Issue link: https://www.e-digitaleditions.com/i/1450659
36 Pharmaceutical Technology Europe BIO/PHARMA OUTSOURCING INNOVATION eBOOK 2022 P h a r mTe c h . c o m Jones: Typically, the manufacturing of an orphan medi- cine is undertaken at a considerably smaller scale than tra- ditional commercial manufacturing, because of the very limited patient population. When orphan designated drugs receive approval for expanded indications, this might af- fect the scale of commercial manufacturing. Therefore, a CDMO with the experience of manufacturing at custom- ized scales, as well as the f lexibility to respond to changes in the types of products being sold, the volume of sales, and the facilities and equipment needed for production, may be better able to help a sponsor company overcome barriers in terms of cost and timeline during an orphan drug development. PTE: How can a CDMO help a sponsor obtain regulatory approval for an orphan drug? What specific services and/ or facilities can a contractor provide? Jones: When information is needed on specific technolo- gies, or processes that require alteration, the CDMO's regu- latory team can respond quickly with minimal risk of error. A CDMO is also able to offer sponsors a global network of regulatory affairs experts that have local knowledge, to facilitate successful agency interactions. In addition, CMC activities could be easily and quickly prioritized to ensure that they do not present as bottlenecks to the execution of the orphan development programme and submission of application to regulatory agencies. A CDMO should al- ready be intimately familiar with its dosage form(s), which can be customized to potentially address a specific orphan medicine's need. They are then able to provide complete advice and provide support on the formulation, develop- ment, and manufacturing intricacies. Leveraging a CDMO to obtain regulatory approval for an orphan drug may also result in overall improvement of efficiency in orphan drug product development time and accelerate product approval. PTE: How can a CDMO help a sponsor control the costs, specifically, of the development and manufacture of an orphan drug? Mollan: A major global CDMO could provide access to an international network of technical expertise and state- of-the-art facilities. An integrated CDMO can save costs by accelerating timelines, reducing programme risks, and ensuring a smooth transition between early and late-phase manufacturing sites. These benefits are achieved by har- monizing equipment, processes, pharmaceutical excipi- ent vendors, collaboration tools, and project management structure between sites. PTE: Can a CDMO help a sponsor company determine which medical needs might warrant the development of an orphan drug? Mollan: For the most part, CDMOs do not determine which diseases to target, but can help refine the target product profile and ensure that the final product meets the needs of the target patient population. Developing a for- mulation and process that can accommodate a very wide range of potential drug loading is a key decision that can significantly de-risk changes in drug loading requirements as more information becomes known. Many factors drive the development of the target product profile, and these include the physicochemical and pharmacokinetic prop- erties of the API, patient considerations such as age and co-morbidities, and manufacturability of the drug product used in the treatment. References 1. FDA, Developing Products for Rare Diseases & Conditions, FDA. gov, accessed 26 Jan. 2022. 2. M.E. Schneider, "Orphan Product Designation Requests Climb in the US, Remain Steady in the EU," Regulatory Affairs Profes- sionals Society, 11 March 2021. 3. D. Horgan et al., "Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases – Is It Time to Update the EU's Orphan Regulation? And if so, What Should be Changed?" Biomed Hub 2020;5:1-11. doi: 10.1159/0005 PTE Quality/Regulations Ascendia Pharmaceuticals ..................................................................... 17 Baxter .......................................................................................................... 2 Catalent Pharma Solutions ..................................................................... 37 CoreRx ......................................................................................................... 5 mAbxience ................................................................................................ 21 Syngene ....................................................................................................... 9 Ad Index COMPANY PAGE "A major global CDMO could provide access to an international network of technical expertise and state-of-the-art facilities." — Matthew Mollan, Catalent