Grumpy-XL schreef op 26 december 2022 11:50:
ojrd.biomedcentral.com/articles/10.11... Het is natuurlijk heel aantrekkelijk om voor een orphan drug een tweede indicatie te hebben. Dan maak je mega gebruik van de beschermende voordelen en financieele voordelen van de orphan drug wetgeving.
pubmed.ncbi.nlm.nih.gov/32533523/Voor Leniolisib zijn al een aantal trial phases doorlopen. Het is de vraag in hoeverre voor een tweede indicatie al de trial fases opnieuw moeten worden doorlopen.
Phase 1: This phase typically consists of 50 to 100 healthy volunteers. During phase 1, researchers assess the drug’s safety, determine the side effects, and evaluate how the drug should be taken.
Phase 2: Typically, 100 to 300 patients with the condition for which the medicine, device, product, or treatment has been developed participate in this next phase. Researchers look to assess the short-term safety and effectiveness of the drug, find the dose at which it works best with the least side effects, and conduct a small-scale placebo comparison.
Phase 3: In phase 3, the pool of volunteers must be must larger. Typically, between several hundred and several thousand patients participate, so researchers can confirm the drug’s safety and effectiveness and compare the new drug to other compounds (e.g., a placebo or other therapies).
Following the completion of Phase 3, a drug is approved (or not approved) by the U.S. Food and Drug Administration (FDA) for use by specific patients. Phase 4 may only begin if the drug is approved.
Phase 4: Finally, after the drug is approved researchers look to study the effectiveness of the drug in a wide variety of patients as well as monitor the safety in a large group and enable the development of new uses for the compound. These studies often involve thousands of patients and can inform future research and development.