Demystifying clinical-trials


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Demystifying clinical-trials

  1. 1. Clinical Trials Demystified Clinical Trials Demystified By Dan Sfera, Clinical Trials Guru Table of Contents1.) Introduction 32.) Are We Really Gurus? 43.) What Is A Clinical Trial? 54.) Are Clinical Trials Safe? 85.) Your Rights As A Clinical Trial Participant 96.) Your Privacy 127.) Informed Consent & Your First Study Visit 128.) IRB’s Protect Safety and Rights of All Trial Partcipants 149.) You are More Valuable Than You May Think 1510.) Therapeutic Misconception 1611.) Money, Money, Money 1912.) Know Thy Expectations 2013.) Conclusion 21Copyright © 2010 2
  2. 2. Clinical Trials Demystified1.) IntroductionFirst of all, both Don and myself believe it’s important to tell you a little about usand what we hope this e-book and our website, The Clinical Trials Guru willaccomplish, both in terms of helping the clinical trials industry, as well as society asa whole (I’m reaching just a little). My name is Dan Sfera and I have worked inthe clinical trials industry as a Research Site Director for the past 6 years. Prior tothat, I was involved in clinical trials as a Research Study Coordinator while I wasan undergraduate at The University of Arizona. I currently am a majority owner ofSouth Coast Clinical Trials, Inc., a dedicated psychiatric research clinic located inSouthern California, and am responsible for Business Development and Marketingfor our three clinics: Anaheim, San Bernardino and Norwalk, CA.My colleague and co-guru, Don Walters, has been involved in the clinical trialsindustry for over 10 years, primarily as a Recruitment Specialist. He currentlyserves as the Director of Community Outreach for South Coast Clinical Trials. Asa recruitment guru, Don understands the issues that research clinics have to dealwith in order to meet their enrollment numbers. If you ever have any questionsabout recruitment and clinical trials, send Don an email.The purpose of our website and this e-book are basically to help demystify theclinical trials industry by talking about it in plain English, and doing soEVERYDAY. Our intended audience are people who may be considering aclinical trial for themselves, or for a family member or a friend. Our intentions areNOT to enroll you into a study, or to convince you that clinical trials are your bestoptions in seeking better medical treatments (although sometimes that is indeedtrue). An interesting thing that occurred as our website evolved, is the fact thatresearch professionals and clinical trial industry types have also started interactingwith us through Twitter, Facebook, LinkedIn, and our company Fan Page onFacebook, and many have even contributed their ideas to our audience by allowingus to interview them (in most cases even live and unscripted!), and welcoming theidea of being featured in our videos. For an industry that is notoriously somysterious and secretive, we found that to be a welcome occurrence, and we’ve gotCopyright © 2010 3
  3. 3. Clinical Trials Demystifiedplenty 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 andauthentic content, engaging with study participants (some who regularly appear inour videos), former study participants, industry leaders, and industry insiders, arejust some of the things our audience expects from us everyday on our site. Weunderstand that trust is a hard thing to earn in this industry, and we too arefrustrated by the lack of educational and useful content that the clinical trials spaceapparently suffers from (both online and offline). The day we stop providingauthentic and useful information on clinical trials is the day someone should pullthe plug on our blog. Our goal is to empower people who are interested, to knowmore about clinical trials than they already do. We hope you get a chance toparticipate 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 industryexperience, we know the ins and outs of both, participating in a clinical trial as wellas running an actual clinical trial from the Research Clinic’s perspective.Furthermore, we have the desire to discuss clinical trials and share our thoughts aswe continue to run our company, South Coast Clinical Trials. The website, was built to be the actual “guru”.We are not physicians, and will never give any medical advice. At no point intime will we ever attempt to sell you anything (unless it’s to other industryprofessionals) or convince you to join a trial. That is not the purpose of thiswebsite. This website is a side project of ours and was created as a by-product ofour passion for this subject. We want those people who may be consideringjoining a clinical trial to be empowered and informed enough to make their owndecisions about clinical trials, and to never blindly follow anyone elses advice.Copyright © 2010 4
