Guideline Recommendation for Endpoints Used in Clinical Trials for Functional Dyspepsia / 대한소화기학회지

Functional dyspepsia is a disease, in which there is no organic lesion but chronic and repetitive postprandial fullness, early satiation, epigastric pain, and epigastric burning. Functional dyspepsia is not life-threatening but its symptoms are relapsing and remitting and persist over a lifetime, limiting the social life and reducing the quality of life. Therefore, the treatment for acute relapsing period may help improve the short-term symptoms. Continuous medication may be needed to improve the long-term symptoms. Research designs to demonstrate the short-term efficacy of therapeutic agents may differ from clinical trials to demonstrate long-term efficacy. There are many difficulties in clinical trial design, implementation, and screening because there are no international standards of clinical trials for functional dyspepsia. The purpose of this guideline recommendation is to develop a standard for clinical trials, such as clinical trial subjects and evaluation methods, in the development of therapeutic agents for functional dyspepsia. The ultimate aim is to enhance the safety and efficacy of therapeutic agents for functional dyspepsia and promote the development of new therapeutic agents.

Present Status and Problems on Molecular Targeted Therapy of Cancer / Journal of the Korean Cancer Association, 대한암학회지

Numerous clinical trials of molecular targeted drugs for cancer have been conducted, with remarkable results for certain drugs and accumulation of "negative data" causing a hitch in the development plan for some other compounds. Five recent issues and problems of molecular targeted therapies were discussed critically. Drug discovery and effects against driver mutations (activating mutations) and problems: possibility for circumventing inherent and acquired resistance with the aim of achieving radical cure. Synthetic lethality: reasonable patient selection in individualized treatment strategy. Response rate and progression-free survival improvement with or without overall survival benefit and enhancement of toxicity in bevacizumab therapy: best endpoints for the evaluation of effect of antiangiogenic therapy. Negative data on small-molecule targeted therapy, primarily vascular endothelial growth factor tyrosine kinase inhibitors: loose GO or NO-GO decision criteria for further development of new compounds in early clinical trials. Effect of immunotherapy: difficulty to verify by proof of principle study. We are faced to many questions for the development of efficient personalized therapy. Accumulation of scientific global preclinical and clinical evidences is essential to use these new therapeutic modalities for the improvement of oncologic health care.

Endpoints in advanced breast cancer: methodological aspects & clinical implications.

Breast cancer is an increasingly important public health problem in developing countries, with disproportionately high mortality. The increasing availability of active agents against advanced breast cancer makes the development of novel treatments and their choice in clinical practice progressively more complex. Furthermore, there is often a tension between the adequacy of endpoints used in clinical trials and the clinician's aim of improving survival and quality of life, the two most important therapeutic goals in advanced breast cancer. However, overall survival (OS) is no longer a suitable indicator of treatment efficacy within clinical trials in settings for which effective subsequent-line therapy exists. Conversely, progression-free survival (PFS) currently represents the most sensitive parameter to assess the efficacy of a new drug or combination in such settings. When coupled with a favourable toxicity profile and cost, the demonstration of an improved PFS may be enough evidence for the superiority of a treatment. Despite arguments favouring the use of PFS as a primary endpoint in clinical trials, clinicians who need to make sense of the available literature may be reluctant to use PFS as an indicator of clinical benefit when deciding among different therapeutic strategies for their patients. This choice is further complicated if one fails to distinguish between the use of an efficacy parameter as an indicator of therapeutic objective for individual patients and as a clinical trial endpoint. This brief review aims at helping clinicians in their daily need to interpret the literature and make informed treatment choices for patients with advanced breast cancer.