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About Orphan Drugs

The people participating in a trial.

They are ill or healthy volunteers who are willing to participate in a study under the conditions described by law. The laboratory offers a small compensation for the inconveniences incurred. The subject gives his/her written consent, after receiving oral and written information on the protocol. (S)he is expected to respect the rules. However, the subject can leave the trial without the need for any special procedure, and this should not be detrimental to the relationship with his/her physician.

The accepted basis for the conduct of clinical trials in humans is founded on the protection of human rights and the dignity of the human being with regard to the application of biology and medicine, as, for example reflected in the 1996 version of the Helsinki declaration. The subject's protection in a clinical trial is safeguarded through the risk assessment based on the results of toxicological experiments prior to any clinical trial, screening by ethics committees and Member States' competent authorities, as well as the rules on the protection of personal data. See Directive 2001/20/EC of the European Parliament and of the Council of 4 April 2001 concerning the drawing together of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use.

What type of protection for these people ?

Any clinical trial conducted in an EU member state must have received a favorable opinion from a consultative committee composed of members of the medical, legal and scientific professions qualified to give an opinion on the legitimacy of the clinical trial and the safety of the poeple taking part in it. (D2001/20/CE).

The ethics committee gives its opinion by taking, notably, the following elements into account :

The ethics committee has a maximum of 60 days from the date of receipt of a valid application to give its reasoned opinion to the applicant and the competent authority in the Member State concerned.

In the United States, a federal law provides that any clinical trial must have been first approved by an 'Institutional Review Board' or IRB before it can be started and it is followed up periodically by this IRB. The purpose of the IRBs that work together with the relevant administrative authority (FDA), is to ensure that the risks linked to research are insignificant compared to the benefits expected.

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Why participate in a clinical trial and who can participate ?

The decision to take part in a clinical trial means that you benefit from a new treatment that may be more effective than what is already available.

It also means that you contribute to research for future generations and to a better understanding of how the treatment works.

Anybody can volunteer to participate in a clinical trial. However, an accurate description of the persons that may participate in the trial is provided for each clinical trial: age, sex, type of disease, medical or surgical family history, current condition, current drug treatments... Some clinical trials are performed with healthy volunteers (phase I trials), while others only involve patients for which the drug tested may be a treatment. The factors allowing a subject to participate in a clinical trial, because (s)he corresponds to the target population described in the protocol, are called inclusion criteria. The factors that prevent the subject from participating are called exclusion criteria. These criteria are not used to reject people personally. Instead, they help to ensure that researchers will be able to answer the questions they plan to study by identifying appropriate participants.

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What is a sponsor ?

The sponsor is a person or legal entity whoinitiates biomedical research. In order to perform it, (s)he chooses one or several investigators, who are physician with appropriate experience and who are in charge of supervising and conducting the clinical trial. The physician promise to immediately report all serious adverse reactions to the sponsor.

Whith respect to law, the sponsor is responsible for the research and is therefore subject to many legal obligations. He ensures that the material and technical conditions adapted to the trial and compatible with safety requirements are implemented. The sponsor compensated those participating in the trial in the case of attributable injury a third-party insurance is taken out before the start of the trial.
The sponsor is also responsible before the administrative authorities for the smooth running of the clinical trial and the diffusion of any information about the safety of people participating in the trial.

Who can sponsor a clinical trial ?

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What happens during a clinical trial ?

It depends on the protocol of the trial. The medical team will monitor the health of the participants at the beginning of the trial, and give instructions for following the protocol. They will then monitor the state of health by performing the necessary biological tests, and will keep in touch with the participants after the end of the trial.

Some clinical trials require more biological tests and medical consultations than a disease usually requires. This is because researchers need to collect a maximum of data to interpret.

Participation in a clinical trial will only be useful to the community if the participant abides by the protocol and carefully follows the 'instructions of the medical team; the role of physicians consists not only in ensuring the smooth runningt of the clinical trial but also in answering the questions of the participants.

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What are the adverse reactions and the risks of a clinical trial ?

Adverse reactions are side effects that appear during or after the drug treatment. They include headache, nausea, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

The risks of a clinical trial are linked to :

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What do you need to know before participating in a clinical trial ?

Questions to ask your physician.

It is recommended that you have as much information as possible about the clinical trial before giving your informed consent.

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The European Directive on clinical trials

The main aim of the Directive is to simplify and harmonise the administrative provisions governing clinical trials. It aims to provide an environment for conducting clinical research that protects participants without hampering the discovery of new essential medicines. It applies equally to all commercial and non-commercial trials and is applicable from the 1st May 2004.

One of the requirements of the Directive is that all drug trials are to be conducted according to Good Clinical Practice (GCP).

The Directive also states that all drug trials must have a nominated sponsor/co-sponsors. The sponsor's responsibilities fall into three areas :

The UK Department of Health has issued guidance for sponsorship responsibilities for non-commercial trials.

For further information :

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