Compassionate Use

We understand that individuals with rare diseases do not have many treatment options available to address their medical needs. At Healx, we are committed to developing safe and effective therapies for rare diseases and providing those therapies to patients as quickly as possible. As part of that commitment, we support expanded access and compassionate use programmes.

Healx has a transparent process for determining whether it provides experimental therapy under compassionate use.

The company will evaluate whether:

• there is substantial scientific evidence to support both the safety and the efficacy of a product for an indication, typically after positive clinical data is available;
• it has been established that access on a compassionate use basis will not compromise clinical trials or the regulatory pathway;
• the product is intended to treat a serious or life-threatening disease, and existing approved therapies are not appropriate or effective, such that consideration of using an unapproved therapy is justified;
• there is adequate supply of the experimental therapy; and the compound can be administered – and it is logistically feasible to make it available – outside of the clinical trial setting.

If Healx decides that compassionate use is appropriate and should be available for a product, then the company evaluates an individual’s request for compassionate use using the following criteria:

• the patient’s underlying medical conditions do not pose safety risks that have not been sufficiently studied and there is sufficient evidence to believe the benefit to the patient justifies the risk;
• the request has been made by a qualified and licensed physician with expertise and facilities appropriate for the administration of the investigational therapy, monitoring, managing and reporting side effects, as well as patient experience;
• the patient’s condition is of a type and stage commensurate with the indication for which the investigational therapy is likely to be approved;
• there are no satisfactory alternative treatments (confirmed by the HCP);
• the patient does not qualify for participation in ongoing or pending clinical trials; and
• all necessary regulatory/institutional approvals have been obtained to allow the administration.

Requests for access to experimental therapies must be made by a qualified and licensed physician via Healx’s Contact Us page. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 5 business days after receipt.

For more information on each of Healx’s current clinical trials, please use the drop down under the Programmes tab at the top of our website. The linked webpages also include links to clinicaltrials.gov, the US Government website containing additional information about Healx’s current clinical trials.

This page was last edited June 2022.