Expanded Access to Investigational Therapies
SpringWorks Therapeutics is committed to developing safe and effective therapies for patients with severe rare diseases and cancer. We believe that wherever possible, use of an investigational therapy for a patient as part of a clinical trial is preferable because clinical trials are designed to generate comprehensive safety and efficacy data that may lead to the approval of new medicines and, consequently, to wider availability for patients in need.
In cases when a patient may not qualify for a clinical trial, there may be no trials available, or a patient has exhausted all available treatment possibilities, SpringWorks may be able to provide access to eligible patients through our Expanded Access Program. This refers to the use of a SpringWorks investigational therapy outside of our clinical trials and prior to its commercial availability.
Criteria for Considering a Request for Expanded Access
SpringWorks is committed to reviewing all requests for Expanded Access in line with criteria consistent with International, regional and local regulatory authorities. The following criteria – indicated below, but not limited to – must be met by the individual patient, the requesting healthcare provider (HCP) and the SpringWorks investigational therapy in order for a request to be considered.
- The patient has a serious, debilitating or life-threatening medical condition with no suitable treatment available, has exhausted current standard of care or declined medically-appropriate treatment for ethical or personal reasons.
- The patient does not qualify to participate in any ongoing clinical trial sponsored by SpringWorks in an accessible geographical location.
- Expanded Access does not impede or compromise the ongoing clinical development program or regulatory approval of the investigational therapy.
- Sufficient clinical data are available for both the disease condition and investigational therapy and such data indicate that the potential benefits of access to the investigational therapy outweigh any known potential risks as determined by the treating physician.
- There is adequate supply of the investigational therapy to support an Expanded Access Program and such supply will not negatively impact a clinical trial or product anticipated for product launch or other required purposes.
- The HCP must be currently licensed and report side effects of the investigational therapy.
- The HCP must obtain informed consent from the patient or his/her legal guardian(s) for treatment use of the investigational therapy.
- The HCP must supervise administration of the investigational therapy in line with SpringWorks defined access criteria (which in some countries may include a protocol for treatment use).
Making a Request for Expanded Access
Requests for access to a SpringWorks investigational therapy must be made voluntarily by the patient’s treating physician. Requests cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver.
To make a request for expanded access, physicians in the United States should send an email to firstname.lastname@example.org. Physicians from outside of the United States should email email@example.com. The following information must be included in any request:
- Date of request;
- Requesting physician’s name, contact information, address (including country), and professional qualifications;
- Name of the requested SpringWorks investigational therapy, the therapeutic indication and intended treatment plan;
- Medical rationale for the request, including an explanation why alternative therapies cannot be used, why the patient does not qualify for an ongoing or planned clinical trial, why the potential benefit from the use of the investigational product outweighs the potential risks and why those risks are not unreasonable in the context of the disease or condition being treated.
We are committed to evaluating all requests for expanded access in a fair and equitable manner. SpringWorks will acknowledge requests from licensed physicians as soon as possible, generally within two business days of receipt and a decision will be made and communicated as soon as possible thereafter. SpringWorks cannot guarantee that all requests for access will be granted, even when eligibility criteria are met.
Any Expanded Access to investigational therapies must comply with the applicable country-specific laws and regulations, including importation requirements, approvals from applicable regulatory bodies, and by an appropriate Institutional Review Board or Ethics Committee. SpringWorks may require agreements and/or certifications executed by the treating physician and other information to be provided prior to final approval of any request.
Patients and caregivers seeking information about access to our investigational therapies, either through a clinical trial or through Expanded Access, should consult with their treating physician.
Should expanded access programs become available for our investigational therapies, we will update this website with hyperlinks to any available expanded access programs on www.clinicaltrials.gov.
SpringWorks may revise this Policy at any time in line with the 21st Century Cures Act.