Ionis Expanded Access Policy
Our mission is to develop medicines that transform the lives of those affected by severe diseases by improving their health and well-being. We are committed to creating new medicines for people where no other therapeutic approaches have proven effective.
We believe that the goal of making life-changing medicines available to the patient community is achieved through rigorous science, innovation, a commitment to people in need, and well-designed clinical studies followed by regulatory approval. The best way to access an investigational drug, i.e., a medicine being studied which has not received regulatory marketing approval, is through participation in a clinical trial.
Expanded access is one potential pathway for people with life-threatening diseases to gain access to an investigational drug outside of a clinical trial when no satisfactory alternative treatment is available. Ionis brought together our medical, clinical research and development, regulatory and senior leadership colleagues to thoroughly evaluate and challenge our policy on expanded access. We have further refined our position through communications with patient advocacy groups and expert independent bioethicists. In alignment with regulatory guidance and developed using the FDA criteria for expanded access, our Ionis policy considers the following criteria to determine if an Ionis investigational drug can be made available for treatment outside of a clinical trial.
- The individual has a serious or immediately life-threatening disease or condition.
- There is no comparable or satisfactory alternative therapy for the disease or condition.
- The individual does not qualify to participate in any ongoing clinical trial.
- Sufficient clinical evidence of safety and effectiveness for the indication has been established and concluded that the potential benefit outweighs the potential risks.
- The investigational drug is under active development for the requested disease indication.
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access.
- Adequate supply of investigational drug exists.
- There is a regulatory mechanism in the country or region to support expanded access.
We review and consider expanded access to our investigational drugs regularly, especially as new information about an investigational drug and its impact emerges from our clinical trials. While reviews like this may not lead to an expanded access program allowing access outside of clinical trials, upholding our commitment to patients means ensuring decisions of this magnitude are discussed and reviewed continually. We will update this site to provide information on available Ionis expanded access program(s) including the request process.
Please note that participation in one of our clinical trials is currently the only way to access Ionis’ investigational drugs.
Some of our investigational drugs are being developed with an industry partner. Application of this Ionis Expanded Access Policy to these medications is dependent on the partnership agreement we have in place.
We encourage individuals to speak directly with their physician regarding current or future treatment options, clinical trials, and expanded access programs. Physicians may contact Ionis directly at [email protected] with any additional questions about our investigational drugs. We anticipate acknowledging receipt of requests for information sent to this email within 5 business days. In line with the 21st Century Cures Act, Ionis may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov after such record becomes active.