Latest ArticlesFocusing on the specialty of locally applied locally acting drugs, we discuss and explain the special considerations in its design and evaluation of clinical trials.
The technical guidelines on the research and development of locally applied locally acting drugs issued by domestic and foreign regulatory agencies and related literature were extensively investigated. The relevant drug research and development situation and review practice were combed. Combined with case analysis and expert discussion opinions, the technical standards for clinical research and development of local drugs under different registration types were discussed.
The sponsor should formulate the corresponding research and development strategy based on the clinical medication needs and developmental background. For the innovative drugs, in addition to following the general consideration of clinical trials of innovative drugs, specific studies should also be carried out at the same time considering the characteristics of drug dosage forms, the routes of administration, and application sites, etc. Meanwhile, due to the technical and ethical difficulties of sampling in local pharmacokinetics and pharmacodynamics research, pharmaceutical quality research and comprehensive non-clinical research are particularly important. Some necessary exploratory and confirmatory clinical trials should be carried out combining the clinical data of the original active ingredient to prove the clinical advantages in safety and effectiveness etc. of the improved drug. For generic drugs that is locally administered and locally acting, beside pharmacy and non-clinical comparative studies with the original reference preparations, the sponsors may also need to carry out necessary human pharmacokinetics, pharmacodynamics, and even clinical equivalence trials to prove the consistency of quality and efficacy of generic drugs and reference preparations.
To explore the consideration on the key techniques of clinical trial design of drugs for the treatment of atopic dermatitis.
By summarizing the clinical research status of drugs for the treatment of atopic dermatitis, and investigating the progress of technical guidelines for the drugs in China and abroad in recent years, relevant technical considerations are proposed based on the China's experience in Drug R&D and the process of review.
The design of whole clinical trial of drugs treating atopic dermatitis should be based on the characteristics of the disease and investigational drug, the purpose of the study. Clinical trials for pediatric patients should be conducted as early as possible under the premise of prior research evidence and ensuring the safety of subjects. The appropriate study population should be selected, and a series of key elements, such as control drugs, efficacy and safety indicators, study period, etc., should be reasonably set up in the clinical trial design, in order to assist the scientific research and the development of the drugs. The positioning of the study population should be based on the drug action mechanism and drug effect intensity, and the patients with corresponding severity should be selected. The efficacy of the study should be comprehensively evaluated to assess the improvement of skin lesions, symptoms, quality of life, etc., and attention should be paid to the efficacy and safety of the study when used in pediatric patients as well as in long-term treatment.
To discuss the scientific considerations on the design of drug therapeutic efficacy indicators for the treatment of atopic dermatitis.
By investigating the progress of global clinical trials of new drugs, relevant guidelines and the literatures, the scientific design of efficacy indicators in clinical trials of atopic dermatitis was discussed.
Generally, the design of drug efficacy indicators for the treatment of atopic dermatitis should comprehensively evaluate the symptoms, signs, quality of life and long-term control of the disease. The main efficacy indicators should reflect the clinical benefits and remain consistent with the treatment objectives. The design, validation and application of patient-centered assessment scale are crucial, and are still challenging in the scientific evaluation of drug efficacy.
To explore the key technical elements of clinical trial design for the treatment of plaque psoriasis vulgaris.
By searching both relevant domestic and international literature, and combined with research and evaluation practices in China, scientific considerations on innovative drug clinical trial design for the treatment of psoriasis are summarized and proposed.
The overall design of clinical trials for the treatment of psoriasis vulgaris should be based on the characteristics of the disease's pathophysiology and drug action mechanisms, with a reasonable selection of control and population, as well as the design of trial duration, efficacy, and safety indicators. To comprehensively evaluate the effectiveness and safety of drugs, attention should be paid to the improvement of patients' quality of life, relapse or rebound after drug withdrawal, and long-term medication while evaluating the improvement of psoriasis symptoms and signs after drug treatment.
Taking the development of dermatology drugs as an example, proposals are provided after analysis by summarizing the common clinical questions about drugs development encountered in communication, in order to provide reference for the same problems and improve the efficiency and quality in communication system.
