Extemporaneous orphan drugs and preparations for rare diseases: organisation and management of an inter-regional network

Study Objectives: Piedmont and Aosta Valley hospital pharmacists have been working to create an inter-regional network to manage the extemporaneous compounding preparations for patients suffering from rare diseases. This network will work actively with the currently existing inter-regional network for the prevention, surveillance, diagnosis, and treatment of rare diseases. Methods: Activities of the Hospital Pharmacies Network include: profiling the extemporaneous compounding preparations prepared by the Piedmont and Aosta Valley hospital pharmacies to treat rare diseases; creating an appropriate data form for each preparation, which includes a list of ingredients, procedures, appearance and stability of the preparation, warnings, therapeutic use, expiry date, label, quality control, and literature references; writing up a list of instructions for each preparation to make it easier for the patients to use the medication; creating follow-up forms for new or dangerous preparations in order to improve the pharmaco-surveillance system, to confirm efficacy and quality data, and to acquire data for epidemiological and pharmaco-economic analysis. Results: Both a registry and a network of extemporaneous compounding preparations have been created. Follow-up data allows to monitor the efficacy and safety of the preparations. Other aims are to create an orphan drug extemporaneous compounding preparations registry in order to standardise preparation methods among pharmacists with regards to quality, efficacy and safety, and to extend this network to all Italian regions. Conclusion: The registry and the network are useful tools that are currently available to disseminate data concerning extemporaneous compounding preparations for rare diseases by standardising preparation methods, dispensation and monitoring, and thus improving the quality of patient care.