Expanded Access to Investigational Products

Introduction

The purpose of this policy is to describe the requirements and procedures that allow physicians to request access to Basilea Pharmaceutica International Ltd. investigational products in accordance with local laws and regulations, such as those applying to "Expanded Access", "Named‑Patient Supply", "Autorisations temporaires d'utilisation (ATU)", "Compassionate Use" and others. These are all referred to here as "Expanded Access".

Basilea believes that conducting clinical trials and obtaining the necessary regulatory approvals/marketing authorizations for medicinal products ensures that safe and effective medicinal products can be made available for patients. However, Basilea acknowledges that patients with serious or life-threatening diseases or conditions that cannot be treated satisfactorily with any currently‑authorized medicinal product, with no other treatment options, may sometimes seek treatment with investigational products. In these cases, the treating physician can request an investigational product for the patient from Basilea before regulatory approval has been granted, if allowed by the applicable local laws and regulations.

Basilea will assess these requests for access to investigational products in accordance with its policies, and provided there is sufficient scientific evidence to support both the safety and efficacy of such treatment for the patient, may grant access the investigational product.

Policy

Basilea will assess a request for the supply of an investigational product for Expanded Access in consideration of applicable local laws and regulations.  The following criteria must also be met for a patient to be able to participate in a Basilea Expanded Access program:

  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 the investigational product, or is unable to participate in such a trial due to geographical limitations.
  4. The investigational product has been, or is being, tested in a clinical trial.
  5. The provision of the investigational product to the patient will not delay, interfere with, or compromise, any clinical trial or related approval processes for the medicinal product.
  6. There is sufficiently robust preliminary safety and efficacy data about the investigational product, including dosing information.
  7. There is sufficient scientific evidence to expect that the patient may derive a clinically meaningful benefit, and that the treatment with the investigational product will not involve risks that are outside the acceptable safety profile of the investigational product.
  8. A request for access to the investigational product 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 or her country of practice. The treating physician must agree to comply with all regulatory requirements, and must be willing to conduct all necessary medical monitoring, safety reporting, and data collection activities.
  9. Any Expanded Access to the investigational product must comply with the laws and regulations applicable to the country in which the product will be prescribed and used, including (as applicable) medicine importation requirements, approvals from the competent regulatory bodies, and approvals from the competent Institutional Review Board or Independent Ethics Committee.

 

Treating physicians should submit Expanded Access requests on behalf of patients directly to expanded.access@basilea.com. 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, including names or patient ID numbers.

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

The decision as to whether to provide the investigational product to the treating physician will be made by Basilea based solely on an evaluation of whether the request 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 independent experts as required.

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