  4. 4. Clinical Trials DemystifiedWhile many people have had great experiences with clinical trials, many have hadterrible experiences, and it has therefore become very challenging for someone whohas never volunteered for a trial to form an opinion of his or her own. We havesearched the Web for some time and were unable to locate any sites that are purelyinformative outside of ,the FDA’s official site. Most of theclinical trial websites out there have an agenda, and their agenda is basically gettingyou 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 andchances are that less than 2% of the population would even qualify for one of ourstudies. The main objective of this site is to inform you about the clinical trialsprocess 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 ones collectivethoughts and experiences about clinical trials and their particular knowledge of thesubject. For example, we’ve had cancer gurus talk about their experiences withtrials, study coordinators share their thoughts on the industry, current and formertrial participants share their feelings, and even clinical trial professionals fromdeveloping 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 toshowcase all of the real gurus’ ideas out there. Now, enough about us and ourwebsite, 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 thedrug 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 agiven indication, or a group of similar indications. These compounds are thentested on animals, and the compound eventually moves on to phase 1 studies inhumans.Phase 1 studies usually seek healthy normal volunteers. “Normal” here means thatthese individuals do not actually have the disease or disorder that the drug proposesCopyright © 2010 5
  5. 5. Clinical Trials Demystifiedto treat. The only exception to this rule are sometimes for oncology (cancer) orpsychiatric 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 toleratesit) of the drug is examined. These trials usually pay the most to the studyparticipant, ranging from 1500 to 5000 (or more) U.S. dollars. These Phase 1studies require the study volunteer to remain in an inpatient setting where they canbe closely observed for any side effects and tolerability/safety issues. These studiesusually last between 4-8 weeks (sometimes longer). Usually the compound istested 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 thePhase 1 trials. Phase 2 studies usually target the patient population for theindication that the drug proposes to treat. The optimum dose of the compound isattempted to be determined, however,the main objective is to look at safety of thedrug, 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 aPhase 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 usuallyrecruit a large number of study participants, usually between 500 and 2500 totalparticipants. The dose and the majority of side effects are believed to have beendetermined by this point, so the main objective is to determine whether the drugworks in comparison to a placebo (sugar pill). Safety and side effects are stillclosely observed as well. These studies usually occur in outpatient settings andinvolve many study visits (between 10-30 at times). If the drug proves to haveefficacy (does it actually work?) over placebo, or a comparable drug that is alreadyapproved and on the market, the drug company will file an NDA (New DrugApplication) for FDA review. The FDA may take as long as 1 year to make adecision on whether the drug will be approved, or whether even more trials areneeded to determine safety or efficacy. At this point, labeling issues (whatinformation appears on the bottle, and what the drug company can claim regardingthe drug) are addressed.Copyright © 2010 6
  6. 6. Clinical Trials DemystifiedPhase 4 studies are usually post marketing studies. These studies are conductedafter a drug has been approved by the FDA, but labeling issues still need to beaddressed. Oftentimes, certain side effect issues need to be cleared up so a drugcompany 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 (Foodand Drug Administration) and the results of clinical trials have allowed manypeople with some diseases such as cancer to live longer. Such testing can also pointthe way to future research. However, many more drugs fail to make it through theentire approval process than the ones that do get approved. Your chances of anadverse event or side effect from the study drug significantly decrease as the trialsmove from Phase 1 to Phase 4, however all side effects are never completelydiscovered even after a drug is approved and on the market.Researchers will attempt to match a study participant’s medical history and thestudy inclusion/exclusion criteria to see which people can participate. Thecontroversy here is that often the inclusion/exclusion criteria are so strict that onenever truly knows how the drug would affect an individual who would not qualifyfor 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 beforedeciding to join a trial, and should ask the study doctor and study staff as manyquestions as possible. Once in a study, YOU MAY WITHDRAW AT ANYTIME FOR ANY REASON WHATSOEVER.The bottom line is that without clinical trials, new medications may never bedeveloped 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 adviceon whether you should or should not join a particular trial. It is imperative thatyou do as much research as you possibly can about the clinical trial you areCopyright © 2010 7
