House Tiffany, O'Donnell Kevin, Saich Raymond, Di Pietro Fabio, Broekgaarden Ria, Muir Allan, Schaller Thomas
Acid Maltase Deficiency Association (AMDA), San Antonio, TX, USA.
International Pompe Association, Baarn, The Netherlands.
Ann Transl Med. 2019 Jul;7(13):293. doi: 10.21037/atm.2019.04.28.
The Pompe model is the term used by the Pompe community to describe the relationship that exists between the patient community, the medical/scientific community, and industry. The development of the Pompe model represented a new paradigm for the involvement of patients in new treatments-and also for scientists and pharmaceutical companies. It saw patients developing a sense of agency, of involvement in the process of treatment development rather than powerless recipients or (if lucky) occasional spectators. At the same time, as described below, it benefited the other partners in the process with the result that the different components of the model added up to more than the sum of their parts. However, in order for this to happen, each part had to undergo a transformation in mindset. The development of enzyme replacement therapy (ERT) for Pompe disease represented a unique set of circumstances and individuals that helped to bring about this change and, in doing so, created a model that has had far wider applications.
庞贝模型是庞贝病患者群体用来描述患者群体、医学/科学界和产业界之间关系的术语。庞贝模型的发展代表了患者参与新治疗方法的一种新范式，对科学家和制药公司来说也是如此。它让患者产生了一种能动感，参与到治疗方法研发过程中，而不再是无力的接受者，或者（如果幸运的话）只是偶尔的旁观者。同时，如下所述，它也让这一过程中的其他合作伙伴受益，结果是该模型的不同组成部分产生了大于其各部分之和的效果。然而，为了实现这一点，每个部分都必须经历思维模式的转变。针对庞贝病的酶替代疗法（ERT）的发展代表了一系列独特的情况和个体，它们促成了这种变化，并且在此过程中创建了一个具有更广泛应用的模型。
