scholarly journals Trends of Clinical Trials for Drug Development in Rare Diseases

2018 ◽  
Vol 13 (3) ◽  
pp. 199-208
Author(s):  
Ryuichi Sakate ◽  
Akiko Fukagawa ◽  
Yuri Takagaki ◽  
Hanayuki Okura ◽  
Akifumi Matsuyama
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Diseases

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

scholarly journals Current Status and Trend of Clinical Development of Orphan Drugs in China

2021 ◽  
Author(s):  
Ziling Xiang ◽  
Wengao Jiang ◽  
Bo Yan ◽  
Junhao Jiang ◽  
Hang Zheng
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
Clinical Development ◽  
Current Status ◽  
Policy Makers ◽  
Critical Issues ◽  
Low Coverage ◽  
Orphan Drug Development

Abstract Background:Rare diseases have been increasingly recognized as medical and healthy burden worldwide, a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great interest for both the regulatory agency and pharmaceutical companies to keep tract on the clinical orphan drug development in China.Objective and Method:This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Platform for Drug Clinical Trials and Information Registration（http://www.chinadrugtrials.org.cn）of China Food and Drug Administration, dating from 2013 to March 8, 2021.Results:A total of 246 clinical trials for orphan drugs are extracted from the Platform, covering 22 rare diseases and 90 drugs. Among the 22 rare diseases, 3 (14 %) have more than 50 trials each , 17 (77%) had less than10 trials, and 10 (46%) only with one trial. Among 90 orphan drugs, 60 (67%) were chemical drugs, and 30 (33 %) were biological products. In addition, international multi-center trials accounts for nearly 10% of the total trials. The number of the trials with the Data Monitoring Security Committee (DMC) is 25 (10%) and the number of the trials with the trial injury insurance for subjects is 154 (63%). Furthermore, more than half of the total trials are carried in east (333, 30%) and north China (298, 27%), whereas a small portion are in the northwest (62, 6%) and northeast china (45, 4%).Conclusions:The clinical development of orphan drugs for rare diseases in China has made some progress in the passing decades. However, a couple of critical issues still need to be addressed, such as unmet needs for some rare diseases, low coverage of insurance and DMC, and uneven distribution of medical resources for clinical researches. Recommendations are put forward accordingly, which can provide improvement goals for policy makers and stakeholders involved in drug development for rare diseases.

scholarly journals Letter from the Editors

2020 ◽  
Vol 2 ◽  
pp. 1
Author(s):  
Editorial Office
Keyword(s):  
Clinical Trials ◽  
Medical Journal ◽  
Open Access ◽  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Case Reports ◽  
Orphan Drugs ◽  
Original Research ◽  
Orphan Drug Development

Last year we successfully introduced a new journal: The Journal of Rare Diseases and Orphan Drugs (JRDOD) is a peer-reviewed open-access medical journal that publishes original research, reviews, case reports, and letters covering a broad field of its specialty. We intend to publish articles stimulating to read, educate, and inform readers with the most up-to-date research in genetics, rare diseases, and new orphan drug development in different stages of clinical trials. Journal topics are centered on patients living with undiagnosed rare diseases, the importance of a diagnosis, individual approaches to treatments. We hope that this journal will increase awareness of many difficult to diagnosed and treat medical conditions.

Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists

2020 ◽  
pp. 089719002093097
Author(s):  
Kanya K. Shah ◽  
Stephen Kogut ◽  
Angela Slitt
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
The United States ◽  
Priority Review ◽  
Pharmaceutical Companies ◽  
Federal Programs ◽  
Financial Barriers ◽  
Safety And Efficacy

A rare disease, or orphan disease, in the United States is a condition with a national prevalence of fewer than 200,000 diagnoses. As therapies for rare diseases are developed and brought to market, pharmacists should understand the challenges of drug development for rare diseases and aid in educating patients about the approval process for rare disease therapies. Developing drugs for treating rare diseases presents unique challenges in proving the drug’s safety and efficacy with adequate study design, power, and validity. Results of the clinical trials for rare diseases may be weakened by small patient populations, limited disease information, and difficulty defining end points and biomarkers. In addition to investigational barriers, pharmaceutical companies face financial barriers in justifying the investment of bringing a rare disease therapy to market. Federal programs, such as the Orphan Drug Act of 1983, expedited review, the Rare Pediatric Disease Priority Review Vouchers (RPD PRV) program, and the 21st Century Cures Act, give pharmaceutical companies motivation to develop therapies for rare diseases. The objective of this article is to provide pharmacists with an understanding of the challenges in designing clinical trials for drugs for rare diseases and discuss federal programs that address efforts to develop safe and efficacious drugs for rare diseases.