  7. 7. Clinical Trials Demystifiedconsidering. If you need me to, I’ll put you in touch with a few “gurus” who maybe able to offer some extra advice, but ultimately, the decision will be up to you andyour family.4.) Are Clinical Trials Safe?One of the first things people will ask themselves when considering joining aclinical trial: “is this safe for me?” This is a very good question to ask yourself, butin all honesty, no one can claim that any clinical trial will be 100% risk free. Ifanyone 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 hearsomeone claim that there are no risks associated with joining a clinical trial, RUNaway, and tell myself and Don all about it here. After all, there wouldn’t be a needfor 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 questionthat is absolutely, without a doubt, 100% yes, then a clinical trial is just not rightfor you. Once you reach the decision to continue exploring the possibility ofjoining 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 thedrug are nowhere near being understood or discovered. Up until this point, thedrug was tested in animals, but the effects on humans is virtually unknown outsideof the theories established by scientists. However, these types of studies pay themost money to its participants, and many people are perfectly ok with the risks inorder to get their compensation. The possibility of getting placebo also plays a rolehere. It is from these studies that the terms “human lab rat” and “professionalguinea pig” originated.Phase 2 and 3 studies have relatively similar degrees of risks. The toxicity of thedrug as well as its major side effects have most likely been determined. Now thedrug is being tested on the actual targeted population that the drug was designedCopyright © 2010 8
  8. 8. Clinical Trials Demystifiedfor. For example, a Phase 1 trial may test a diabetes drug on a healthy person, butin a Phase 2 and 3 trial, a person with diabetes will test the study drug. The riskshere are still side effects that may be unknown, but now we also throw in thepossibility of your disease or disorder worsening rather than improving. With thisadded risk of a worsening condition due to getting a placebo or perhaps the factthat the drug doesn’t work, you must calculate whether you should consider joiningthe trial if you are actually doing well with your current treatment. These studiesalso pay significantly less than the phase 1 studies due to less relative risks, and thepossibility 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 asyou were before.With all these different possibilities and combinations of risks versus yourexpectations, you can see that the subject of clinical trials truly varies on a case bycase basis, and almost no blanketed statements can be made except for perhaps thefact that no matter what the outcome for your particular case, data was collectedand science was advanced, if even a little.5.) Your Rights As A Clinical Trial ParticipantIf you are ever considering joining a clinical trial, there are a few major things youwill 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, nomatter what study you may end up participating in, you need to know that youhave many rights that protect you as a study volunteer. If you feel that any of theserights have been violated or not followed appropriately, you should contact theIRB (Institutional Review Board). They are the committee whose soleresponsibility is to ensure the safety of trial participants, and their number will beon the Informed Consent that you have to sign before or on your first study visit.(More on Informed Consents later).Copyright © 2010 9
  9. 9. Clinical Trials DemystifiedHere 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 youThese are your basic rights in any study. It is in your best interest to be as informedas possible about all of the details of the study, but these are the big things youneed to keep in mind. Just keep in mind that the research clinic also has the rightto 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 believeit or not, happen often enough for me to mention here. Also, the research site doesnot have to pay you for incomplete visits, although they should pay you for theamount of total studies that you did complete. Issues regarding payment are alsofound in the Informed Consent, and it is your responsibility to ensure that you arebeing compensated accordingly. Throughout the study you should speak up aboutconcerns and any questions you may have. Any reputable research clinic will makesure you have sufficient time to have your questions answered.Every state has their own variation of a Bill of Rights for people who participate inclinical trials. I happen to live and work in California, so I have posted the CA Billof Rights for you to view below. No matter what state you live in, research clinicsmust follow Good Clinical Practice (GCP) which the FDA (Food and DrugAdministration) enforces. Good Clinical Practice guidelines pretty much havethese Bill of Rights covered as well.Here are the Bill of Rights:Experimental Research Subject’s Bill of RightsCalifornia law, under Health & Safety Code Section 24172, requires that anyperson asked to take part as a subject in research involving a medical experiment, orany person asked to consent to such participation on behalf of another, is entitledto receive the following list of rights written in a language in which the person isfluent. This list includes the right to:Copyright © 2010 10
