Experimental treatments outside of clinical trials
Experimental treatments provided to patients within the controlled environments of clinical trials are scientifically sound. But what about experimental treatments outside the regulated framework of clinical trials? Options and frameworks exist for patients in specific circumstances, as long as the need is justified and the treatment is supported by scientific evidence.
Off-label use of authorised drugs
When approved drugs are used for an unapproved indication, age group, dosage or route of administration, this is considered off-label use. It is justified if there is scientific evidence that the approved drug is safe and beneficial to patients suffering from an illness or disease that the drug is not approved for, or if no other satisfactory treatment is available for a patient.
Investigational medicinal products
The use of experimental products that are not yet approved or licensed is justified if there is scientific evidence that it is safe and likely beneficial to patients, if its production complies with the Good Manufacturing Practice guidelines, or if no other satisfactory treatment is available for a patient.
Clinical implementation of investigational medicinal products depends heavily on individual treatment centers' policy and national legislation.
Delay between trial and drug availability
There is a time gap between clinical trial and market availability of a drug. To ensure patients have access to experimental, not-yet-authorised or marketed treatments supported by scientific proof of efficacy and safety, frameworks such as medical need programs and compassionate use programs are in place.
Medical Need Program
For medicinal products authorised in Belgium, this program is available in the case of serious chronic or life-threatening diseases untreatable by an authorised and/or commercially available product. A medicinal product can be subject of a medical need program as long as a demand to obtain market authorisation for the product indication in question is in process, the indication has been authorised but the product is not yet commercially available, or clinical trials are ongoing in this indication.
Compassionate Use Program
Unauthorised medicinal products are sometimes authorised to use out of compassionate reasons, upon request of individual pharmaceutical companies. Patients with a debilitating or life-threatening disease that cannot be treated by an authorised product can use an unauthorised medicinal product, as long as the product has applied for marketing authorisation or is currently undergoing clinical trials for the indication.
Beware of questionable experimental treatments
Some private clinics and physicians provide off-label and investigational drugs outside of a clinical trial that are not supported by scientific evidence and/or not compliant with GMP regulation. There is no guarantee that patient rights, safety and well-being are respected by these treatments.