Clinical Drug Development for the Treatment of Pulmonary Arterial Hypertension: Collaboration With the Pharmaceutical Industry and Regulatory Agencies

2010 ◽  
Vol 9 (4) ◽  
pp. 214-219
Author(s):  
Robyn J. Barst
Keyword(s):  
Clinical Trials ◽  
Pulmonary Arterial Hypertension ◽  
Drug Development ◽  
Phase 1 ◽  
Preclinical Studies ◽  
Phase 2 ◽  
Phase 3 ◽  
Preclinical Testing ◽  
New Drug ◽  
Pulmonary Arterial

Drug development is the entire process of introducing a new drug to the market. It involves drug discovery, screening, preclinical testing, an Investigational New Drug (IND) application in the US or a Clinical Trial Application (CTA) in the EU, phase 1–3 clinical trials, a New Drug Application (NDA), Food and Drug Administration (FDA) review and approval, and postapproval studies required for continuing safety evaluation. Preclinical testing assesses safety and biologic activity, phase 1 determines safety and dosage, phase 2 evaluates efficacy and side effects, and phase 3 confirms efficacy and monitors adverse effects in a larger number of patients. Postapproval studies provide additional postmarketing data. On average, it takes 15 years from preclinical studies to regulatory approval by the FDA: about 3.5–6.5 years for preclinical, 1–1.5 years for phase 1, 2 years for phase 2, 3–3.5 years for phase 3, and 1.5–2.5 years for filing the NDA and completing the FDA review process. Of approximately 5000 compounds evaluated in preclinical studies, about 5 compounds enter clinical trials, and 1 compound is approved (Tufts Center for the Study of Drug Development, 2011). Most drug development programs include approximately 35–40 phase 1 studies, 15 phase 2 studies, and 3–5 pivotal trials with more than 5000 patients enrolled. Thus, to produce safe and effective drugs in a regulated environment is a highly complex process. Against this backdrop, what is the best way to develop drugs for pulmonary arterial hypertension (PAH), an orphan disease often rapidly fatal within several years of diagnosis and in which spontaneous regression does not occur?

Intelligent Drug Development

2014 ◽  
Author(s):  
Michael Tansey
Keyword(s):  
Clinical Trials ◽  
Cost Effectiveness ◽  
Drug Development ◽  
Clinical Research ◽  
Development Process ◽  
Drug Development Process ◽  
Individual Trial ◽  
Specific Technology

Clinical research is heavily regulated and involves coordination of numerous pharmaceutical-related disciplines. Each individual trial involves contractual, regulatory, and ethics approval at each site and in each country. Clinical trials have become so complex and government requirements so stringent that researchers often approach trials too cautiously, convinced that the process is bound to be insurmountably complicated and riddled with roadblocks. A step back is needed, an objective examination of the drug development process as a whole, and recommendations made for streamlining the process at all stages. With Intelligent Drug Development, Michael Tansey systematically addresses the key elements that affect the quality, timeliness, and cost-effectiveness of the drug-development process, and identifies steps that can be adjusted and made more efficient. Tansey uses his own experiences conducting clinical trials to create a guide that provides flexible, adaptable ways of implementing the necessary processes of development. Moreover, the processes described in the book are not dependent either on a particular company structure or on any specific technology; thus, Tansey's approach can be implemented at any company, regardless of size. The book includes specific examples that illustrate some of the ways in which the principles can be applied, as well as suggestions for providing a better context in which the changes can be implemented. The protocols for drug development and clinical research have grown increasingly complex in recent years, making Intelligent Drug Development a needed examination of the pharmaceutical process.

scholarly journals Participant-funded clinical trials on rare diseases

2020 ◽  
Vol 93 (4) ◽  
pp. 267.e1-267.e9
Author(s):  
Rafael Dal-Ré ◽  
Francesc Palau ◽  
Encarna Guillén-Navarro ◽  
Carmen Ayuso
Keyword(s):  
Clinical Trials ◽  
Rare Diseases

Perspectives on Virtual (Remote) Clinical Trials as the “New Normal” to Accelerate Drug Development

2021 ◽  
Author(s):  
Demissie Alemayehu ◽  
Robert Hemmings ◽  
Kannan Natarajan ◽  
Satrajit Roychoudhury
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
New Normal

scholarly journals Drug Repurposing Opportunities in Pancreatic Ductal Adenocarcinoma

2021 ◽  
Vol 14 (3) ◽  
pp. 280
Author(s):  
Rita Rebelo ◽  
Bárbara Polónia ◽  
Lúcio Lara Santos ◽  
M. Helena Vasconcelos ◽  
Cristina P. R. Xavier
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Pancreatic Ductal Adenocarcinoma ◽  
De Novo ◽  
Drug Repurposing ◽  
Metastatic Tumor ◽  
Therapeutic Options ◽  
Ductal Adenocarcinoma ◽  
Clinical Indication ◽  
Novel Treatments

Pancreatic ductal adenocarcinoma (PDAC) is considered one of the deadliest tumors worldwide. The diagnosis is often possible only in the latter stages of the disease, with patients already presenting an advanced or metastatic tumor. It is also one of the cancers with poorest prognosis, presenting a five-year survival rate of around 5%. Treatment of PDAC is still a major challenge, with cytotoxic chemotherapy remaining the basis of systemic therapy. However, no major advances have been made recently, and therapeutic options are limited and highly toxic. Thus, novel therapeutic options are urgently needed. Drug repurposing is a strategy for the development of novel treatments using approved or investigational drugs outside the scope of the original clinical indication. Since repurposed drugs have already completed several stages of the drug development process, a broad range of data is already available. Thus, when compared with de novo drug development, drug repurposing is time-efficient, inexpensive and has less risk of failure in future clinical trials. Several repurposing candidates have been investigated in the past years for the treatment of PDAC, as single agents or in combination with conventional chemotherapy. This review gives an overview of the main drugs that have been investigated as repurposing candidates, for the potential treatment of PDAC, in preclinical studies and clinical trials.

Sign in / Sign up

Export Citation Format

Share Document