. It is in your best interest to be as informed as possible about all of the details of the study, but these are the big things you need to keep in mind. Just keep in mind that the research clinic also has the right to withdraw you from the study at any given time even if you still want to continue. These decisions are usually made by the Doctor in charge of the study, and believe it or not, happen often enough for me to mention here. Also, the research site does not have to pay you for incomplete visits, although they should pay you for the amount of total studies that you did complete. Issues regarding payment are also found in the Informed Consent, and it is your responsibility to ensure that you are being compensated accordingly. Throughout the study you should speak up about concerns and any questions you may have. Any reputable research clinic will make sure you have sufficient time to have your questions answered. Every state has their own variation of a Bill of Rights for people who participate in clinical trials. I happen to live and work in California, so I have posted the CA Bill of Rights for you to view below. No matter what state you live in, research clinics must follow Good Clinical Practice (GCP) which the FDA (Food and Drug Administration) enforces. Good Clinical Practice guidelines pretty much have these Bill of Rights covered as well. Here are the Bill of Rights: Experimental Research Subject’s Bill of Rights California law, under Health & Safety Code Section 24172, requires that any person asked to take part as a subject in research involving a medical experiment, or any person asked to consent to such participation on behalf of another, is entitled to receive the following list of rights written in a language in which the person is fluent. This list includes the right to:



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1. Be informed of the nature and purpose of the experiment. 2. Be given an explanation of the procedures to be followed in the medical experiment, and any drug or device to be utilized. 3. Be given a description of any attendant discomforts and risks reasonably to be expected from the experiment. 4. Be given an explanation of any benefits to the subject reasonably to be expected from the experiment, if applicable. 5. Be given a disclosure of any appropriate alternative procedures, drugs or devices that might be advantageous to the subject, and their relative risks and benefits. 6. Be informed of the avenues of medical treatment, if any, available to the subject after the experiment if complications should arise. 7. Be given an opportunity to ask any questions concerning the experiment or the procedures involved. 8. Be instructed that consent to participate in the medical experiment may be withdrawn at any time and the subject may discontinue participation in the medical experiment without prejudice. 9. Be given a copy of the signed and dated written consent form. 10. Be given the opportunity to decide to consent or not to consent to a medical experiment without the intervention of any element of force, fraud, deceit, duress, coercion, or undue influence on the subject’s decision.



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6.) Your Privacy HIPAA The fear of someone getting a hold of your personal information is understandable. In this day and age, identity theft is on the rise, and it makes sense to be extra vigilant in regards to your personal information. Many people have this fear when it comes to clinical trials. It is important to understand that laws and regulations exist to protect your personal information when you participate in clinical trials. The only people who have direct access to your personal health info when in a study are the individuals immediately involved with your care at the research clinic. Thats it. No one else. No drug companies, no marketers, no insurance companies, nada. Study centers are responsible for de-identifying all personal info from an individual in the trial. Therefore, when pharmaceutical companies go to review the data, they don’t see your name and social security number, but they see a unique study identifying number to identify you and all the data from the study associated with you. Therefore, you can rest assured that you will not be getting junk mail or spam from anyone because of your participation in a clinical trial. Furthermore, your insurance company has no way of knowing that you participated in a clinical trial either. (Some exceptions with insurance companies do exist with cancer trials so ask your study doctor about that first). 7.) Informed Consent & Your First Study Visit During your first visit to the study center (screening visit), you will come across a lot of important forms and information that you will have to sort through as you come one step closer towards making your decision about joining the study. I recommend you bring a family member or a loved one with you to help guide you in your decision making process. If you got to the point where you are even showing up to your first study visit, you have already come a long way. Chances are, you’ve been pre-screened over the phone or in-person by someone at the research center. Some research centers are hybrids of study centers and a physician private practice. My company, SCCT, falls under this category. In these cases, your physician conducts studies within his practice and chances are he might