  10. 10. Clinical Trials Demystified1. Be informed of the nature and purpose of the experiment.2. Be given an explanation of the procedures to be followed in the medicalexperiment, and any drug or device to be utilized.3. Be given a description of any attendant discomforts and risks reasonably to beexpected from the experiment.4. Be given an explanation of any benefits to the subject reasonably to be expectedfrom the experiment, if applicable.5. Be given a disclosure of any appropriate alternative procedures, drugs or devicesthat 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 subjectafter the experiment if complications should arise.7. Be given an opportunity to ask any questions concerning the experiment or theprocedures involved.8. Be instructed that consent to participate in the medical experiment may bewithdrawn at any time and the subject may discontinue participation in themedical 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 medicalexperiment without the intervention of any element of force, fraud, deceit, duress,coercion, or undue influence on the subject’s decision.Copyright © 2010 11
  11. 11. Clinical Trials Demystified6.) Your PrivacyHIPAAThe 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 extravigilant in regards to your personal information. Many people have this fear whenit comes to clinical trials. It is important to understand that laws and regulationsexist 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 astudy 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 anindividual in the trial. Therefore, when pharmaceutical companies go to review thedata, they don’t see your name and social security number, but they see a uniquestudy identifying number to identify you and all the data from the study associatedwith you. Therefore, you can rest assured that you will not be getting junk mail orspam from anyone because of your participation in a clinical trial. Furthermore,your insurance company has no way of knowing that you participated in a clinicaltrial either. (Some exceptions with insurance companies do exist with cancer trialsso ask your study doctor about that first).7.) Informed Consent & Your First Study VisitDuring your first visit to the study center (screening visit), you will come across alot of important forms and information that you will have to sort through as youcome one step closer towards making your decision about joining the study. Irecommend you bring a family member or a loved one with you to help guide youin your decision making process. If you got to the point where you are evenshowing 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 atthe research center. Some research centers are hybrids of study centers and aphysician private practice. My company, SCCT, falls under this category. In thesecases, your physician conducts studies within his practice and chances are he mightCopyright © 2010 12
  12. 12. Clinical Trials Demystifiedmention a clinical trial to you whenever he deems it appropriate. If he happens tobe 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 andyour doctor have an existing relationship, and many people first hear about clinicaltrials through their physicians. In my opinion, these types of research centers areideal for both the study participant and the drug companies to deal with but ofcourse, I’m a little biased.Regardless of the type of study center, screening visits are virtually the same. Thefirst (and most important) procedure is going over the Informed Consent. You willsit in a private room with the doctor or a member of his study team. The memberof the study team will go over the purpose of the study, the risks, benefits, knownside effects, as well as all procedures that will be required during the trial. Theseforms usually range anywhere from 9 pages to over 26 in some cases, and basicallyspell out every last detail of the study. These are legal forms that require signaturesand dates and initials and resemble any typical legal document that you can thinkof. You should be encouraged to ask any questions regarding the study, and cantake as long as you want in terms of making a decision on whether to join the studyor not. You should even be encouraged to take the form home and discuss it withyour family or other support system. As a side note, you can withdraw from thestudy at anytime, even immediately after signing the consent form, and for anyreason.Once the consent form has been explained, discussed and signed by all applicableparties (some studies require a caregiver or other such individual to be presentduring this process) you will begin the procedures of the actual study. This is wherestudies differ amongst themselves depending on the therapeutic indication or phaseof the study. Virtually all studies will require blood draws, urine samples (for drugsand pregnancy), ECG’s (monitor’s your heart activity), vital signs, and some typeof study participant survey such as quality of life assessments or something alongthose lines. The purpose of all of this is to capture your data before you start thestudy drug.Copyright © 2010 13
