Access to Investigational Medicines

Kiniksa Pharmaceuticals, Ltd. (Kiniksa) is focused on developing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need and is conducting clinical trials to support this effort. Clinical trials allow Kiniksa to evaluate investigational products in order to generate the efficacy and safety information needed to obtain approval of those treatments and make them available to the appropriate patient population. Information regarding Kiniksa’s ongoing clinical trials can be found within the pipeline section of our website or on clinicaltrials.gov.

However, Kiniksa understands that not all patients may be eligible for its clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. For those patients, Kiniksa will consider providing access to a physician who requests access to a specific Kiniksa investigational drug for the treatment of an individual patient outside of a clinical trial if certain criteria are met. Outside of a clinical trial, Kiniksa will consider providing access to its investigational products in the situations below under limited circumstances only, and if permitted by applicable law.

Considerations for Access to Investigational Products

  • Kiniksa may provide continued access to its investigational products to research participants in a clinical study after a clinical trial is complete in select countries provided that applicable laws and criteria are met.
  • Kiniksa may provide physician-requested Expanded Access to its investigational products to patients with serious or immediately life-threatening diseases who lack other therapeutic options, cannot join an active clinical trial, and where the potential benefits of the investigational product outweigh the known risks in the disease indication (as described by the criteria below).

Criteria Used for Considering Requests for Expanded Access

Kiniksa uses the following criteria, in its sole judgment, to help evaluate requests for Expanded Access:

  • The patient has a serious or life-threatening illness or condition and is (a) no longer responsive to any available treatment option, (b) no longer able to tolerate any available treatment option, or (c) there is no comparable or satisfactory alternative therapy for the disease or condition;
  • A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational product available;
  • The investigational product is in active clinical development in the disease state of interest with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
  • Making the investigational product available will not have a negative impact on or delay the conduct of clinical trials or regulatory review or approval of the investigational product for broader patient access;
  • There is a regulatory mechanism in the country or region to support Expanded Access;
  • A mechanism is in place for reporting of safety data during the Expanded Access treatment; and
  • Adequate supply of the investigational product is available.

Kiniksa evaluates the benefit-risk profile of its investigational products based on emerging clinical data. Each of Kiniksa’s development programs is different. Making one investigational product available for the treatment of a specific patient does not mean that it will be made available in response to other requests as individual patient circumstances and medical histories differ. As such, each request will be considered on a case-by-case basis.

Process for Requesting Expanded Access

Kiniksa is committed to evaluating all requests in a timely, fair, and unbiased manner. Patients interested in seeking Expanded Access to a Kiniksa investigational product should talk to their treating physician. The treating physician may request information about applying for access to one of Kiniksa’s investigational products by contacting Kiniksa via email (ExpandedAccess@kiniksa.com).

Kiniksa will typically acknowledge receipt of a request for Expanded Access within 3 business days. Kiniksa will review all Expanded Access requests in consideration of the above criteria and may require more detailed information in order to fully evaluate individual requests.

All physicians who receive Kiniksa investigational product through Expanded Access are required to comply with all applicable laws and regulations and contractual conditions requested by Kiniksa, including, but not limited to, obtaining patient consent, patient monitoring and safety reporting, and obtaining appropriate regulatory and ethics committee approvals from their corresponding institution(s). Each request will be given careful consideration by a Kiniksa Review Team whose decisions are final.