How can I enhance my orphan drug development?
Accelerating orphan drug development from candidate selection to commercial manufacture and supply
With over 300 million people worldwide living with identified rare diseases, the development of new treatments to address these unmet clinical needs is an area of great interest in the pharmaceutical industry.
Despite improvements in regulatory support and commercial rewards for developing orphan drugs, difficulties remain for drug developers in bringing these medicines to market quickly; including limited R&D budgets, alongside an ever-evolving regulatory framework, challenging product development and clinical recruitment.
Integrated capabilities and expertise
At Quotient Sciences we have supported over 50 development programs for rare disease in the past 5 years. Our extensive formulation know-how, clinical pharmacology expertise, coupled with our agile and flexible approach to clinical and commercial manufacturing, makes us the ideal partner.
From pre-formulation and formulation development through clinical development and scale up for commercial manufacturing we provide an end-to-end solution for the development of orphan drug products. Our extensive expertise and capabilities for orphan drug development includes:
- Pre-formulation and API characterisation.
- Formulation and process development.
- First-in-human clinical testing and acceleration into POC trials.
- Bespoke clinical manufacturing and supply (Phase I-III) of drug products into patient trials.
- Rapid scale-up and commercial manufacturing of low-volume products.
As a fully integrated drug development, clinical testing and manufacturing organization, Quotient Sciences addresses the challenges associated with developing programs for rare diseases and orphan drug development . We are dedicated to accelerating the development of new drugs for patients around the world providing individual services or fully integrated programs through our unique Translational Pharmaceutics platform.
Connect with us today to learn how we can help support the development and manufacturing needs of your orphan drug program.
Related content
Learn more about orphan drug development
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WhitepaperAccelerating the Developing of Orphan Drugs for Rare DiseasesWorldwide there are over 300 million people living with identified rare diseases. This white paper will discuss four principal CMC challenges for the developers of orphan drugs, and the potential solutions which are emerging.
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Case StudiesNew Study Article: Absorption, metabolism and excretion of opicapone in human healthy volunteersNand Singh, Medical Director contributions to Bial Study Paper. This paper documents the study - absorption, metabolism and excretion of opicapone (2,5-dichloro-3-(5-[3,4-dihydroxy-5-nitrophenyl]-1,2,4-oxadiazol-3-yl)-4,6-dimethylpyridine 1-oxide), a selective catechol-O-methyltransferase inhibitor investigations.
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Scientific PostersWhy Archiving is a Crucial Part of Any Clinical TrialA Quotient Sciences Poster by Erika Reategui, Director, GCP Quality Assurance, Quality Assurance: Why Archiving is a Crucial Part of Any Clinical Trial
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WebinarMoving Drug Substance Off the Critical Path — Streamlined Strategies to Accelerate to First-in-Human & BeyondJoin Quotient’s Director of Drug Development Consulting, Dr Stephen McQuaker Vice President of Bioanalysis, Dr Stuart McDougall, as he explores case studies to demonstrate how drug substance and drug product development plans can be accelerated through innovative science and expertise to get drug substance off the critical path.
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