  13. 13. Clinical Trials DemystifiedYou most likely will NOT be given the study drug at your first visit. They want tomake sure that you are healthy enough to join the trial and that you don’t have anypre-existing medical conditions that might make it seem as if the study drugcaused them. Once it’s determined that you are healthy enough to take the studydrug, you will be scheduled a 2nd study visit.With all the hoopla that comes with a screening visit, you don’t even take the studydrug until a later study visit, and you very well may not even qualify for those nextvisits due to a number of different reasons that may or may not even be clinicallysignificant. You also may have changed your mind between the screening visit andyour 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 PartcipantsAn IRB stands for Institutional Review Board. The function of the IRB is toensure that every study participant is safe while they are in a clinical trial.Furthermore, they ensure that the research clinics and drug companies conductingthe trials are respecting the rights of the study participants, as determined by theFDA and Good Clinical Practice (GCP) guidelines. Every clinical trial is overseen by an IRB. Before a trial even begins enrollingstudy volunteers, the IRB ensures that no human being will be put under anyunnecessary risk. For example, if a drug company is testing a cancer drug for latestage cancer, the IRB ensures that all study participants will either receive the studydrug or the best standard of treatment that has already been approved. Thisprevents these types of trials from using placebo (sugar pill) and therefore, puttingtrial participants under unnecessary risk and immediate danger, if they shouldrandomly be assigned to the placebo.The IRB in charge of your particular trial will be listed in the Informed ConsentForm, and if you feel that your rights or safety have been compromised because ofthe clinical trial, you have the right to call them and explain what is going on. TheIRB’s are supposed to make decisions based on what would benefit the safety andCopyright © 2010 14
  14. 14. Clinical Trials Demystifiedprivacy of the clinical trial participants. In order to ensure that they are trulylooking 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 independentboards 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 and doa search on IRB.9.) You are More Valuable Than You May Think.Clinical Trials are the lifeblood of the pharmaceutical industry. The one metricthat makes a clinical trial successful is patient enrollment, more specifically, theamount of TIME it takes to complete enrollment for a particular trial. The longerit takes a drug company to complete a clinical trial, the less time they have topotentially sell that drug and reap the profits if the drug were to be approved. Inother words, every day that a study is delayed due to any factors, but most likelyslow enrollment, is one day less that the drug company can earn revenue. I thinkthe figure is around 80% of all studies experience some sort of delay due to a slowerthan 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 thiswould be considered unethical by the Institutional Review Boards that monitor thesafety and ethical implications of every study on an ongoing basis. Take my wordfor it, if research sites were allowed to pay you more for your participation, theywould, and the cash rich research centers would dominate the market. I also don’tthink study procedures will get any easier, as the FDA (Food and DrugAdministration) is getting stricter as to the data that they are demanding to reviewafter a trial is finished. What I see happening is pharmaceutical companies andresearch centers getting more creative in their methods of advertising for clinicaltrials. As a study participant, you may soon be overwhelmed by the amount ofoptions you have if you are considering volunteering for a study.Copyright © 2010 15
  15. 15. Clinical Trials DemystifiedI see a greater number of clinical trials in general as drug companies are testing thesame drug for multiple diseases and disorders. I see a lot of advertising online inthe very near future. With all of these new methods of recruiting you, it isimperative that you get informed about clinical trials in general, and morespecifically, your rights as a trial participant. You will need to be aware of this sothat if you one day choose to participate in a trial, you will know whether theresearch center is truly looking out for your best interests or not, and also whetherthe particular trial you are being recruited for is actually the best trial for you (alltrials are NOT the same). Essentially, you will become a “guru”.10.) Therapeutic MisconceptionBetter medical attention in clinical trials, yes. Medical care, no.Let’s get one thing crystal clear. Clinical trials are designed to collect data so thateventually, better and better drugs can be developed. As a clinical trialsprofessional, I would love to tell you that clinical trials will also provide you withexcellent medical treatment and care. I would be lying to you.Unless you are using clinical trials as an alternative of last resorts (life threateningconditions that have not responded well to available treatments), you should not goin to a study expecting any type of treatment. After all, you may very well getplacebo. 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 ofthe context of your participation in the study in the greater scheme of things. Thedrug companies want their drugs to perform well in these studies. Therefore, theyare expecting that the individuals on placebo should not do well in the studyrelative to their non-placebo taking counterparts. In fact, if a drug does not showseparation from placebo, there is very little use for the drug.If the drug companies don’t have unrealistic expectations for everyone who joins astudy, neither should you. Some people will do very well, others will stay the same,Copyright © 2010 16
