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Being involved as a patient organisations in clinical trials with children

Since a number of years new drugs and therapies need to be tested on children as well as adults to prove there efficacy and safety in children. Since in clinical trials with children the parent of the child plays a major role in the decision making, PatientPartner believes that it is important that parents and carers be represented in all stages of the clinical trial process.
In the PatientPartner project we pay special attention to this topic.
To identify how patient organisations are actively involved in clinical trials with children we asked our respondents:

Is (Has) your patient organisation (been) involved in a clinical trial with children?

6% of all respondents stated that their own child was a research subject in a clinical trial.

In comparison 18% of all respondents confirmed that children within their patient organisation had been involved in a clinical trial as a research subject e.g. Teslac and Profidys. A further eleven clinical trials were described giving information on the condition and the drug that was tested in the trial but without disclosing the trials acronym.

19% of all respondents have been actively involved as an information provider for clinical trials with children. The described involvement ranged from organising meetings for parents and children in which they can discuss the upcoming research with the investigators to developing specific child friendly information for children that take part in clinical trials. Furthermore the respondents describe supporting researchers with information on the prevalence of the condition and writing journalistic articles to inform the public and researchers about the conditions of their children.

6% of all respondents have either given advice or have been active in an advisory committee. The respondents described a number of committees in which they have participated either as advisor or a member e.g.:
- the EMEA Scientific Advice Working Party
- clinical trial steering committee
- a clinical trial governance committee
- a parent’s committee that advises on the protocol before its passage to national authorities
- an International forum for researchers

A further 4% of the responding patient organisations has been involved as a reviewer e.g. by being in a reading committee, reading protocols and patient information leaflets.

12% have been involved as a co-researcher in clinical trials with children. The descriptions that were given on the way they had been involved ranged from  relaying information to the researcher and performing a part of the research.

Furthermore respondents describe be actively involved in translating texts for patients:
“We have edited texts for parents and made them comprehensible for patients, as well as doing so for the presentation of the interim and final results”.

A final 5% of all respondents have been a driving force behind a clinical trial with children.
A number of examples as described by our respondents:
- “We provided financial support to several research organisations”
- “We initiated a procedure that makes it possible for a particular treatment to get recommended to the patient”.
- “We participated in the steering committee”.
- “We secured the funding for a specific clinical trials”.
- “We have set up a foundation who specifically aims at funding research which is focussed on children”.

Finally 18% of all respondents have been involved in clinical trials with children in other ways. The descriptions range from being a member of the EMEA paediatric committee, providing input for the clinical trial portfolio (UK) , being involved in issues affecting trial participation, being involved in quality of life research studies, to communicating closely with researchers of a University.
Numerous remarks were made that for patient organisations concerning conditions that are prevalent in children, the patient organisation tries to be involved on all levels in clinical trial development and performance.

Please click here for the graph on patient involvement in clinical trials on all levels.

Please click here for a list of examples of clinical trials and studies that have been described by our respondents.

Region specific differences in being involved in clinical trials with children
Both on the levels of being involved as a research subject, an information provider and as a reviewer no differences in the frequency of involvement between regions could be found.
As for the level of being involved as an advisor, respondents from Central-Eastern Europe tended to be less frequently involved than the respondents from the other regions.
At the levels of being a co-researcher and a driving force, respondents from North-Western Europe were less frequently involved.

Please click here for the graph comparing the involvement of patient organisations in clinical trials in the three European regions.

Disease-group specific differences in being involved in clinical trials with children
Respondents who represent patient organisations for the rare diseases showed a higher  frequency of being involved on  all levels of active involvement in clinical trials with children in comparison with patient organisations representing the more common conditions.

Please click here for the graph comparing the involvement of patient organisations in clinical trials between patient organisations representing the "common" and "rare" conditions.

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