[Rethinking the marketing strategy of anti-tumor drugs by single-arm trials supported].

Single-arm trial refers to a clinical trial design that does not set up parallel control group, adopts open design, and does not involve randomization and blind method. These features, on the one hand, speed up the process of clinical trials, significantly shorten the time to market and meet the needs of patients with advanced malignancies, but also lead to the uncertainty of single-arm clinical trials themselves. Recently, the US Food and Drug Administration held a meeting of the oncologic drug advisory committee to discuss six tumor indications that have been accelerated approved, which once again triggered the discussion of single-arm trials. The basis of accelerated approval by single-arm trial is actually a compromise on the level of evidence-based medical evidence requirements after assessing the benefit risk. Therefore, the sponsor should strictly grasp the applicable conditions of single-arm trial in anti-tumor drugs and conduct single-arm trial scientifically. Post-marketing clinical trial should be implement as early as possible to ensure the benefit of patients. Based on the characteristics of single-arm trial, combined with two guidance relevant to single-arm trial issued by National Medical Products Administration recently, this article is supposed to propose and summarize the strategy of single-arm trial supporting the marketing of anti-tumor drugs.

[Considerations for anti-cancer drug application by single arm trials].

Single arm trial (SAT) was widely used for new drug application (NDA) of novel anti-cancer drugs in recent years. The listing time was greatly shortened by SAT while comparing with randomized controlled trials (RCT). Thus, the companies intended to get NDA through SAT. To encourage innovation and accelerate the developments of anti-cancer agents, we summarize the background and key issues of SAT, discuss the conditions of accepting SAT for NDA, and systematically elaborate the design and principles of SAT in this review.

Presenile (early ageing) changes in tissues of Kaschin-Beck disease and its pathogenetic significance.

The selenium level and activity of glutathione peroxidase in blood of children living in Kaschin-Beck disease (KBD) endemic areas were lower than that in nonendemic areas. KBD children were deficient in selenium, their lipid components, structure and function of the red cell membrane and cartilage tissue were abnormal. That is, the phospholipid (PL) content in the tissues of the patient was less than that of the controls in endemic and non-endemic areas. Especially as the phosphatidylcholine (PC) content decreased significantly, but sphingomyelin (SM) increased, the molar ratio of SM/PC and cholesterol (Ch)/PL increased. Increase of acanthocyte content was seen under the electron microscope and the fragility of erythrocytes was also increased. It indicated that there were membrane defects and membrane damage in KBD. At the same time, the sulfation extent of mucopolysaccharides in cartilage of patients was lower, and the collagen content was higher than that of controls. The presenile changes in lipid composition, structure and function of biomembranes and cartilage metabolism of KBD are very significant in studies on the aetiological pathogenesis of KBD and other ageing diseases.