Investigational drugs such as IDE196 are medical products that have not yet been approved or cleared by FDA and the FDA has not found these drug products to be safe and effective for their specific use. Investigational drug products may, or may not, be effective in the treatment of the condition for which they are being investigated, and use of the product may cause unexpected serious side effects.
Sometimes alternatively referred to as “compassionate use”, an expanded access program is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational drug product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Expanded access may be appropriate when all the following FDA guidelines apply:
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- Patient enrollment in a clinical trial is not possible.
- Potential patient benefit justifies the potential risks of treatment.
- Providing the investigational drug product will not interfere with investigational trials that could support the drug product’s development or marketing approval for the treatment indication.
Based on the policy outlined below, and specifically the guiding principle that there must be sufficient clinical data to support a positive benefit/risk before early access can be considered, IDEAYA currently consider requests for access to IDE196 (darovasertib) through an expanded access program for patients with metastatic uveal melanoma (MUM). We do not offer any of our other developmental medicines through such a program. To learn more about the IDE196 clinical trial, please visit clinicaltrials.gov (NCT03947385).
The safety and efficacy of IDE196 (darovasertib) and other agent(s) under investigation have not been established with the U.S. FDA or other regulatory agencies.
How Expanded Access Works at IDEAYA
Consistent with applicable laws and regulations, IDEAYA may, at its discretion, provide patients with access to our investigational treatments through an expanded access program when there is sufficient evidence of the investigational agent’s safety and efficacy to support its use in a particular situation.
The decision to allow expanded access to an investigational treatment is complex and consideration of a request for expanded access to IDE196 may be granted only if the following criteria, in addition to FDA guidelines, are met:
- The patient’s condition is serious or life-threatening.
- The patient’s disease is metastatic uveal melanoma (MUM) for which the investigational treatment is currently being evaluated.
- There is an adequate supply of the investigational treatment.
- It is logistically feasible to make the investigational treatment available and it can be safely administered in a controlled setting. The referring physician must refer to a physician and site experienced with the safe use of an IDE196 product via clinical study(ies) participation or a major academic center familiar with clinical trial execution.
- Physician is willing to complete and adhere to regulatory mechanism for access and have adequate staff and facilities to safely administer and monitor patient.
- The treatment plan and required observation/follow-up (inclusion/exclusion criteria, follow-up and safety monitoring) must be adhered to as provided.
- Required informed consent must be obtained.
IDEAYA will evaluate each request for expanded access in a timely and fair manner in compliance with the IDEAYA policy and applicable laws. A request for expanded access to an investigational treatment must be made by a qualified and licensed physician to firstname.lastname@example.org.
IDEAYA will acknowledge receipt of a request within 5 business days. Each request will be evaluated by members of our medical and regulatory team based on the criteria above for the requested treatment, and a decision will be provided to the requesting healthcare provider.
IDEAYA may revise this policy at any time and will remain aligned with the 21st Century Cures Act and other applicable laws.