  16. 16. Clinical Trials Demystifiedand some will get worse. So while participating in a trial does not guarantee atreatment, it also does not mean that you shouldn’t participate. The medicalattention (not treatment) that EVERY participant receives in a clinical trial issecond to none. Every function of your body will be analyzed and you may end uplearning something new about your health that otherwise went unnoticed (this hashappened in our studies many times). This is simply not economically possibleanywhere outside of a research setting where the medical bill is paid for by thesponsors.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 thetrial, you have the right to withdraw at any time for any reason, and will be givenappropriate after trial care.Some Clinical Trials Guarantee You Will Receive MedicationAs mentioned before, many clinical trials involve placebo (sugar pill) and manypatients and doctors shy away from encouraging their patients to participate inthese types of studies due to fear that the patient will receive placebo and thereforeexperience a worsening of their condition. I certainly would not want to risk thepossibility of receiving a placebo and place my health in jeopardy no matter howpromising the experimental drug may be.For these people, there is some great news. Many phase 3 studies (the phase thatrecruits 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 currentstandard of treatment that already exists on the market. In many cases, the patientmay very well have already been on this standard of treatment before he eventhought about joining a study. Should this patient join one of these studies, hewould be randomly assigned to either this same medication, or the newexperimental medication. If the patient is currently doing well on his currentmedication, he is risking the fact that he may be randomly assigned to receive theexperimental medication. This “risk” may very well turn out to work even better onthe patient compared to his previous medication.Copyright © 2010 17
  17. 17. Clinical Trials DemystifiedEven if the experimental drug does not work as well as the patient’s previousmedication, chances are that it most likely still works better than a placebo. Whenyou factor in the extremely thorough medical attention the study participant isreceiving (1-5 total hours per study visit), it becomes clear that these types of phase3 studies provide the greatest potential benefit for the trial participant. As an extrasafety measure, if the experimental drug causes unanticipated side effects that maycause immediate harm to the patient, the Investigator is required to withhold thestudy drug, and place the patient back on their previous medication once theadverse event is resolved.Of course, no blanket statements can be made as each individual’s benefits can onlybe observed on a case by case basis, but these types of studies offer the mostpossible benefits and the least degree of risk to the trial participants. The thing thatstill keeps some patients and their doctors uncomfortable is the fact that thetreatment arms in these studies are blind and randomized. In other words, youwon’t know what medication you are receiving from either the experimental drugor the established standard drug already available on the market, and neither willthe study doctor.Because these studies offer the most potential benefits with the least degree of riskfor the trial participant, they are extremely popular and study enrollment goals areusually achieved rapidly. You may not even see advertisements for these types oftrials as there is no real need for it due to the fact that the study doctor has alreadytold 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 trialsyou 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 studymay be for someone like you (my opinion).Copyright © 2010 18
  18. 18. Clinical Trials Demystified11.) Money, Money, MoneyWhy 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 onlyat my own clinic, but also at industry conventions and other such events, choose todo studies for the payments. This especially becomes evident in phase 1, healthyvolunteer 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 doingstudies primarily for the payment, many people, especially those in phase 2-4studies, don’t consider payment the primary reason for doing studies. For manypeople that I’ve come across, traditional therapies that are already available on themarket do not work as well as previously anticipated, and these people seek newertreatments, 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 diseasebecome the primary motivator. Furthermore, many patients feel as though theyactually receive better medical attention when they participate in studies. This maybe true in many cases, as some outpatient study visits may take anywhere from 1-4hours, and all bases are pretty much covered in terms of a clinical perspective (vitalsigns, labwork, ECG’s, MRI’s, CT Scans, quality of life surveys, etc).Many of these procedures, while often important, will never get recommended in anon-study setting due to ever increasing pressure from insurance companies tokeep 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 systemactually is.Copyright © 2010 19
