Want to be a test subject in a medical study?

1. What can I expect as a test subject?

Maybe the new medicine is better than the usual one, but maybe it isn’t. If one knew that in advance, then the investigation was not necessary. However, you do have a few certainties. The drug has already been tested on animals and has shown that it is safe and that it works as the researchers expect. Subsequently, the side effects were investigated. This usually happens with healthy volunteers, who are recruited through advertisements in the newspaper. If the most common side effects outweigh the benefits, the drug is tested on patients. At that stage, your doctor may ask you to participate in a study for a new drug. It examines exactly how good the drug is and whether there are still rare side effects.

It is sometimes different with cancer medicines. Because these drugs often have serious side effects, it is preferable to test them directly on patients after the animal research, and not on healthy volunteers. You will then have less information about safety and also do not know whether this medicine will reduce the complaints at all.

Participating in a study with a new drug therefore does not guarantee that you will find a treatment that responds better than the existing one. By participating, you actively help to find better treatment for people who get this disease after you.

2. What is the chance that I will receive the new medicine?

50 percent. In a study, a means is also chosen with which to compare, the ‘gold standard’. You will not be told in advance which medicine you will receive, but you will afterwards. Your doctor also usually doesn’t know if you’re getting the new or an existing drug. Incidentally, there is a chance that the existing medicine is different from what your doctor would have prescribed if you had not taken part in the study.

3. Can I also get a fake pill?

Yes, if there is no drug for a disease yet, the new drug is compared with a placebo. You will not hear that either. Participating in such a study therefore means a 50 percent chance that you will receive a fake pill. Sometimes placebos are also used in research into complaints or diseases for which there is already a treatment, but that is actually not by the book. In such a study you should be given either the old or the new drug and not a fake pill. So always ask why exactly the research is being conducted, whether placebos are being used, and what drug you would receive if you did not participate.

4. Why is research sometimes done with old medicines?

There are three reasons for this. Firstly, a study may be intended to determine whether a drug works in diseases other than those for which it is already used. Secondly, because manufacturers want to keep track of which side effects occur after the introduction of their product. Because the drug is used by more people in that case, new side effects sometimes come to light. These are so-called Post Marketing Surveillance surveys. It is not of much use to your own health, but you at least help the knowledge about that medicine forward. Thirdly, the manufacturer may conduct a study in an attempt to get the drug more prescribed (a seeding trial). In that case, the doctor receives a fee for administering the data and the research is not so much good for the knowledge of the medicine, but especially for the wallet of the manufacturer and the doctor.

5. How do I find out the purpose of an investigation?

You don’t always find out. You can of course ask your doctor why exactly the research is intended, but the distinction between a Post Marketing Surveillance and a seeding trial is not really clear even for doctors.

6. What is the risk of serious side effects?

Small, but how small is not exactly known. Before an investigation starts, it must be approved by a Medical Ethics Review Committee (METC). Approval guarantees that the safety of the participants has been considered. The METC estimates which side effects could occur and weighs this against the usefulness of the medicine. With a potentially fatal disease, people accept a greater chance of serious side effects than with a fairly harmless complaint such as fungal nails. You should receive this information from the doctor before deciding whether to participate in the study. An insurance policy has been taken out for all approved studies that must compensate for any damage suffered by participants.

The expertise of the members of the METCs is monitored by the CCMO, Central Committee for Research on Human Subjects. This is a government agency that implements the Medical Research Involving Human Subjects Act (WMO).
Post Marketing Surveillance and seeding trials do not always have to be approved by an MREC, because only studies have to be approved in which patients have to do something extra, such as donate blood, come extra often or fill in a questionnaire.

7. Do I have to decide quickly?

New. Even if you are in an acute situation, do not be caught off guard if you are approached. In any case, ask whether the research has been approved by an MREC. The information about the study can be difficult to read. Please take the time to review it and see what is expected of you when you participate in the study. A patient organization can help by assessing whether it is a good study.

8. How do I know if participating is best for me?

You can only determine that yourself by listing the pros and cons. The main benefit, of course, is that participating is your only chance of getting the new drug. Another plus is that patients in studies are usually closely monitored by doctors.

For some people, those extra visits to the doctor and possible physical examinations are actually a disadvantage. There are also objections that you can receive a fake pill or a different treatment than if you did not participate in the study. Furthermore, less is known about the possible side effects in a study.

9. Who can I ask for advice when in doubt?

An attending physician will primarily think about your well-being and will be able to advise you well from that point of view. But despite the fact that your doctor has to put your best interests first, a doctor who does his own research may have conflicting interests. In a seeding trial this can be financial gain, but in other studies the doctor may find the research itself very important or have a lack of participants. Unfortunately, you as a patient cannot assess this properly. There is no central point where you can have this assessed.

A solution in case of doubt is: if the specialist asks to participate, consult the general practitioner, and if the general practitioner asks: consult your specialist.

10. What investigations are underway?

You can ask your doctor about this. You can also search yourself and then inquire with the researchers whether the study is suitable for you. Overviews of studies, both with drugs and with other treatments, can be found on the Internet at:

• www.trialregister.nl: a register of Dutch studies, the results of which will be published in a leading scientific journal in due course.
• http://clinicaltrials.gov: an American site with an international
  overview of medical studies, including more than six hundred Dutch studies.
• www.ikcnet.nl/trials: a summary of Dutch studies in the field of cancer (in English).
• www.ccmo.nl: In the second half of this year, the CCMO will post a database of studies on its website.

11. What if I have a rare disease?

For research into rare diseases, you can inquire with the specialist or the patient association – if there is one. There are also overviews on the internet (see question 10). Because researchers in the Netherlands will find too few patients for a study into rare diseases, you will usually end up in an international study. Orphanet is an organization that maintains research on rare diseases. on www.orphanet.nl you can search the ‘Orphanet Database’. This contains more than six hundred studies in which patients can participate, a small part of which is Dutch. Through www.orpha.net you can also sign up as a volunteer for research.

Knowing more?
View on www.ccmo.nl (click on ‘for the public’) or ask your question directly to the Central Committee for Research on Human Subjects: 070-340 67 00.

Sources: Joop Bouma, Dr Cees Smit, VSOP, Marcel Kenter, CCMO, Siena van der Wilt, Trialbureau IKA.