Compassionate Use Program
We are committed to developing innovative therapies for patients with serious diseases.
Clinical trials are a critical component of this effort, and they help to determine whether a new therapy is safe and effective. Until regulatory authorities make the decision whether to approve a new therapy for a specific disease, it remains experimental and is generally not available to patients with that disease or condition outside of the clinical trials. If you would like to learn more about our clinical programs, visit www.clinicaltrials.gov.
We understand that there are patients who will not be eligible for available clinical trials and don’t have options for alternative therapies. In these circumstances, we will consider allowing access to the investigational therapy through Compassionate Use (CU). CU is for an individual patient when no comparable or satisfactory alternative therapy options are available. This type of access program requires that the request for access to the drug is submitted by the treating physician and that the CU Core Principles are met:
CU Core Principles
- Providing access will not jeopardize either the clinical trials which determine whether the therapy is safe and effective, or regulatory pathways which make the decision whether to approve the therapy.
- The patient has a life-threatening disease and the physician believes there are no satisfactory alternative therapies on the market.
- The patient can potentially benefit from treatment and there is scientific evidence that the potential benefit outweighs risk to the patient’s safety.
- Providing access will not jeopardize the ability for Apellis to conduct essential tasks that are necessary to bring effective therapies to people suffering from the diseases we seek to treat.
Apellis has an active, global, Compassionate Use program. For any additional information, please email email@example.com