Expanded Access and Compassionate Use

Sometimes called “compassionate use,” expanded access is a potential pathway for a patient with a life-threatening or serious disease to gain access to an investigational medicinal product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Expanded Access Policy

At Sapience we are committed to developing safe and effective therapies for high mortality cancers and making them available to patients as quickly and efficiently as possible by conducting rigorous clinical trials needed to gain regulatory approval. Wherever possible, use of an investigational medicinal product by a patient is best evaluated in a clinical trial that is designed to collect safety and efficacy data and to answer important scientific questions about its potential risks and benefits.

It is important to remember that investigational medicinal products have not yet received regulatory approval; thus, their potential risks and benefits for a specified use are not yet established. Physicians and patients should consider all possible benefits and risks when seeking access to an investigational medicinal product.

We support expanded access to our investigational medicinal products when there is substantial scientific evidence to assure both safety and efficacy. Early in development the risks of expanded access tend to outweigh the benefits due to limited data. Therefore, evaluation of requests will generally require availability of positive Phase 3 data. The following criteria for expanded access eligibility will be evaluated for all requests:

Investigational medicinal product program criteria:

• substantial scientific evidence exists to support both the safety and efficacy of the investigational medicinal product for a particular indication, typically after positive Phase 3 data is available;
• expanded access use will not compromise the development program, conduct of clinical trials or regulatory approval pathway;
• there is intent to gain regulatory marketing approval for the investigational medicinal product in the region/country of the request, particularly where there is an expected need for ongoing treatment and administration through normal health services once the medicine is available for prescription;
• investigational medicinal product supplies are adequate to support the expanded access request and can be safely/reasonably/logistically supplied and administered outside of the clinical trial setting.

Patient eligibility criteria:

• existing approved therapies are not appropriate or effective and the patient is not eligible for participation in ongoing or pending clinical trials, such that consideration of using an unapproved therapy is justified;
• the request is for treatment of a serious or life-threatening condition and assessment of clinical safety and efficacy data have determined there is sufficient evidence of a potential benefit that outweighs the known or anticipated risks to the patient;
• there is adequate information to support appropriate dosing for a special population such as pediatric, elderly, renal or hepatic disease, etc.;
• the patient’s underlying medical conditions do not pose safety risks that have not been sufficiently studied;
• the request has been made by a properly licensed and medically qualified physician with expertise and facilities appropriate for the administration of the therapy, monitoring, managing and reporting side effects, as well as patient experience;
• local, regional and institutional laws and regulations in the country/region of administration support availability and early access to investigational therapies.

Requests and Responses for Compassionate Use

Requests for access to investigational medicinal products must be made by a properly licensed and medically qualified physician on behalf of the patient. Requests are considered by qualified and licensed physicians at Sapience on a case-by-case basis in a fair and equitable manner based on the program information available at the time of the request and the patient eligibility criteria listed above. Qualified physicians can submit a request with the following information to Sapience at ExpandedAccess@sapiencetherapeutics.com:

• date of request;
• requesting physician’s name, contact information, address (including country), and professional designation (i.e., MD) or qualifications;
• name of the requested investigational medicinal product along with physician’s intended treatment plan, including therapeutic indication and expected duration of treatment;
• medical rationale for request including an explanation for why alternative therapy cannot be used, why the patient does not qualify for a clinical trial, and why use of the investigational drug is in the patient’s best interest.

Sapience anticipates it will acknowledge receipt of requests within five business days of receipt of the request.

Additional information regarding our pipeline of investigational medicinal products can be found on our pipeline page and information regarding specific Sapience clinical studies can be found at www.clinicaltrials.gov.

This policy is in accordance with United States Code: FD&C Act, 21 U.S.C. §360bbb. Pursuant to this Act: the posting of policies by manufacturers and distributors shall not serve as a guarantee of access to any specific investigational therapy by any individual patient.