Compassionate Use

Prothena’s mission is to create novel protein immunotherapies to transform patients’ lives.

We are committed to developing safe and effective therapies for patients, and strive to put patients first. Our goal is to provide first in class therapies to patients as soon as possible through thoughtful and efficient drug development.

Prothena has received a number of requests for compassionate use of our investigational drugs, and recognizes the importance of developing a Compassionate Use Policy.

We support the need for compassionate use and expanded access programs and our goal is to provide study drug access at the appropriate time and in the correct context for patients with life threatening diseases.

We consider a number of factors when determining whether or not to support compassionate use for a given investigational drug:

  • Will it jeopardize the ongoing development work that Prothena is conducting to bring a therapeutic to market as quickly as possible and to as many patients as possible?
  • Is there adequate drug supply for both the clinical development program (current and future trials) and broad compassionate use? To be equitable, the company should endeavor to provide study drug to all qualified and equally compelling compassionate use patients if it provides study drug to any one patient.
  • Is there enough evidence to reasonably assess that the drug will be safe and effective so that it can be provided to a significant number of patients in an environment that is less controlled than a sponsored clinical trial?
  • To meet regulatory requirements, will the safety data be adequately provided to Prothena by a local physician outside of a sponsored clinical trial?

Prothena has determined, after assessing these considerations, that compassionate use cannot currently be offered for any of its investigational drugs. We will be re-evaluating this policy periodically.

If and when Prothena provides compassionate use for a particular investigational drug, the company will review each individual request for compassionate use and respond to the physician who makes the request. During this process, Prothena will need to ensure that:

  • Granting access will not interfere with current clinical trials or regulatory filings.
  • The patient does not qualify for any ongoing or planned clinical trials.
  • The request is made through a clinician expert in the therapeutic area who has weighed the benefit and risk of providing the investigational drug to the patient.
  • The patient has exhausted other therapies that may help him/her.

The above are general requirements, and specific requirements will be generated for a given investigational drug or clinical program if and when Prothena initiates a compassionate use program.

If you have questions about Prothena’s Compassionate Use Policy, please contact Prothena’s medical information at medicalinfo@prothena.com.