Resumen de The self-experimentation in rare diseases: analysis of the ethic profiles, and guidelines for a possible governance (public management)
Rare diseases gather up a large number of different pathologies, all very different among them but similar, for the low statistical attendance of population (penetration). Therefore there are a very low number of patients sick of the same pathology and their presence in different geographical areas makes objectively difficult to recruit patients in order to study their disease and coordinate and organize the research of the pathology. As a consequence there is a much reduced possibility to carry on clinical studies and genetic researches. In this framework, in order to face the dramatic necessity of sociological and sanitary assistance, treatment and rehabilitation of the (rare disease) patients, family’s members generally organize Rare Diseases Patient Organizations. At the moment these organizations develop an important activity with regard to organization, proposals, direct participation and publication of the scientific research results. These organizations also play an important part in supporting the organization and the performing of clinical trials. They play in total autonomy and are independent from any ethical and scientific oversight by the sanitary authorities and ethical committees. This extraordinary event known as “ResearchLed by Participants”, has been performed thanks to the large contact possibilities and organization offered by the web which made possible the creation of virtual communities of patients, of blog and web site contact for information and communication and, more recently, for collecting statistical data, publication of the patients research results and the recruitment of patients in order to manage the studies following the rules and models proposed, for example, by the web site “PatientsLikeMe”. We must however put in evidence the important ethical issues involved in the research led by the participants. These aspects concern, obviously, to either the scientific validity of these studies or the protection of the subjects who accept to be involved in the experimentation. It is a completely new way of experimentation that requires the capacity to propose and follow a governance that must be accepted and shared by citizens/patients, “third sector”, research workers, institutions and ethical committees able to exploit the potential advantageous knowledge and create ethical rules inspired to those new situations. In this article it will be proposed a theoretical model of governance of this phenomenon and also it will be outlined ethical rules for operative plans.
