scholarly journals Utilization of Biotechnological Drugs in Rare Diseases Requiring Use of Off-Label Drugs in Children in Turkey

2021 ◽  
Keyword(s):  
Rare Diseases ◽  
Off Label ◽  
Biotechnological Drugs

scholarly journals Off-Label Medication: From a Simple Concept to Complex Practical Aspects

2021 ◽  
Vol 18 (19) ◽  
pp. 10447
Author(s):  
Carmen-Maria Rusz ◽  
Bianca-Eugenia Ősz ◽  
George Jîtcă ◽  
Amalia Miklos ◽  
Mădălina-Georgiana Bătrînu ◽  
...  
Keyword(s):  
Rare Diseases ◽  
Drug Repurposing ◽  
Regulatory Agencies ◽  
Systematic Research ◽  
Clinical Practices ◽  
Safe Practice ◽  
Safety Issues ◽  
Off Label ◽  
Simple Concept ◽  
Approved Drugs

Off-label use of drugs is widely known as unapproved use of approved drugs, and it can be perceived as a relatively simple concept. Even though it has been in existence for many years, prescribing and dispensing of drugs in an off-label regimen is still a current issue, triggered especially by unmet clinical needs. Several therapeutic areas require off-label approaches; therefore, this practice is challenging for prescribing physicians. Meanwhile, the regulatory agencies are making efforts in order to ensure a safe practice. The present paper defines the off-label concept, and it describes its regulation, together with several complex aspects associated with clinical practices regarding rare diseases, oncology, pediatrics, psychiatry therapeutic areas, and the safety issues that arise. A systematic research of the literature was performed, using terms, such as “off-label”, ”prevalence”, ”rare diseases”, ”oncology”, ”psychiatry”, ”pediatrics”, and ”drug repurposing”. There are several reasons for which off-label practice remains indispensable in the present; therefore, efforts are made worldwide, by the regulatory agencies and governmental bodies, to raise awareness and to ensure safe practice, while also encouraging further research.

scholarly journals From Off-Label to Repurposed Drug in Non-Oncological Rare Diseases: Definition and State of the Art in Selected EU Countries

2017 ◽  
Vol 1 ◽  
pp. maapoc.0000016 ◽  
Author(s):  
Paola Minghetti ◽  
Elena P. Lanati ◽  
Josie Godfrey ◽  
Oriol Solà-Morales ◽  
Olivier Wong ◽  
...  
Keyword(s):  
Rare Diseases ◽  
Drug Repurposing ◽  
Orphan Drugs ◽  
New Drugs ◽  
Success Rates ◽  
Great Opportunity ◽  
Off Label ◽  
Research Activities ◽  
Oncological Diseases ◽  
Repurposed Drugs

Introduction Almost 8,000 rare diseases exist worldwide, affecting approximately 350 million people. Nevertheless, only 5% receive a specific authorized or licensed treatment. The need for effective and rapidly available therapies is still unmet for many patients. Objective The objective is to define repurposing versus off-label drugs, and to evaluate pathways of repurposed drugs for rare non-oncological diseases in Italy, France, England, and Spain (the EU4 countries). Methods This original paper is based on 3 research activities: (i) a nonsystematic literature research; (ii) a questionnaire-based survey to regulatory experts; and (iii) research on approval timelines and therapy prices of repurposed non-oncology orphan drugs. Official approval dates in England are not available if the National Institute for Health and Care Excellence does not appraise the products. Results Only France provides a specific adaptive pathway from off-label to repurposed drugs. Pricing and reimbursement assessment for the drug samples varied across the EU4 countries: time-to-market for repurposed drugs versus new drugs is longer in all analyzed countries; that is, 979 days versus 462 days in Italy, 502 days versus 350 days in France, and 624 versus 378 days in Spain. Repurposed drugs have higher success rates from development to approval than novel drugs (30% vs. 11%). Small- and medium-sized enterprises owned 9 of 12 repurposed non-oncology orphan drugs, of which only 4 were reimbursed in all EU4 countries. Prices were more homogeneous across EU4 although the reimbursement rates were different. Conclusions Drug repurposing represents a great opportunity to treat rare non-oncological diseases. However, a more homogenous assessment across EU4 could ensure reimbursement and prices high enough to reward organizations investing in this field.

scholarly journals Off-Label Use in Rare Diseases: Myasthenia Gravis, Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes

2018 ◽  
Vol 2 (02) ◽  
pp. E118-E123
Author(s):  
Rudolf Janzen
Keyword(s):  
Myasthenia Gravis ◽  
Rare Diseases ◽  
Standard Treatment ◽  
Scientific Evidence ◽  
Off Label Use ◽  
Off Label ◽  
European Regulations ◽  
Myasthenic Syndrome ◽  
Regulatory Decisions ◽  
Label Use

AbstractThe off-label use of licensed drugs is widespread, and the risk of adverse events is elevated, especially if the scientific evidence is low. The treatment of rare diseases (<5/10,000) therefore may increase the risk of off-label use. The optimized standard treatment is primarily based on the regulatory decisions of the German Federal Joint Committee. The valid guidelines and the regulations of the German health system are discussed in this article. The criteria for indication and monitoring of off-label use are shown, especially focused on the problem of refractory myasthenia gravis. Escalation of treatment results regularly in off-label use. The arguments within the applicable guidelines should be followed. Some new aspects of the European regulations are likewise discussed.

The Collaborative Network Approach: a model for advancing patient-centric research for Castleman disease and other rare diseases

2019 ◽  
Vol 3 (1) ◽  
pp. 97-105
Author(s):  
Mary Zuccato ◽  
Dustin Shilling ◽  
David C. Fajgenbaum
Keyword(s):  
Rare Diseases ◽  
Treatment Options ◽  
Castleman Disease ◽  
High Impact ◽  
Collaborative Network ◽  
Network Approach ◽  
Grant Applications ◽  
Research Questions ◽  
Novel Strategy ◽  
Patient Centric

Abstract There are ∼7000 rare diseases affecting 30 000 000 individuals in the U.S.A. 95% of these rare diseases do not have a single Food and Drug Administration-approved therapy. Relatively, limited progress has been made to develop new or repurpose existing therapies for these disorders, in part because traditional funding models are not as effective when applied to rare diseases. Due to the suboptimal research infrastructure and treatment options for Castleman disease, the Castleman Disease Collaborative Network (CDCN), founded in 2012, spearheaded a novel strategy for advancing biomedical research, the ‘Collaborative Network Approach’. At its heart, the Collaborative Network Approach leverages and integrates the entire community of stakeholders — patients, physicians and researchers — to identify and prioritize high-impact research questions. It then recruits the most qualified researchers to conduct these studies. In parallel, patients are empowered to fight back by supporting research through fundraising and providing their biospecimens and clinical data. This approach democratizes research, allowing the entire community to identify the most clinically relevant and pressing questions; any idea can be translated into a study rather than limiting research to the ideas proposed by researchers in grant applications. Preliminary results from the CDCN and other organizations that have followed its Collaborative Network Approach suggest that this model is generalizable across rare diseases.

Reasons Behind ACIP's Off-Label Vaccine Recs

2011 ◽  
Vol 45 (9) ◽  
pp. 16
Author(s):  
BRUCE JANCIN
Keyword(s):  
Off Label
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