NuCana is focused on significantly improving treatment outcomes for patients with cancer. Our ProTides are designed to overcome the key cancer resistance mechnanisms associated with many chemotherapeutic agents. This should result in more effective and safer medicines.
NuCana believes that participation in clinical studies offers the best pathway for access to their investigational medicines prior to approval. The clinical development process involves rigorous evaluation of the safety and efficacy of investigational drugs through testing in clinical studies, which are conducted in compliance with applicable regulations.
Details about NuCana’s clinical studies, including eligibility requirements for participating in ongoing or planned studies are published in public databases, such as clinicaltrials.gov and clinicaltrialsregister.eu.
NuCana recognizes that there may be patients with cancer or a related condition who will be interested in seeking access to drugs that are in development before regulatory approval, such as:
• Patients who have participated in one of NuCana’s clinical studies who wish to continue treatment once the study has completed.
• Patients who are not eligible for NuCana’s clinical studies and may not have options for alternative therapies, including investigational therapies in studies being conducted by other companies.
These situations are typically referred to as expanded access, but are also known as compassionate use, early access, pre-approval access and emergency use.
NuCana has an ethical responsibility to ensure the safety, quality and integrity of clinical studies for both current and future research participants. During clinical development, and prior to regulatory approval, it is not known whether an investigational medicine is safe or effective. As such, expanded access is associated with several risks for both the patient and the drug development program. For patients, there is a potential safety risk and they may have a false sense of the benefit that the medicine can provide. For the development program, expanded access can adversely impact the approval of a new treatment that could benefit many future patients. Based on this, strict criteria apply for expanded access of NuCana’s investigational medicines.
Following completion of a clinical study, it could take several years for the new medicine to be approved. NuCana will decide if enough information is available to justify expanded access for patients who may benefit from continued treatment with the investigational drug once a clinical study has ended, but before the drug is available on prescription. In addition, this decision will be at the discretion of the treating physician in accordance with local regulations, and informed by available data on the safety and efficacy of the drug, the adequacy of drug supply, and whether alternative effective treatments are already available.
Requests for expanded access for patients who are not eligible to participate in NuCana clinical studies will be considered on a case-by-case basis and the decision will take into account the available clinical data, the benefit-risk profile, the impact on the clinical development program, and the probability of regulatory approval.
All of the following conditions must be met for NuCana to consider a request for expanded access:
1. The patient’s disease or condition is life-threatening.
2. There are no adequate alternative therapies or clinical studies available.
3. Sufficient clinical data are available for the drug so that NuCana can determine an appropriate dose and make a robust benefit-risk assessment.
4. The patient’s treating physician and NuCana’s Chief Medical Officer both agree that there is potential for the patient to receive benefit from the treatment, and there is robust evidence to support the possibility that the patient will benefit.
5. Adequate drug supply can be assured to support both ongoing clinical studies and expanded access, until (and if) the drug becomes commercially available.
6. The patient is not eligible for one of NuCana’s clinical studies.
7. Expanded access will not adversely impact the clinical development program.
8. The request must be made by the patient’s treating physician, unsolicited by NuCana or any other individual or organization. The request must be supported by evidence that the safety of the patient will be assured through continual access to medical supervision during exposure to the investigational drug.
NuCana is committed to evaluating all requests in a fair and equitable manner. If the above criteria are met, NuCana may consider expanded access requests from treating physicians subject to applicable country-specific laws and regulations. However, NuCana can not guarantee that an expanded access program will be available, nor that a particular patient will be offered access to the investigational drug.
The requesting physician must agree to obtain appropriate regulatory authority 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 NuCana whose decisions are final.
If the investigational drug later receives approval from the regulatory authorities, and is available for prescription in the patient’s country, NuCana will phase out expanded access to allow the health system to prescribe the new medicine in the normal manner. Alternatively, if emerging evidence suggests that the investigational drug does not demonstrate a positive benefit-risk profile, or if the drug is not approved by regulatory authorities, NuCana will discontinue all expanded access and work with patients and their physicians to help them safely transition to other treatments.
This policy may be reviewed and amended on an ongoing basis. For additional information about this policy, please contact expandedaccess@nucana.com. NuCana aims to provide responses within one week.
NuCana plc
3 Lochside Way, Edinburgh, EH12 9DT, UK
expandedaccess@nucana.com
Version 1 - Last Updated: 01 October, 2021