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mention a clinical trial to you whenever he deems it appropriate. If he happens to be conducting a trial that he feels may be beneficial to you, he will tell you about it. Often, this is the best way to get introduced to the world of clinical trials. You and your doctor have an existing relationship, and many people first hear about clinical trials through their physicians. In my opinion, these types of research centers are ideal for both the study participant and the drug companies to deal with but of course, I’m a little biased. Regardless of the type of study center, screening visits are virtually the same. The first (and most important) procedure is going over the Informed Consent. You will sit in a private room with the doctor or a member of his study team. The member of the study team will go over the purpose of the study, the risks, benefits, known side effects, as well as all procedures that will be required during the trial. These forms usually range anywhere from 9 pages to over 26 in some cases, and basically spell out every last detail of the study. These are legal forms that require signatures and dates and initials and resemble any typical legal document that you can think of. You should be encouraged to ask any questions regarding the study, and can take as long as you want in terms of making a decision on whether to join the study or not. You should even be encouraged to take the form home and discuss it with your family or other support system. As a side note, you can withdraw from the study at anytime, even immediately after signing the consent form, and for any reason. Once the consent form has been explained, discussed and signed by all applicable parties (some studies require a caregiver or other such individual to be present during this process) you will begin the procedures of the actual study. This is where studies differ amongst themselves depending on the therapeutic indication or phase of the study. Virtually all studies will require blood draws, urine samples (for drugs and pregnancy), ECG’s (monitor’s your heart activity), vital signs, and some type of study participant survey such as quality of life assessments or something along those lines. The purpose of all of this is to capture your data before you start the study drug.



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You most likely will NOT be given the study drug at your first visit. They want to make sure that you are healthy enough to join the trial and that you don’t have any pre-existing medical conditions that might make it seem as if the study drug caused them. Once it’s determined that you are healthy enough to take the study drug, you will be scheduled a 2nd study visit. With all the hoopla that comes with a screening visit, you don’t even take the study drug until a later study visit, and you very well may not even qualify for those next visits due to a number of different reasons that may or may not even be clinically significant. You also may have changed your mind between the screening visit and your next visit, and chose to quit the trial. You have every right to do so. 8.) IRB’s Protect Safety and Rights of All Trial Partcipants An IRB stands for Institutional Review Board. The function of the IRB is to ensure that every study participant is safe while they are in a clinical trial. Furthermore, they ensure that the research clinics and drug companies conducting the trials are respecting the rights of the study participants, as determined by the FDA and Good Clinical Practice (GCP) guidelines. Every clinical trial is overseen by an IRB. Before a trial even begins enrolling study volunteers, the IRB ensures that no human being will be put under any unnecessary risk. For example, if a drug company is testing a cancer drug for late stage cancer, the IRB ensures that all study participants will either receive the study drug or the best standard of treatment that has already been approved. This prevents these types of trials from using placebo (sugar pill) and therefore, putting trial participants under unnecessary risk and immediate danger, if they should randomly be assigned to the placebo. The IRB in charge of your particular trial will be listed in the Informed Consent Form, and if you feel that your rights or safety have been compromised because of the clinical trial, you have the right to call them and explain what is going on. The IRB’s are supposed to make decisions based on what would benefit the safety and



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privacy of the clinical trial participants. In order to ensure that they are truly looking out for the study participant’s rights, the FDA routinely audits the IRB’s (they even conduct undercover “sting” operations routinely). For the trial participants out there, it is important to understand that independent boards such as IRB’s exist for the sole purpose of protecting your rights and safety. For much more on IRB’s, just go to our site www.theclinicaltrialsguru.com and do a search on IRB. 9.) You are More Valuable Than You May Think. Clinical Trials are the lifeblood of the pharmaceutical industry. The one metric that makes a clinical trial successful is patient enrollment, more specifically, the amount of TIME it takes to complete enrollment for a particular trial. The longer it takes a drug company to complete a clinical trial, the less time they have to potentially sell that drug and reap the profits if the drug were to be approved. In other words, every day that a study is delayed due to any factors, but most likely slow enrollment, is one day less that the drug company can earn revenue. I think the figure is around 80% of all studies experience some sort of delay due to a slower than expected rate of enrollment. (Anyone feel free to correct me if I am wrong, but like always, the exact number is not important, it is the concept). I don’t foresee patient compensation increasing to speed up enrollment rates as this would be considered unethical by the Institutional Review Boards that monitor the safety and ethical implications of every study on an ongoing basis. Take my word for it, if research sites were allowed to pay you more for your participation, they would, and the cash rich research centers would dominate the market. I also don’t think study procedures will get any easier, as the FDA (Food and Drug Administration) is getting stricter as to the data that they are demanding to review after a trial is finished. What I see happening is pharmaceutical companies and research centers getting more creative in their methods of advertising for clinical trials. As a study participant, you may soon be overwhelmed by the amount of options you have if you are considering volunteering for a study.



