Participation in a trial
Why are there clinical trials?
The search for new medicines starts with fundamental research in laboratories. Researchers attempt in laboratories to discover why and how a certain disease occurs. If they succeed, the next step is to develop a suitable treatment with medicines. What is developed in the laboratory must after all still prove its effectiveness in the clinic. This is achieved in clinical trials; study-based research in clinics.
Next to newly developed medicine, we also have trials that provide a new combination of medicine that is not tested out before. Lastly, it could also be the case that we think a medicine that is already on the market for a different disease might also be beneficial for hematologic diseases.
Why is it important to participate in a clinical trial?
Most people with cancer want to know the best treatment for their disease. The answer is mostly due to fellow sufferers who went before you and who have opted to participate in a clinical trial. After all, a great deal can be improved in cancer therapy and we are constantly working to determine the best therapy for your disease.
Studies with cancer cells on a petri dish, in a test tube or in mice, however, do not always correspond to what happens in reality with cancer patients. Participation in trials is the only way of obtaining more information and improving the treatment of cancer patients.
What are the benefits of a clinical trial?
Through clinical trials, the following can be achieved:
- Patients gain faster access to new, promising treatment methods;
- It sometimes emerges from a trial that a specific treatment that appeared promising is either not effective or unnecessary;
- The study results can result in determination of a new treatment standard for a specific form of cancer;
- A trial is a first step in making fundamental research (= research into the occurrence of cancer and possibilities for treating cancer) available for clinical practice;
- The results of a trial eventually lead to good practical medicine.
How does a clinical trial differ from ‘standard’ treatment?
If you participate in a clinical trial, you will receive more examinations and visits from the treating specialist than you would if you do not participate in a trial.
Trials are more often carried out in a specialist cancer center or a university (academic) hospital than ‘standard’ treatment. Nonetheless, it is also possible to participate in trials in general hospitals.
If you do take part in a trial, you will be informed in more detail than is normal for a ‘standard’ treatment. Another difference is that you must give explicit permission to participate in the treatment. You will follow a treatment timetable prescribed by your specialist. You will receive clear and honest information about this timetable. If you consider this information understandable and sufficient, you give permission for participation. This is known as ‘informed consent’.
If you participate in a trial, it is possible that your treatment team will be larger than with ‘standard’ treatment. Doctors, nurses, social workers and other professionals in health care may make up the treatment team. They will closely monitor the progress of your treatment process.
What kind of trials are there?
There are different phases through which new medicine, a new combination of medicine or medicine already on the market but tested for hematologic diseases, is tested. They are listed below.
These trials correspond with the first administration of the medicine in humans, following toxicological studies in animals. Phase 1-trials are aimed at identifying the behavior of the medicine in humans (absorption, distribution in the blood, excretion from the body). Phase 1-trials therefore have no immediate therapeutic interest, and are not implemented on patients with a specific disorder. These trials are implemented amongst healthy volunteers in specially-equipped rooms, after obtaining permission to carry out this kind of study. Both low and high dosages of medicines are tested. At the end of Phase 1, a zone of "safety dosages" is identified. The above does not apply to chemotherapy drugs in particular. Because chemo has so many side effects, it is not administered to healthy volunteers. Instead, patients that did not respond on any treatment before are asked if they want to participate in these types of studies. These results will determine the transfer of a medicine to Phase 2.
Phase 2 trials have the main objective of determining whether the medicine is indeed effective for the intended indication. The search is also implemented to find the optimum dosage for achieving this effectiveness with the minimum side effects. These trials generally consist of a limited number of very specific trials. For example a trial for identifying the dosage, whereby various dosages of the medicines are compared with one another in order to determine the optimum dosage. These trials are carried out on patients with the disorder to be treated with the medicine. These clinical Phase 2 trials are controlled studies. This means that a comparison is made between the new medicin and a placebo. Sometimes even with a third existing medicine that acts as the control. Based on these comparisons, any distortion of the results of the trials is avoided. The information obtained in Phase 2 determines the transition of the medicine to Phase 3.
Phase 3 consists of a series of trials aimed at better understanding the characteristics of the medicines and differentiating this medicine from other existing medicines. These trials differ from Phase 2 trials by the larger number of patients involved (sometimes thousands) and by the longer duration of these trials. In these Phase 3 trials, more specific populations are also investigated. The initial information leaflet for the medicine is drawn up on the basis of the results of the trials carried out to date. In this way, indications for the use of the medicine can be issued to doctors. It is only at the end of Phase 3 trials that the health organizations evaluate the results and issue permission for commercialization. This is vital because the medicine could be prescribed by doctors, and possibly reimbursed by mutuality.
Phase 4 trials are carried out following approval of the product. The companies decide to carry out trials to obtain additional information about the product. Either in respect of other products, or for evaluating other possible therapeutic effects of the product. Possibly also to further document the safety of the product, in populations of patients that better reflect the general population.
What information do I need to obtain before and during the trial?
Information about the new medicine:
- the possible complications
- the possible advantages
- the results of previous trials
Information about the study:
- Study/tests necessary
- The medicine you may receive instead of or in addition to the medicine to be studied
- Whether you are participating in a standard-controlled or placebo-controlled study
- The results during the current study that are important to you, for example serious, undesirable side effects that make it necessary to stop the trial
- The right during the current trial to cease further participation; you don’t have to specify a particular reason
- Information about after care and follow-up checks.
- It is possible you will have to keep a logbook on the medication and complete questionnaires about your health.
Your treating physician is able to tell you everything about the list of topics provided above.
Information about your privacy: information concerning your privacy is provided in the Patient Information Letter that you will receive before the beginning of the study. You can also find more information on our website page about Privacy.