Expanded Access to Investigational Product Derazantinib

Introduction

The purpose of this policy is to describe the requirements and procedures for making requests for expanded access to Basilea Pharmaceutica International Ltd.'s (‘Basilea’s’) investigational product derazantinib.

Basilea believes that conducting clinical trials and obtaining the necessary regulatory approvals/marketing authorizations provides patients with the best mechanism for access to our products. However, Basilea recognizes that some patients with serious or life-threatening diseases or conditions that cannot be treated satisfactorily with any currently authorized medicine may not be eligible to participate in a clinical trial, or may not have other treatment options.
Basilea supports expanded access (sometimes also called ‘compassionate use’ or ‘named‑patient use’) to derazantinib when there is sufficient scientific evidence to support both the safety and the efficacy of such treatment, when it is logistically feasible, and when it is permitted by applicable laws and regulations. The clinical trial record containing information about derazantinib can be found at https://clinicaltrials.gov/ct2/show/NCT03230318.

Policy

Basilea will consider providing an individual patient access to derazantinib outside of a clinical trial when all of the following criteria are met:

  1. The disease or condition of the patient is serious or life-threatening.
  2. The patient has received appropriate standard treatments, such as standard of care, without clinical success, and there is no comparable or satisfactory alternative therapy to treat the patient's disease or condition.
  3. The patient is ineligible to participate in any ongoing clinical trial with derazantinib, or is unable to participate in such a trial due to geographical limitations.
  4. Derazantinib is in the process of undergoing investigation in clinical trials, or has entered the marketing authorization application process.
  5. The provision of expanded access does not delay, interfere with, or compromise, the completion of the clinical trial process and/or the marketing authorization application process.
  6. There is sufficiently robust preliminary safety and efficacy data about derazantinib, including dosing information.
  7. There is sufficient scientific evidence to expect that the patient may derive a clinically meaningful benefit, and that derazantinib will have an acceptable safety profile.
  8. A request for access to derazantinib is submitted by the treating physician on behalf of the patient, and the treating physician is properly licensed, authorized, and fully qualified to administer the product in his/her country. The treating physician must comply with all regulatory requirements, and be willing to conduct all necessary medical monitoring, safety reporting, and data collection.
  9. Any expanded access to derazantinib must comply with the laws and regulations applicable to the country in which the product will be prescribed, including (as applicable) medicine importation requirements, approvals from the competent regulatory bodies, and approvals from the competent Institutional Review Board or Ethics Committee.

 

Treating physicians should submit expanded access requests on behalf of patients directly to expanded.accessbasileacom. The submission must contain a brief patient history, sufficient information on the patient's current medical status, and the treating physician's contact information. The submission must not contain any information identifying the patient, such as names, including patient ID numbers.

Basilea will acknowledge receipt of such a request within 3 working days.

The decision to provide derazantinib to a treating physician will be made by Basilea based on an unbiased evaluation of the request that meets the above conditions. This decision will be made in a timely manner to be responsive to the patient’s needs, but with appropriate consultation and discussions with Basilea's medical and clinical teams, the patient’s treating physician, regulatory authorities, and outside experts as required.

If the request is supported by Basilea, the treating physician must apply for, and receive, approval from the applicable country regulatory authority, to allow for importation of the investigational product.