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I see a greater number of clinical trials in general as drug companies are testing the same drug for multiple diseases and disorders. I see a lot of advertising online in the very near future. With all of these new methods of recruiting you, it is imperative that you get informed about clinical trials in general, and more specifically, your rights as a trial participant. You will need to be aware of this so that if you one day choose to participate in a trial, you will know whether the research center is truly looking out for your best interests or not, and also whether the particular trial you are being recruited for is actually the best trial for you (all trials are NOT the same). Essentially, you will become a “guru”. 10.) Therapeutic Misconception Better medical attention in clinical trials, yes. Medical care, no. Let’s get one thing crystal clear. Clinical trials are designed to collect data so that eventually, better and better drugs can be developed. As a clinical trials professional, I would love to tell you that clinical trials will also provide you with excellent medical treatment and care. I would be lying to you. Unless you are using clinical trials as an alternative of last resorts (life threatening conditions that have not responded well to available treatments), you should not go in to a study expecting any type of treatment. After all, you may very well get placebo. However, this does not mean that your participation will be nightmarish, or that your condition will get worse. It simply means that you should be aware of the context of your participation in the study in the greater scheme of things. The drug companies want their drugs to perform well in these studies. Therefore, they are expecting that the individuals on placebo should not do well in the study relative to their non-placebo taking counterparts. In fact, if a drug does not show separation from placebo, there is very little use for the drug. If the drug companies don’t have unrealistic expectations for everyone who joins a study, neither should you. Some people will do very well, others will stay the same,



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and some will get worse. So while participating in a trial does not guarantee a treatment, it also does not mean that you shouldn’t participate. The medical attention (not treatment) that EVERY participant receives in a clinical trial is second to none. Every function of your body will be analyzed and you may end up learning something new about your health that otherwise went unnoticed (this has happened in our studies many times). This is simply not economically possible anywhere outside of a research setting where the medical bill is paid for by the sponsors. Therefore, if you have been frustrated by a lack of medical attention to your health, clinical trials may be a viable option. Finally, if you really are not doing well in the trial, you have the right to withdraw at any time for any reason, and will be given appropriate after trial care. Some Clinical Trials Guarantee You Will Receive Medication As mentioned before, many clinical trials involve placebo (sugar pill) and many patients and doctors shy away from encouraging their patients to participate in these types of studies due to fear that the patient will receive placebo and therefore experience a worsening of their condition. I certainly would not want to risk the possibility of receiving a placebo and place my health in jeopardy no matter how promising the experimental drug may be. For these people, there is some great news. Many phase 3 studies (the phase that recruits the largest number of participants) do not have a placebo arm in the trial. In these studies, the experimental drug is being compared with the current standard of treatment that already exists on the market. In many cases, the patient may very well have already been on this standard of treatment before he even thought about joining a study. Should this patient join one of these studies, he would be randomly assigned to either this same medication, or the new experimental medication. If the patient is currently doing well on his current medication, he is risking the fact that he may be randomly assigned to receive the experimental medication. This “risk” may very well turn out to work even better on the patient compared to his previous medication.



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Even if the experimental drug does not work as well as the patient’s previous medication, chances are that it most likely still works better than a placebo. When you factor in the extremely thorough medical attention the study participant is receiving (1-5 total hours per study visit), it becomes clear that these types of phase 3 studies provide the greatest potential benefit for the trial participant. As an extra safety measure, if the experimental drug causes unanticipated side effects that may cause immediate harm to the patient, the Investigator is required to withhold the study drug, and place the patient back on their previous medication once the adverse event is resolved. Of course, no blanket statements can be made as each individual’s benefits can only be observed on a case by case basis, but these types of studies offer the most possible benefits and the least degree of risk to the trial participants. The thing that still keeps some patients and their doctors uncomfortable is the fact that the treatment arms in these studies are blind and randomized. In other words, you won’t know what medication you are receiving from either the experimental drug or the established standard drug already available on the market, and neither will the study doctor. Because these studies offer the most potential benefits with the least degree of risk for the trial participant, they are extremely popular and study enrollment goals are usually achieved rapidly. You may not even see advertisements for these types of trials as there is no real need for it due to the fact that the study doctor has already told his own patients about the trial and his patients probably signed up for it. If you are seeking studies with the greatest upside and least risk, these are the trials you should be seeking, but you may have to look hard for them and be proactive. Basically, the less advertisement you see or hear about a trial, the better the study may be for someone like you (my opinion).