The clinical improvement questions were collected and classified regarding dermatology drugs' communication applications in the communication subsystem of the Center for Drug Evaluation of National Medical Products Administration drug technical review system. The common questions were analyzed and regulatory considerations for improvement were elaborated.
Efficient communication system can promote the interaction between research and regulation, solve more individual problems, satisfy the needs in drug developments; however, developers and regulators need to work together to improve the quality and efficiency of communication.
Model-informed drug development (MIDD) is a major shift of new mode of drug development based on the establishment of PK-PD model using blood concentration- (i.e., exposure, PK) and efficacy/safety (i.e., response, PD)-corresponding data, in order to analyze exposure-response relationship, replace dose-response relationship that is difficult to obtain, and establish the chain of evidence on drug safety and efficacy. The above model can integrate data from in vitro and in vivo, animals and human beings, adults and children, literature and trials, original and new dosage forms, domestic and foreign trials, and other sources to solve a wide range of key clinical problems. It is particularly important to transform the exploration paradigm of new drug development into a model verification mode. Therefore, MIDD is not only related to the change of technical means, but also the integration of multidisciplinary research, as well as a change of research and development strategy and scientific regulatory. It greatly improves the efficiency and success rate of new drug research and development. Based on the summary of MIDD success cases, this paper tries to clarify the basic principle, concept and method, implementation path, and takes anti-psoriasis monoantibody as an example to establish a complete MIDD working mode.
Autoimmune skin diseases are a kind of diseases caused by the immune response to self-antigens, resulting in damages to skin with or without to other tissues and organs. The traditional immunosuppressants exert their curative effect, but accompanied by a wide range of adverse reactions. In-depth understanding of pathogenesis facilitated the development of various cytokine-based therapies or drugs targeting the signaling pathways, which has significantly improved the patient's condition. However, these therapies often require long-term maintenance treatment, and the targeting pathways of many autoimmune skin diseases are still not quite clear. Therefore, emerging therapeutic strategies and innovative drugs are still in need to balance the homeostasis of the immune system and induce the long-term remission of the disease, providing new hope for the treatment of autoimmune skin diseases. This review discusses the development of drugs treating autoimmune skin diseases according to different mechanisms of action and targets.
Atopic dermatitis is a chronic inflammatory disease of skin that is commonly seen and easy to relapse. Treatments nowadays still have many drawbacks and adverse effects, thus cannot meet the needs of clinicians and patients. In recent years, our expanding knowledge of the complex pathomechanisms of atopic dermatitis has given rise to new interventions of targeted therapy, which will help clinicians better treat and manage the atopic dermatitis. This review summarizes the advances in new medications for pediatric atopic dermatitis.
Psoriasis is a chronic immune-mediated inflammatory skin disease. A growing understanding of the mechanism has led to the advent of biological agents that specifically target the cytokines which are involved in the pathogenesis. Because of the significant efficacy with high safety shown in the treatment of psoriasis, biologic agents have becoming more and more usual in clinical practice. Rapid progress in biotechnology allows the development of an increasing number of novel biologic agents, such as biologics targeting existing and promising inflammatory mediators, bispecific agents targeting multiple different antigens and agents including nanobodies and peptides. This review summarizes the current application of biologic agents in clinical practice, new drugs that are under study, potential targets and novel technology of biologics.
Vitiligo is a kind of refractory depigmentation skin disease, which seriously affects the appearance and psychology of patients. At present, the pathogenesis of vitiligo includes heredity, oxidative stress, autoimmunity and environmental factors. The treatment includes systematic application of glucocorticoid, local application of glucocorticoid or calcineurin inhibitor, phototherapy and melanocyte transplantation. In this article, we reviewed the commonly used therapeutic drugs for vitiligo in the past and summarized the new progress of therapeutic drugs for the treatment of vitiligo. The emergence of new small-molecule targeted drugs, such as Janus kinase inhibitor baricitinib, has brought hope for vitiligo.
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