About Clinical Studies

A clinical trial is a medical research study designed to assess whether or not a new drug or treatment is well-tolerated and effective in treating people with a particular disease.

Strict regulations govern the development and approval of new drugs.

All new drugs that reach the human stage are required to go through an orderly series of clinical trial steps called phases. All clinical trial phases involve thorough follow-up and assessment of the effects of the new drug in human volunteer participants. Researchers design each phase to address a different question.

Early trial phases often involve assessment of the effects and the safety of the drug in healthy volunteers. In later phases, the effects of the drug are evaluated in patients with the disease for which the drug is intended to treat. In some trials, the new drug is compared to a sham drug, called a placebo. In others, the new drug may be compared to existing treatments, to check whether the new drug offers advantages. In all clinical trial phases researchers monitor all participants carefully and regularly to gather data about the effects of the drug.

Some patients with serious diseases may be keen to try new drugs that offer them hope of improvement or cure and may find the intricacies of this regulated process frustrating. But researchers and pharmaceutical companies are legally obliged to comply with these regulations in order to ensure that there is optimal patient safety at every stage and that new drug efficacy and safety is evaluated by rigorous scientific standards.

At Ionis, our efforts begin and end with the patient in mind.

From discovery to development to getting medicines into the hands of those who need it, we are inspired by the needs of the patient communities we serve and fueled by the promise of our science. Every aspect of what we do is grounded in patient perspectives.

Take a closer look at some of the people who inspire our work by clicking here.

Ionis Pharmaceuticals is driven by our belief that sick people depend on us. Our mission is to develop first-in-class and best-in-class medicines that transform the lives of those affected by severe diseases by improving their health and well-being. We are committed to creating new, safe, and effective medicines to treat diseases 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 patients in need, and well-designed clinical studies followed by regulatory approval.

The best way to access an investigational product, i.e. a medicine which has not yet received regulatory marketing approval, is through participation in one of our clinical studies.

We understand that there are seriously ill patients with life-threatening diseases who will not be eligible for our clinical studies and for which no approved medicine is available. Outside of a clinical study expanded access (also referred to as “compassionate use”) to investigational products will be considered, with the primary purpose of supporting those patients with a serious or immediately life-threatening condition who have exhausted all available medical options and are unable to enroll in any ongoing clinical study.

Criteria Used for Considering Requests for Expanded Access

To obtain expanded access from an investigational Ionis medicine, all of the following conditions must be met:

• The patient has a serious or immediately life-threatening disease or condition.
• There is no comparable or satisfactory alternative therapy for the disease or condition.
• The patient does not qualify to participate in any ongoing clinical study sponsored by Ionis, which includes lack of access due to geographic limitations.
• Sufficient clinical evidence of safety and effectiveness for the indication has been established and concluded that the potential benefit outweighs the potential risks to the patient requesting the medicine and to other patients.
• Product is under active development for the requested disease indication and making the medicine available will not negatively impact or delay the conduct of clinical studies or regulatory review or approval of the investigational drug for broader patient access.
• Adequate supply of investigational product exists.
• There is a regulatory mechanism in the country or region to support expanded access.