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11.) Money, Money, Money 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.



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12.) Know Thy Expectations What are your expectations from a clinical trial? If you are like me, you may believe in the idea of the clinical trials process and are willing to give it a shot. If you are able to weigh the benefits against the risks of the trial, do your homework and are aware of what phase of the clinical development process the study is currently in, and you are ok with ALL of the proposed study procedures, then a clinical trial just MIGHT be right for you. If you reach this point of not only this e-book, but also of your decision making process (and you should involve family if at all possible), then you should ask yourself what you expect to get out of the trial. You may answer numerous things some of which may be: for the money, to obtain better treatment than I’m currently getting, to obtain better medical attention than I’m currently receiving, to give me something to do in my spare time, to help others, or sometimes even to perhaps get cured of some condition. If anyone has any other reasons for joining a trial, let me know! Once you understand your expectations, you are able to make a more informed decision regarding the study, as well as asking the research staff educated questions in order to weigh your expectations against the possible risks. If everything at this point still seems ok for you then it is appropriate for you to join the study. During the course of the trial you may find that all of your expectations were met, some of your expectations were met, or none of your expectations were met. For example, if your main motivation is money, as in most of the people who join Phase 1, healthy volunteer studies (I don’t know why you would do this type of study without money being a major motivating factor), you may have a successful experience as long as your study went relatively smoothly and you got your payment at the end of the trial. If your body had no significant side effects, you will consider your trial a success.



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In phase 2-4 studies, the situation gets a little more complex. Money is still important, but you probably are seeking to be treated or even cured of your condition for which you are currently dealing with. The risks here are several, although lesser in magnitude than the potential risks of a Phase 1 trial. You risk getting placebo and having your condition worsen, receiving the study drug and your condition worsening, receiving study drug and experiencing an unpleasant side effect, receiving the comparator drug and your condition worsening or staying the same. Conversely, you can receive the study drug and respond better to it than you do to your previous medication, or be cured altogether (very small chance but may happen). You can also receive the comparator drug and have your condition improve. You may even receive placebo and have your condition improve (especially in psychiatric trials). By knowing these various possible outcomes, and understanding your expectations, you can make an informed decision on whether to get into a trial or not. Your expectations and current satisfaction with your condition determine the degree of risk involved for you. No case is identical, and no blanket statements can be made other than the fact that science will benefit regardless of YOUR outcome. 13.) Conclusion We all believe in clinical trials, kind of… Whether or not you know it, you probably believe in the concept of clinical trials, more specifically, drug testing in humans. How so you may ask? Unless you are one of those rare individuals who does not believe in the need for ever taking prescription (or non prescription in some cases) medication, you are acknowledging the fundamental truth of clinical trials. If you ever took any kind of medication that was approved by the FDA, or the European equivalent, the medication that you took was at one point in time an



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investigational product in a clinical trial. This investigational product went through the entire clinical trials process, from pre-clinical development to animal studies. Once it got past that point, your medication entered a phase 1 healthy volunteer study. After phase 1 testing, the side effects were analyzed and testing began in patients for whom the drug was believed to work on. These same patients whom many people referred to as “guinea pigs” were amongst the first group of people to perhaps benefit from the desired actions of the study drug. With that being said, the other side of this coin is that for every drug that passed the clinical trials process and eventually gained FDA approval, many more drugs failed in their attempts to prove either safety or efficacy in one phase or another. In these cases, the study participants helped society at large by testing a product that for whatever reason, either did not work as well as an already approved medication, or was deemed unsafe to some extent by a regulatory agency. Some patients in the clinical trials for these “failed” drugs may have actually benefited from the drug by actually responding better to the investigational product than they did with approved products. In these cases, the patients at least would know that researchers are getting closer to finding a product that can improve not only their lives, but the lives of others. The argument that Big Pharma would make here is that the approved products make up for the failures of the other products that do not make it to market. With each failed trial, pharma gets closer and closer towards finding the perfect product for any given disease or disorder. Without clinical trials, none of this would be possible. Just remember this next time you reach for that Tylenol.

Date post:	11-Mar-2016
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