Even though the number of patients treated with these products may be very limited, quality is a must for the patients and a fundamental requirement of the regulatory authorities. Patients suffering from rare disorders are entitled to the same level of quality as any other patients. Issues in the pharmaceutical development of drugs for rare disorders are numerous and mainly due to the small number of patients.
Lucane Pharma has extensive expertise in the development of pharmaceutical grade API material into appropriate and acceptable dosage forms for very small numbers of patients suffering from life-threatening diseases. There is often a challenge to provide the medication at varying doses depending on body weight for neonates, children and adults. Production challenges for small volumes include the optimization of batch size and shelf life.