Citations 2
Placebo-controlled study in neuromyelitis optica—Ethical and design considerations
Cree, Bruce , Bennett, Jeffrey L. , Sheehan, Mark , Hartung, Hans-Peter, Aktaş, Orhan , Kim, Ho Jin , Paul, Friedemann , Pittock, Sean J, Weinshenker, Brian G. , Wingerchuk, Dean M. , Cutter, Gary , Patra, Kaushik, Flor, Armando , Barron, Gerard, Madani, Soraya, Ratchford, John N. , Katz, Eliezer , Pittock, Sean J. , Wingerchuk, Dean, Pittock, S J , Cohen, Jeffrey Allen , Fujihara, Kazuo , Bennett, Jeffrey L , Weinshenker, Brian G , Cree, Bruce Ac , Paul, Friedemann
Multiple Sclerosis Journal | December 14, 2015 | Volume: 22 | Issue: 7 | Pages: 862-872
Background: To date, no treatment for neuromyelitis optica (NMO) has been granted regulatory approval, and no controlled clinical studies have been reported. Objective: To design a placebo-controlled study in NMO that appropriately balances patient safety and clinical–scientific integrity. Methods: We assessed the “standard of care” for NMO to establish the ethical framework for a placebo-controlled trial. We implemented measures that balance the need for scientific robustness while mitigating the risks associated with a placebo-controlled study. The medical or scientific community, patient organizations, and regulatory authorities were engaged early in discussions on this placebo-controlled study, and their input contributed to the final study design. Results: The N-MOmentum study (NCT02200770) is a clinical trial that randomizes NMO patients to receive MEDI-551, a monoclonal antibody that depletes CD19+ B-cells, or placebo. The study design has received regulatory, ethical, clinical, and patient approval in over 100 clinical sites in more than 20 countries worldwide. Conclusion: The approach we took in the design of the N-MOmentum trial might serve as a roadmap for other rare severe diseases when there is no proven therapy and no established clinical development path.
Position statement on ethics, equipoise and research on charged particle radiation therapy
Sheehan, Mark , Peach, Ken , Binik, Ariella , Wilson, Puthenparampil , Lodge, Mark , Kehoe, Sean , Brada, Michael, Clarke, Steve , Crellin, A., Dunn, Michael , Fossati, Piero , Harris, Steve , Hocken, Michael, Hope, Tony, Ives, Jonathan , Kamada, Tadashi , London, Alex John , Miller, Robert C. , Pijls-Johannesma, Madelon, Short, Susan , Skene, Loane, Tsujii, Hirohiko, Tuan, Jeffrey , Weijer, Charles , Timlin, Claire L. , Burnet, N.G. , Parker, Michael James , Savulescu, Julian , Short, Susan C
Journal of Medical Ethics | September 18, 2013 | Volume: 40 | Issue: 8 | Pages: 572-575
The use of charged-particle radiation therapy (CPRT) is an increasingly important development in the treatment of cancer. One of the most pressing controversies about the use of this technology is whether randomised controlled trials are required before this form of treatment can be considered to be the treatment of choice for a wide range of indications. Equipoise is the key ethical concept in determining which research studies are justified. However, there is a good deal of disagreement about how this concept is best understood and applied in the specific case of CPRT. This report is a position statement on these controversies that arises out of a workshop held at Wolfson College, Oxford in August 2011. The workshop brought together international leaders in the relevant fields (radiation oncology, medical physics, radiobiology, research ethics and methodology), including proponents on both sides of the debate, in order to make significant progress on the ethical issues associated with CPRT research. This position statement provides an ethical platform for future research and should enable further work to be done in developing international coordinated programmes of research.