Riva Luciana, Campanozzi Laura, Vitali Massimiliano, Ricci Giovanna, Tambone Vittoradolfo
Unità di Bioetica, Istituto Superiore di Sanità, Rome, Italy.
Istituto di Filosofia dell'Agire Scientifico e Tecnologico - FAST, Università Campus Bio-Medico di Roma, Rome, Italy.
Ann Ist Super Sanita. 2019 Apr-Jun;55(2):179-185. doi: 10.4415/ANN_19_02_10.
Nowadays one of the most critical aspects of innovative cell-based therapies is the unregulated industry, as it is becoming a competitor of the regulated system. Many private clinics, worldwide, advertise and offer cell-based interventions treatments directly to the consumer and this poses a risk to both vulnerable patients and health systems. Several countries have implemented Compassionate Use Programmes (CUP) that provide patients with medicines that have not yet completed the approval pathway, in the event that no reasonable alternative exists. Recently, in the public discourse, compassionate use has been increasingly associated with a patient's right to try. Thus, the aim of this study was to assess public knowledge of the clinical trials process with specific reference to innovative stem cell treatments, and trust in the institutions responsible for regulatory activities. We also asked people about their "right" to use unregulated therapies.
We developed an ad hoc questionnaire on three main areas of concern and administered it to 300 people in the patient waiting room at an Italian university hospital.
Our findings suggest that people have a good knowledge of the clinical trials process and trust in healthcare institutions. Nonetheless, one person in two believes it is a right to use unregulated therapies.
We stress the need, in the age of cellular therapies, for a commitment to support vulnerable patients and to strengthen awareness among the public about the substantial boundary that differentiates experimental therapies from unproven therapies. There should not be a "right to try" something that is unsafe but rather approved treatments and in line with good clinical practice. The trend, which emerged on this issue from our study, is quite different, confirming the urgent need to improve health information so that it is as complete as possible.
如今，创新型细胞疗法最关键的问题之一是行业缺乏监管，因为它正成为受监管体系的竞争对手。全球许多私人诊所直接向消费者宣传并提供基于细胞的干预治疗，这对脆弱的患者和卫生系统都构成了风险。一些国家实施了同情用药计划（CUP），在没有合理替代方案的情况下，为患者提供尚未完成审批流程的药物。最近，在公众讨论中，同情用药越来越与患者的尝试权联系在一起。因此，本研究的目的是评估公众对临床试验过程的了解，特别是关于创新干细胞治疗的了解，以及对负责监管活动的机构的信任。我们还询问了人们使用未经监管疗法的“权利”。
我们针对三个主要关注领域设计了一份专门的问卷，并在一家意大利大学医院的患者候诊室对300人进行了调查。
我们的研究结果表明，人们对临床试验过程有较好的了解，并信任医疗机构。尽管如此，仍有二分之一的人认为使用未经监管的疗法是一种权利。
我们强调，在细胞疗法时代，需要致力于支持脆弱的患者，并加强公众对区分实验性疗法和未经证实疗法的实质性界限的认识。不应该有尝试不安全事物的“权利”，而应该是经过批准且符合良好临床实践的治疗方法。我们研究中在这个问题上出现的趋势截然不同，这证实了迫切需要改善健康信息，使其尽可能完整。
