to investigational drugs policy
This document describes Leap Therapeutics’ position and reflects our Corporate Policy regarding access to Leap Therapeutics Investigational Medicinal Products (IMP) outside of a clinical trial.
The scope of this policy is the use of a Leap Therapeutics IMP outside of a clinical trial, in active development, that is not approved for marketing in the country where the access program is being conducted.
Leap Therapeutics operates two types of access programs, as described in this document:
- Post-Trial Access – Continued treatment for patients successfully completing one of our clinical studies
- Early Access – The provision of access to a Leap Therapeutics IMP for patients suffering from a disease that is not possible to be treated with currently available medicines, or patients are not able to participate in one of our clinical studies.
Leap Therapeutics is committed to developing drugs to change the practice of cancer medicine. Our first priority in this commitment is to bring forward promising cancer drugs through our clinical trials. Leap Therapeutics development resources are focused on conducting Good Clinical Practice clinical trials and obtaining regulatory approval of investigational products. When a request for the use of an investigational product outside the scope of a clinical trial is received, Leap Therapeutics will review each request making reasonable accommodations so seriously ill patients who have exhausted other appropriate treatment options may, under the conditions described in this policy and in accordance with applicable local law, have appropriate access to investigational products before they are commercially available.
Where there is a possible need for patients to gain early access to an IMP, Leap Therapeutics will consider the request on a case by case basis, using the following criteria:
- There is a need for ongoing treatment with an IMP, we recognize that this must be through normal health services once it is available for prescription. As such we will only conduct clinical studies and provide early access outside of studies, in those countries where Leap Therapeutics intends to make the medicine available through normal prescription channels after its approval.
- There are sufficient clinical data available with respect to both the IMP, molecular science, and the disease condition for which the request is being sought to anticipate that any potential benefits from treatment are likely to outweigh any associated risks to the patient
- While addressing this patient need, Leap Therapeutics will ensure that if we agree to provide early access to the IMP, that there is sufficient medicine available to ensure the supply will not be compromised for those patients receiving it through the early access program, or for those patients in our clinical studies. This is because we want to ensure that when providing early access, it does not prevent us completing clinical studies and regulatory approval that would lead to wider access through normal routes of prescription. Any early access programs we provide will be in line with the laws and regulations of the country involved.
To be sure that our new medicines will work effectively and be safe enough to be prescribed for the diseases patients are suffering from, we study them rigorously through clinical trials to see whether they provide adequate treatment. The results of these clinical studies allow the regulatory authorities to independently assess whether to approve the new medicines to be prescribed. As this can take several years, patients who participate in our clinical studies may have to wait for the IMP to become available on prescription. Where there may be a benefit for a patient to continue to be treated after the clinical study has ended, but before it is available by prescription, we will decide if we have enough information to justify early access. This will depend on the severity of the disease we are studying, how much evidence we have that the IMP works as expected and is sufficiently safe, and whether alternative effective medicines are already available by prescription. To be sure we can assess whether the IMP continues to work effectively and is safe enough to continue to be used during early access, we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.
COMPASSIONATE OR EMERGENCY USE OUTSIDE OF CLINICAL STUDIES
The best way to see if a new medicine is sufficiently safe and effective is to run clinical studies. As such we prefer that, where possible, patients have the opportunity to participate in our clinical studies. We recognize however that not everyone has this opportunity. Where patients who cannot enroll in a clinical study are suffering from serious or immediately life threatening diseases, and there are no satisfactory alternative treatments available, we will consider early access if there is enough evidence available in their condition (as described above) and whether there is a possibility of them being able to access the IMP by prescription in the future. In the same way as after a clinical study, we want to be sure we can assess whether the IMP continues to work and is safe enough to continue to be used. As such we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.
DISCONTINUATION OF EARLY ACCESS
When the IMP receives approval from the regulatory authorities for the disease we are studying, and is available for prescription in the patient’s country, we will phase out the early access to allow the health system to prescribe the IMP in the country context.. If however it is approved but not available for prescription for some reason, and there is no alternative medicine available, we will work with country health authorities to discuss if we can jointly set up a patient-assistance program to allow patients who are benefitting from it, who do not have alternative treatment options, to continue to receive the IMP. Patient safety is our primary concern, and we respect patient safety by continually evaluating all the evidence that we have available to ensure that early access use of the IMP remains effective and appropriately safe. If there is a change in the outcome of trials or efficacy of the IMP, Leap Therapeutics will work with patients and physicians to transition patients to alternative treatments
Leap Therapeutics is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Leap Therapeutics may request more detailed information to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Leap Therapeutics whose decisions are final. Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to EarlyAccess@leaptx.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt. Response will be provided with 5 business days of receipt of a completed application.