ISCR - Indian Society for Clinical Research

The abc of patient queries

Frequently Asked Questions
Q1. What is clinical research?
Clinical research literally means research performed in the clinic. By implication, clinical research involves patients and medical practitioners and is essential to diagnosis and treatment of disease. A large part of clinical research is directed towards confirming the effectiveness and safety of new medicines and systematically gathering information that will help maximize the benefits while minimizing the risks associated with the use of medicines to treat disease. Applying this information to medical practice helps us use medicines to our best advantage.
Q2. What are clinical trials ?
Clinical trials are ethically and scientifically designed studies to confirm or compare the effects of medicines used to treat disease in human patients. Clinical trials are only conducted after laboratory studies and studies of the use of a medicine in animals has indicated that the medicine would be sufficiently safe to use in humans, and is expected to have benefits that are at least as good, if not better than existing therapies.
Q3. Is it risky for patients to participate in clinical trials?
At a philosophical level, life itself is risky. We are always at risk of accident and injury. What we eat, drink, and inhale, including the air we breathe, carries with it some risk to our well-being, small though it may be. The act of taking a medicine, no matter how extensively tested, or how familiar, carries with it the risk of side effects. However, all effective medicines reduce the risks and discomforts associated with the disease for which the medicine is taken. Regulations across the world do not allow new medicines to be tested in human patients unless the expected benefits associated with taking a medicine are far in excess of the estimated risk of side effects. It is true, however, that some rare side effects of a new medicine may not have come to light at the time a trial is conducted, or that occasionally a new medicine may not turn out, at the end of the clinical research program, to be as effective as current therapy. These risks are usually very small and often more than compensated for by the possibility that the new medicine will turn out to be better, more effective, or have fewer side-effects. Moreover, many trials are conducted with products that are already extensively tested, approved, and marketed to millions of patients. Here, the risks may be no greater than when the same medicine is prescribed in the course of a normal medical consultation.
Q4. Are there any benefits of participating in a clinical trial?
While all patients who receive effective treatment for their disease will benefit from such treatment, those who participate in clinical trials often receive additional benefits. Trial patients usually receive free treatment. This usually includes a waiver of consultation fee, waiver of fee for investigations included in the trial plan such as blood and urine tests, x-ray, sonography, scanning, etc., and free supply of trial medicine/s. In some trials the patient also receives reimbursement of the cost of travel to the clinic or hospital. Additional payments may sometimes be made to encourage patients to participate. Trial patients are often asked to visit the hospital or clinic at a time different from those for other patients or are seen at a different room or clinic so that they do not have to wait in queue and the doctor can spend more time with the trial patient without holding up other patients. On occasion, trial patients may be the first to receive a revolutionary new medicine and experience dramatic or life-saving benefits unimagined in the past - sometimes years in advance of other patients.
Q5. How can I find out what trials are going on, and whether I can participate?
It is usually your doctor who will inform you of ongoing trials and your eligibility to participate. However, more often than not, your doctor may not be aware of trials going on in your city in which he or she is not an investigator. In order to increase patients' access to clinical trials we, at ISCR, have plans to build a directory of ongoing trials that will be available on this website and will provide patients with information on trials that they may be eligible for. On occasion sponsors of clinical trials publish advertisement in local newspapers to aid recruitment of patients to a clinical trial.
Q6. How do I ensure that the trial that I may be considering to participate in is being conducted ethically and that patients' safety and well-being will be well cared for?
Before enrolling in a clinical trial, it is worthwhile to acquaint oneself with the details of the study. Is the medicine to be used in the trial an investigational product or is it a marketed drug? For investigational products, written permission from the Drugs Controller General of India is required for each trial. In addition, patients have greater assurance of ethical practice if the study plan has been reviewed and approved by an Ethics Committee following Good Clinical Practice guidelines. In studies following such guidelines patients are given detailed information about the trial and potential associated risks and benefits before being asked to decide to participate.
Q7. How can I enroll for a clinical trial?
