Expanded Access and Compassionate Use Policy (US)
Tolero Pharmaceuticals, Inc. is committed to the development of therapies to benefit patients who have not had success with existing, available therapies. Our aim is to provide these therapies to the broadest number of patients possible, through performance of clinical research with the goal of obtaining regulatory approval. Approval by regulatory authorities is the only way to make medicines broadly available to patients by prescription from a qualified healthcare provider. The performance of clinical research allows Tolero Pharmaceuticals to properly evaluate our investigational therapies to generate requisite safety and efficacy data to secure such an approval. Please see clinicaltrials.gov for a full listing of ongoing Tolero Pharmaceuticals clinical trials.
Currently, Tolero Pharmaceuticals does not provide access to its investigational products outside of enrollment in clinical trials. We do recognize, however, that some patients with serious or immediately life-threatening diseases may not be eligible for any clinical trials, and may not have other options. Therefore, Tolero Pharmaceuticals may, in the future and in limited instances, consider expanded access to investigational products for patients residing in the United States and who may benefit from our investigational therapies currently in development. Tolero Pharmaceuticals’ Expanded Access and Compassionate Use Policy, as summarized below, describes the principles and general procedures that Tolero Pharmaceuticals will follow when considering a request for such use.
CRITERIA FOR EVALUATING ACCESS TO INVESTIGATIONAL AGENTS OUTSIDE OF TOLERO-SPONSORED CLINICAL TRIALS
As stated above, Tolero Pharmaceuticals does not currently provide access to these therapies in an expanded access setting. In the event that Tolero Pharmaceuticals may, in the future, consider such a request, each request for expanded access shall be evaluated in accordance with the Tolero Pharmaceuticals Expanded Access and Compassionate Use Policy, the principles and criteria of which are summarized as follows:
- The patient must have a serious or immediately life-threatening cancer.
- The investigational therapy must be in active clinical development with completion of Phase I study and with a safe dose identified. Tolero Pharmaceuticals must currently be studying the investigational therapy in human subjects. Once a medicine has been approved by the Food and Drug Administration, expanded access is no longer available.
- There must be no other viable therapy options, including participation in ongoing and relevant clinical trials. In order for Tolero Pharmaceuticals to grant expanded access to an investigational therapy, there must be no comparable or satisfactory alternative therapy for the disease or condition, or the patient must have exhausted all available therapies typically used to treat the disease, and no longer be responsive to, or able to tolerate, these treatments. In addition, the patient must not be able to qualify for participation in, or have access to, any ongoing or soon opening Tolero Pharmaceuticals sponsored clinical trials. Geographic limitations to participation in clinical trials are not sufficient to meet this criterion.
- There must be adequate supply of the investigational therapy as needed to accommodate the duration of the requested treatment.
- Access on an expanded basis cannot delay, interfere with, or compromise ongoing clinical trials or the potential approval for the product. Granting access to an investigational therapy cannot interfere with completion of ongoing clinical trials that could support regulatory approval of the therapy.
- There must be a positive benefit-risk ratio for the patient. The potential benefit to the patient must always be considered to outweigh the collective potential risks to the patient of offering the therapy. Data from phase 2 or phase 3 studies is typically necessary to support a robust risk-benefit analysis. Simple target or genomic analysis may not be sufficient to justify access to an investigational drug. Consideration will also be given as to whether or not the relevant patient’s underlying medical condition may pose a safety risk that has not been sufficiently studied. The requesting physician must provide the initial risk-benefit analysis.
- The request must be made by a physician who is qualified and licensed in the United States, who agrees to directly supervise treatment, and who has the expertise and facilities appropriate to administer the investigational therapy. The requesting physician must also obtain relevant health authority and Institutional Review Board approval/clearance, comply with relevant local and/or country regulations, and agree to follow any conditions or restrictions set by Tolero Pharmaceuticals for the particular drug and patient.
PROCEDURE FOR REQUESTING EXPANDED ACCESS AND RESPONSE TIMES
Requests for access to investigational therapies must be made by a qualified and licensed physician by contacting Tolero Pharmaceuticals Medical Information at 1-800-430-1415. The physician requesting access must provide specific case information, including pertinent patient history, as well as the following:
- A scientific rationale for the potential benefit that the investigational therapy could provide based on available preclinical and clinical data
- A statement that approved therapies, or therapies that have shown significant clinical benefit, which are typically used to treat the disease have been exhausted and the patient is no longer responsive to, or able to tolerate, these therapies
- A statement that there are no other viable therapy options, including participation in ongoing relevant clinical trials
No Personally Identifiable Information or Personal Health Information should be provided in any request. Tolero Pharmaceuticals will make every effort to acknowledge individual requests that have been submitted in accordance with the above requirements within five (5) business days of receipt.
HOW DECISIONS ARE MADE
Tolero Pharmaceuticals is committed to a fair and unbiased evaluation of each request made for access to investigational therapies. All decisions are based solely on the clinical circumstances surrounding each request, in accordance with the principles detailed above. Whenever possible, patients will be referred to ongoing clinical trials as the primary route to access investigational medicines. While the above criteria are those that will be considered by Tolero Pharmaceuticals in its evaluation of whether or not to offer expanded access, Tolero Pharmaceuticals cannot make any guarantee that an investigational therapy will be made available.
CONTACT FOR FURTHER INFORMATION
Persons with questions about Tolero Pharmaceuticals’ policy and procedures for expanded access or about expanded access to Tolero Pharmaceutical’s investigational drugs may contact us at 1-800-430-1415.
To learn more about the FDA’s regulatory framework for expanded access, including expanded access for individual use, please see: