Managed Access Programmes

Background

Evaluation of medicinal products in clinical trials

Before a medicinal product becomes available on the market, it must undergo a clinical trial programme to establish its safety and efficacy profile. Review of the clinical trial results by the relevant health authorities may then lead to approval and marketing authorization of the product. Please find a list of Sobi’s ongoing clinical programmes here.

Managed Access

There may be instances where a patient has a life threatening or seriously debilitating disease/condition and all available treatment options are exhausted and enrolment into a clinical trial is not possible. In such a situation, the patient’s treating physician may be able to request access to an investigational medicine prior to regulatory approval, provided this is permitted by applicable local laws and regulations.

Sobi has a process in place, referred to as “Managed Access Programmes” through which certain treatments can be made available to eligible patients, provided specific criteria and requirements are met. There are several different terms that fall within Managed Access, these include Compassionate Use, Named Patient Use, and Individual Patient Request amongst others. Terms used may differ from country to country depending on local laws and regulations.

Types of Managed Access in Sobi

Managed Access Cohort

This term describes the process by which an investigational medicine is made available to a group of patients, who have the same disease or condition, under a specific treatment protocol.

Individual Managed Access Requests

Where there is no ongoing Managed Access Cohort, Sobi may make an investigational medicine available to an individual patient, if the following eligibility criteria and additional requirements are met:

• The patient has a serious or life-threatening disease without a satisfactory alternative therapy or treatment and an unsolicited Managed Access request has been submitted to Sobi by the treating physician
• The patient is not eligible or able to enter an ongoing clinical trial
• There is sufficient data to indicate that the benefit of the investigational medicine outweighs the potential risks in the context of the disease or condition being treated
• There is a sufficient supply of the investigational medicine available and providing the medicine will not interfere with ongoing clinical studies or other development activities
• The patient meets any eligibility criteria set by Sobi’s Medical Committee evaluating Managed Access requests
• Laws and regulations in the country of request allow Managed Access

Please note that submitting a request does not guarantee that the request will be granted.

In evaluating and granting Managed Access requests, Sobi is committed to being fair, impartial, and transparent. Decisions are based on the clinical data currently available for the requested investigational medicine in the disease or condition in question.

It is important to remember that investigational medicines have not yet received regulatory approval; therefore, their potential risks and benefits may not yet be established. Physicians should consider all possible benefits and risks when seeking access to an investigational medicine for their patients.

Healthcare Professional request for Managed Access

Managed Access requests for Sobi's medicinal products must be submitted by the patient's treating physician. To submit a request, please enter the portal using the link below. If you wish to contact Sobi you can use the email address [email protected]. A committee of medical experts will evaluate the request based on the criteria and conditions mentioned above and communicate the decision to the treating physician. Sobi makes every effort to complete the review process within two business days after receipt of the request. If approved, a supply process will be initiated where the requesting physician may need to provide additional order-related information.