Cohen Perry D, Herman Linda, Jedlinski Sheryl, Willocks Peggy, Wittekind Paula
Parkinson Pipeline Project, Washington, DC 20015, USA.
Neurotherapeutics. 2007 Jul;4(3):537-44. doi: 10.1016/j.nurt.2007.04.008.
A patient-centered paradigm for clinical research and medical care is presented as a solution to the problem of declining innovation and increasing costs and development time in the pipeline for new therapies. Fundamental differences in values and motivations among scientists, clinicians, industry sponsor, and patients in neurotherapeutics provide a framework for analysis of ethical conflicts and the loss of public confidence in medical research. Parkinson advocates' views on clinical trial participation, perceived risks and benefits, placebo controls, and sham surgery are presented. These views reflect the sense of urgency and the unique perspective that comes from living with this progressive, debilitating condition full time. A patient-centered paradigm that includes authentic voices of patients as collaborators at every stage of development will help to resolve conflicts, build trust, recruit trial participants, and accelerate new therapies. Key elements are adaptive clinical trial methods and the development of information technology for the assessment of outcomes and surveillance of safety over the life cycle of a medical product. Supported by the Parkinson's Disease Foundation, the Parkinson Pipeline Project is a grassroots group of Parkinson's patients whose goal is to represent an authentic voice for patients in the treatment development process. This group promotes education and communication between members of the Parkinson's community and active stakeholders in medical research, industry, and regulatory agencies. Its members are an example of a new breed of knowledgeable consumers, armed with first-hand access to research findings and reinforced by on-line connections to like-minded peers throughout the world.
本文提出了一种以患者为中心的临床研究和医疗范式，作为解决新疗法创新下降、成本增加以及研发时间延长问题的方案。神经治疗领域的科学家、临床医生、行业赞助商和患者在价值观和动机上的根本差异，为分析伦理冲突以及公众对医学研究信心丧失提供了一个框架。文中介绍了帕金森病倡导者对于临床试验参与、感知到的风险和益处、安慰剂对照以及假手术的观点。这些观点反映了紧迫感以及因长期患有这种渐进性、使人衰弱的疾病而产生的独特视角。一种在开发的每个阶段都将患者的真实声音纳入其中作为合作者的以患者为中心的范式，将有助于解决冲突、建立信任、招募试验参与者并加速新疗法的研发。关键要素包括适应性临床试验方法以及开发用于评估医疗产品生命周期内结果和监测安全性的信息技术。帕金森管道项目由帕金森病基金会支持，是一个由帕金森病患者组成的草根团体，其目标是在治疗开发过程中代表患者发出真实的声音。该团体促进帕金森病群体成员与医学研究、行业和监管机构中的活跃利益相关者之间的教育和交流。其成员是新一代知识渊博的消费者的典范，他们能够直接获取研究结果，并通过与世界各地志同道合的同行建立在线联系而得到强化。