If you are suffering from a disease or health problem for which a clinical trial is currently ongoing in your city, you may be eligible to enroll. Ask your doctor, or call the number provided in a clinical trial advertisement. On your first contact with the trial staff you may be asked a few questions to make a preliminary assessment of your eligibility for the trial. If you are, with your consent, you may be asked to undergo more detailed screening tests to confirm eligibility. Once your eligibility is confirmed, you will be provided with full information on the trial. After fully understanding the information you may decide to participate in the trial. If required, you may request additional information. If you decide to participate, you will have to sign an "Informed Consent Form" and agree to abide by all the trial requirements that would have been explained to you.
Q8. If I change my mind, can I withdraw from the trial after I have signed the Informed Consent Form?
You can change your decision to participate and withdraw from a trial at any stage irrespective of whether you have, or have not, signed the "Informed Consent Form". You need not give any reasons for withdrawing from a study. Your investigator (trial doctor) is required to ensure that you receive the same quality of care irrespective of whether you are participating in a clinical trial or have withdrawn from the study. However, you will not be eligible for any trial-related benefits after you have withdrawn from a study.
Q9. If I benefit from the trial and want to continue to take the trial medicine even after the trial is over, what are my options?
If the trial was conducted using a marketed formulation, you should ask your doctor whether it would be appropriate for you to continue to take the medicine. If so, your doctor will give you a prescription which will allow you to buy the medicine from the pharmacy store or chemist shop and continue to use it. For investigational products, you may not be able to continue to receive the medicine after the trial is over. For short-course medicines continuation may not be required. For diseases that require continued treatment, you may have to switch to an alternative marketed medicine till such time that the trial medicine is released for sale. This is so because government regulations do not allow use of investigational product outside of a clinical trial. For life-threatening diseases, there may be an Extension Protocol or a Continued/Compassionate Use Program being run with the permission of regulatory authorities. If so, your trial doctor will be able to tell you how to enroll for such a program and continue to receive the study medicine till the time that it becomes commercially available or the development program is discontinued for whatever reason.
Q10. Who will pay for my treatment if I have a trial-related injury?
The sponsor of a clinical trial, usually a pharmaceutical company or a research organization, is morally bound to arrange for free treatment of side effects of a study drug, any illness or injury directly resulting from exposure to the study drug, or any injury directly related to procedures undertaken in the course of a clinical trial. While serious trial-related injury requiring major expenditure for treatment is rare, the sponsor has an obligation to ensure that the affected subject/s receive full and appropriate treatment. The cost of such treatment is borne by the sponsor or reimbursed to the patient by the sponsor. In order that this provision is not misused, the opinion of the investigator and, if required, that of experts appointed by the Ethics Committee may be taken to determine whether the injury is trial-related or not.
Q11. Why are patients asked to participate in clinical trials?
While we have made great progress in the last 100 years in our fight against disease and premature death from illness, the great majority of diseases still remain to be conquered. Discovering and developing new medicines to fight disease is a collaborative effort requiring contributions from industry, government, the medical community, and patients. Patients are asked to participate in the final stage of drug development - the stage where a few thousand patients volunteer to be the first to take the medicine, thus clearing the way for millions of others to benefit. Observations made during the process of clinical trials are vital to the proper use of the medicine after it is released for general use. Unless patients volunteer to participate in clinical trials, new medicines cannot be brought into the world.
Q12. Must I enroll for a clinical trial if my doctor asks me to?
A patients participation in a clinical trial is an entirely voluntary act. Your doctor may suggest your participation in a trial if he/she believes it to be in your best interests. Your doctor may be hoping that the trial medicine will be more effective, or that you will be able to tolerate its effects more than your current medication, or simply that you will benefit from the convenience of its mode of administration or from free treatment. However, you must satisfy yourself that you will be able to make the required number of visits to the hospital/clinic for the trial. For most trials, the patient needs to visit the doctor more frequently than in the course of routine treatment. In some trials, when not considered inappropriate or harmful to the patients interests, dummy tablets, called placebo, may be used (see next question). In such cases you must satisfy yourself that you are comfortable with the use of dummy medication, whether or not in conjunction with other medicines. You must read the information sheet/s provided to you, and make your own judgment about participating in the trial. Some patients may find that the benefits of participating in a trial are not attractive enough to compensate for the inconvenience of repeated visits to the clinic or the possible risk of side effects, and that participation boils down to altruism and a willingness to contribute to science and society. If so, you must find out about the scope of the study and decide for yourself whether the value of the study in terms of generating new knowledge makes it worth your participation.
