“In alignment with local regulations and laws, Bayer has put in place a policy governing the compassionate use of an investigational drug.”
- Dr. David Weinreich, Head of Therapeutic Area Oncology, Ophthalmology & Neurology, Bayer
The purpose of compassionate use programs is to provide patients who have an unmet medical need with a promising medicine which has not yet been authorized by the respective health authorities for the treatment of a serious, chronic, or life-threatening disease or condition.
Prerequisites for compassionate use
To be considered for inclusion in a compassionate use program, a medicinal product must have already demonstrated a favorable benefit/risk profile in preceding clinical Phase III studies. In addition, the medicinal product should be in the process of submission to a regulatory authority for potential marketing authorization. Local laws and regulations may govern the availability, administration, distribution and/or use of medicinal products prior to receiving marketing authorization. Finally, the severity of the disease or condition and the availability of alternative, effective therapies have an impact on whether a medicinal product will be made available prior to the marketing authorization.
Compassionate use at Bayer
Bayer, where permitted by local regulations and laws, will provide via the treating physician access to medicinal products prior to the marketing authorization for compassionate use through a number of mechanisms.
In the first place, patients should be considered for inclusion in a clinical trial before being offered compassionate use programs. A patient may only enter a compassionate use program if he/she cannot be included in a suitable clinical trial.
Bayer will consider access to an unauthorized medicinal product only when the following criteria are met:
- Sufficient clinical safety and efficacy data suggest a favorable benefit/risk profile for the proposed use.
- The patient(s) for whom treatment with an unauthorized medicinal product is sought:
- Is / are suffering from a severely debilitating or life-threatening disease which cannot be treated satisfactorily by an authorized drug in the country where the request has originated.
- Has / have received appropriate standard treatment without success (or no standard treatment exists for the patient’s condition) and no satisfactory alternative drug is available.
- Is / are ineligible or, due to geographical limitations, cannot participate in any ongoing clinical study.
- Meet(s) the inclusion criteria.
The treating physician must follow all applicable safety-reporting regulations of the respective country.
In the USA, there is a specific type of compassionate use program called the ‘Expanded Access Program’ (EAP).
For patients who cannot participate in a clinical trial of an investigational drug but have a serious disease or condition that may benefit from treatment with the drug, FDA regulations allow manufacturers of such drugs to provide those patients with access to the drug under certain situations, known as “expanded access".
In 2013, Bayer provided medicinal products through expanded access programs or compassionate use programs to over 4,000 patients suffering from serious, chronic, or immediately life-threatening diseases worldwide.