Our Managed Access Policy

Accessing Investigational or Unapproved Therapies Outside of Clinical Trials

Managed Access Programs are intended to advance access to treatment for patients with serious or immediately life-threatening diseases or conditions when participating in a clinical trial is not feasible or when a patient has already completed a clinical trial.

For each of our therapies, Insmed carefully evaluates the offering of various Managed Access Programs. In any instance where managed access is granted, the provision of Insmed therapies must comply with applicable local laws and regulations.

The Managed Access Programs Insmed may offer for a product include:

• Compassionate Use Program
• Post-Trial Access Program
• Named Patient Program

Compassionate Use Program (Referred to as Expanded Access in the U.S.)  

For investigational gene therapies and one-time therapies

Insmed recognizes the substantial unmet medical needs of patients with rare genetic diseases and is committed to contributing to the development of new therapies.  As gene therapy is a highly complex field with unique considerations around clinical trial design, supply chain, and manufacturing, we are currently focused on establishing the safety and efficacy of our investigational gene therapies through clinical trials. Therefore, at this time, we are unable to make our gene therapies accessible through a compassionate use program.

For Other Insmed Investigational Therapies

Insmed may, for designated therapies, consider offering access to individual patients with a serious or rare disease through a compassionate use program.  For a compassionate use program to be considered, the minimum following criteria must be satisfied:

• Insmed must have sufficient information on the appropriate use of its medicine for the patient’s disease, such as optimal dosage, treatment schedule, and treatment duration;
• Sufficient evidence must exist to support a favorable benefit-risk profile for the investigational therapy in the patient’s disease;
• Patient enrollment in a clinical trial must not be feasible;
• Providing the investigational medical product must not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication;
• There must be no other comparable or satisfactory therapeutic alternatives available.

Post-Trial Access Programs

Insmed may consider providing continued access to investigational therapy for patients who have participated in and completed an Insmed-sponsored clinical trial, but who may not have immediate access to the treatment at the end of their participation. A healthcare professional (HCP) request for Post-Trial Access must be based on the assessment of the patient’s medical needs, including the availability of alternative therapies.

Named Patient Program

Insmed may consider facilitating access to its therapy in a country where the product is not approved, after this product has received regulatory approval in at least one country.