Authored by Fizza Batool*
Abstract
Background: Low prevalence, lack of knowledge about the disease course, and phenotype heterogeneity hamper the development of drugs for rare diseases. Rare disease registries can be helpful by playing a role in understanding the course of the disease, and providing information necessary for clinical trial design, if designed and maintained properly. We describe the potential applications of a rare disease and what type of information should be incorporated to support the design of clinical trials in the process of drug development supported a broad inventory of written record expertise. We evaluated two existing Rare disease in more detail to check the completeness of these Rare disease for trial design.
Results: Before and during the application for regulatory approval rare diseases can improve the efficiency and quality in clinical trial design by informing the sample size calculation and expected disease course. In exceptional circumstances information from rare diseases has been used as historical controls for a one-armed clinical trial, and high-quality rare diseases may be used for registry-based randomized controlled trials. In the post marketing phase of (conditional) drug approval disease-specific rare diseases is likely to provide more relevant information than a productspecific registry.
Conclusion: A rare disease registries can be very helpful to improve the efficiency and quality of clinical trial design in several ways. To alter the pertinence and optimum use of rare un-wellness longitudinal information assortment is indispensable, and specific data collection, prepared for repeated measurement, is needed. The developed listing will facilitate to outline the suitable variables to incorporate. Attention should be paid to the inclusion of patient-relevant outcome measures in the rare diseases from the start. More research and experience is needed on the possibilities and limitations of combining rare diseases information with clinical trial data to maximize the availability of relevant evidence for regulatory decisions in rare diseases.
Abbreviations: Asterix: Advances in tiny Trials style for regulative Innovation and excellence; CDE: Common data elements; CHMP: Committee for medicinal products for human use; DIPG: Diffuse intrinsic pontine glioma; ECFS: European Cystic Fibrosis Society; EPAR: European Public Assessment Report; ERN: European Reference Network; FDA: US Food and Drug Administration; FEV: Forced expiratory volume; ICHOM: International Consortium for Health Outcomes Measurement; RCT: Randomized controlled trial; RDR: Rare disease registry; VOD: Veno-occlusive disease
Introduction
In rare illness the clinical development of effective medicine is difficult because of low prevalence of the disease and sometimes sizable composition heterogeneousness. The small numbers give limited opportunity for confirmatory clinical trials, as it is difficult to recruit enough patients. For much rare illness the disease course is insufficiently identified, resulting in uncertainties with relevancy the optimum run style, including choice of endpoints, for a new drug. Even if the endpoints are clear, there is often insufficient information about their occurrence - in case of binary endpoints-, or distribution - in case the endpoint is a continuous variable. This lack of information, combined with heterogeneity, has consequences for the efficiency of preparing and designing a clinical trial. More specifically, assessing the practicableness of a shot, which is directly connected with robust sample size calculations, becomes a difficult task. When the sample size is calculated based on limited information, this increases the risk of under- or overestimation of the sample size, and possibly a failed trial [1]. Furthermore, such scarcity of information can have considerable impact on the regulatory process and evaluation of the available evidence for the risk/benefit assessment of a new drug, and possible market authorization.
In this respect rare diseases can be an invaluable source of information. With an estimated number of 5000–7000 distinct rare diseases, in Europe over 700 rare diseases are active for a similar number of rare diseases. Rare diseases can give insight in the natural history of the disease and the variability of the patient population. The decision to start rare diseases is often made at a stage when the available information about the rare disease is still scarce, and the development of a treatment might lie secluded within the future. Even in such an early phase, it is important to be aware of and prepared for all possible future functions of the information contained in the rare diseases; the identification of relevant endpoints or the use as a data source for the design of a therapeutic clinical trial are two notable examples. Therefore, rare diseases should be designed in such a way that all relevant information is incorporated and can be used most efficiently. The term ‘registry’ will denote any form of information assortment. However, not all information collections that are given as illness registries are appropriate for run style or as further info for restrictive analysis. Several types of registries can be distinguished, and there is no consensus on the nomenclature and classification.
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