Q13. I am told that dummy medicines may be given to patients participating in clinical trials. Isn't this tantamount to fraud being perpetrated on unsuspecting patients?
Scientifically designed clinical trials often involve the use of dummy medication called placebo. The use of placebo is important to distinguish the effect of a medicine from the effects of the underlying disease or other factors such as natural changes in the disease process with the passage of time. Placebo is generally used in one of three ways: as run-in, as double dummy, and as a parallel group. In the run-in design, patients who complain of mild symptoms of a disease are given dummy medication to see whether the symptoms go away on their own or really require treatment with a medicine. Although it may seem strange but many patients with mild symptoms will respond to placebo, or report substantial benefit from dummy pills. This is especially true of chest pain, symptoms of anxiety, and mild elevations of blood pressure, sleep disturbances, and so on. Up to a third of all patients with these conditions report relief of symptoms with placebo. It is obvious that placebo responders should be excluded when the true effect of a medicine is being evaluated. The double dummy design has to do with masking the identity of medicines being given to patients - a technique called blinding. When two medicines are being compared in a clinical trial, it is important for patients and their doctors not to know which is which, because this may have a placebo effect on patients besides biasing the doctor in favor of one or the other of the two medicines. To achieve this, the two medicines are made to look the same and distributed to the patients in the trial in a random manner by the pharmacist according to a secret code. However, this is sometimes not possible, as when one of the two medicines is a tablet while the other is a capsule. It is in such circumstances that the double dummy technique is used - some patients receive a medicine tablet and a dummy capsule while other patients receive a medicine capsule and a dummy tablet. Neither patient nor doctor knows which medicine the patient is receiving - the capsule or the tablet. Blinded studies can also be conducted using the parallel group design. Here the aim is to compare a medicine and a look-alike dummy to see whether the medicine is any better than the dummy. Some patients receive the medicine while other patients receive the dummy. This design is only permissible when the disease is not particularly harmful to the patient and patients can afford to go without treatment for some time, for example patients seeking treatment for baldness or forgetfulness, or when the trial medicine is being given on top of other medicines to see whether it has any additional effects. In conclusion, dummy medicines are frequently used in clinical trials, but they are never used to the detriment of patients.
Q14. Is it true that doctors get money from pharmaceutical companies to conduct clinical trials?
Doctors and other hospital staff conducting a clinical trial for a pharmaceutical company are usually compensated for the time they spend on a clinical trial. Clinical trial patients are usually not required to pay consultation fees to the doctor. Consequently trial doctors lose the consultation fees that would otherwise have been paid to them by the patient. Moreover, trial doctors have to spend more time on a trial patient than on a normal patient, further losing out on earnings. Pharmaceutical sponsors therefore need to compensate the trial doctors for this loss besides reimbursing them for the expenses incurred for conducting the trial.
Q15. I think foreign pharmaceutical companies are using Indian patients as guinea pigs for their experiments. Why don't they do their trials in their own countries?
Almost all modern medicines that we use for treatment and prevention of diseases in our country have been discovered and developed in foreign countries. Patients in our country have benefited enormously from such medicines, be they antibiotics like penicillins or medicines for the heart, without having contributed much to their discovery and development. In recent years, however, the pace of research has increased tremendously. This has led to a relative shortage of eligible patients for clinical trials, and the speed of drug development has sometimes had to slow down because of slow recruitment of patients in clinical trials. India, being one of the worlds most populated nations, has a relatively large patient population, and worldwide patient recruitment in clinical trials can be considerably speeded up if India were to participate in international clinical trials. Some international pharmaceutical companies have therefore started inviting Indian doctors to participate in international clinical trials. This helps speed up the pace of drug development, while at the same time providing Indian doctors with valuable research experience and giving Indian patients the opportunity of early benefit from new medicines if they so desire. Foreign companies also find India attractive for clinical research because of the low cost of research, the presence of well-trained medical professionals, and the ease of communications.