  19. 19. Clinical Trials Demystified12.) Know Thy ExpectationsWhat are your expectations from a clinical trial?If you are like me, you may believe in the idea of the clinical trials process and arewilling to give it a shot. If you are able to weigh the benefits against the risks of thetrial, do your homework and are aware of what phase of the clinical developmentprocess the study is currently in, and you are ok with ALL of the proposed studyprocedures, 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 makingprocess (and you should involve family if at all possible), then you should askyourself what you expect to get out of the trial. You may answer numerous thingssome of which may be: for the money, to obtain better treatment than I’mcurrently getting, to obtain better medical attention than I’m currently receiving, togive me something to do in my spare time, to help others, or sometimes even toperhaps get cured of some condition. If anyone has any other reasons for joining atrial, let me know!Once you understand your expectations, you are able to make a more informeddecision regarding the study, as well as asking the research staff educated questionsin order to weigh your expectations against the possible risks. If everything at thispoint 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. Forexample, if your main motivation is money, as in most of the people who joinPhase 1, healthy volunteer studies (I don’t know why you would do this type ofstudy without money being a major motivating factor), you may have a successfulexperience as long as your study went relatively smoothly and you got yourpayment at the end of the trial. If your body had no significant side effects, you willconsider your trial a success.Copyright © 2010 20
  20. 20. Clinical Trials DemystifiedIn phase 2-4 studies, the situation gets a little more complex. Money is stillimportant, but you probably are seeking to be treated or even cured of yourcondition 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 riskgetting placebo and having your condition worsen, receiving the study drug andyour condition worsening, receiving study drug and experiencing an unpleasantside effect, receiving the comparator drug and your condition worsening or stayingthe same.Conversely, you can receive the study drug and respond better to it than you do toyour previous medication, or be cured altogether (very small chance but mayhappen). You can also receive the comparator drug and have your conditionimprove. 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. Yourexpectations and current satisfaction with your condition determine the degree ofrisk involved for you. No case is identical, and no blanket statements can be madeother than the fact that science will benefit regardless of YOUR outcome.13.) ConclusionWe 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 oneof those rare individuals who does not believe in the need for ever takingprescription (or non prescription in some cases) medication, you are acknowledgingthe fundamental truth of clinical trials.If you ever took any kind of medication that was approved by the FDA, or theEuropean equivalent, the medication that you took was at one point in time anCopyright © 2010 21
  21. 21. Clinical Trials Demystifiedinvestigational product in a clinical trial. This investigational product went throughthe entire clinical trials process, from pre-clinical development to animal studies.Once it got past that point, your medication entered a phase 1 healthy volunteerstudy. After phase 1 testing, the side effects were analyzed and testing began inpatients for whom the drug was believed to work on. These same patients whommany people referred to as “guinea pigs” were amongst the first group of people toperhaps 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 passedthe clinical trials process and eventually gained FDA approval, many more drugsfailed in their attempts to prove either safety or efficacy in one phase or another. Inthese cases, the study participants helped society at large by testing a product thatfor 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 theclinical trials for these “failed” drugs may have actually benefited from the drug byactually responding better to the investigational product than they did withapproved products. In these cases, the patients at least would know that researchersare getting closer to finding a product that can improve not only their lives, but thelives of others.The argument that Big Pharma would make here is that the approved productsmake up for the failures of the other products that do not make it to market. Witheach failed trial, pharma gets closer and closer towards finding the perfect productfor any given disease or disorder. Without clinical trials, none of this would bepossible. Just remember this next time you reach for that Tylenol.Copyright © 2010 22