What a Compassionate Use Programme isn’t

By definition, a compassionate use is a treatment, yet not fully evaluated, for patients who do not have other options. Other circumstances when a medicine is given should not be confused with compassionate use.

  • Neither a clinical trial nor an experiment: in no case can the medicinal product given on a compassionate use be a placebo, nor a treatment or combination of treatments that the patient has had before, lacking efficacy or poorly tolerated. Even though it is not completely certain whether the treatment the patient receives on a compassionate use basis is useful, if there is no other option and there is the slightest chance that the product will be effective, the patient should benefit from it.
     
  • A substitute for product development: a Compassionate Use Programme cannot replace clinical trials. Again, a CUP is not an experiment. It cannot conclude whether the product is safe and effective on a group of patients. A CUP can be organised in parallel to clinical trials, but it can only be authorised if clinical trials are already in progress, and recruitment in a CUP should not be an obstacle for the recruitment of patients in clinical trials (for example if patients prefer to receive the medicine on a compassionate basis and not take the risk of receiving a placebo).
     
  • An off-label use: a medicine used off-label is, by definition, a medicine used in a different way than the authorised one. For example, a medicine can be given for a different disease, or at a different dose than indicated on the label. Thus off-label use only applies to medicines that are already authorised. Compassionate use only applies to medicines that are not yet authorised for any condition.
     
  • A financial aid programme or a humanitarian programme: financial aid programmes (also called humanitarian programmes) are for medicines that are authorised and placed on the market, but at a cost that the patient cannot afford (for example medicines that are not 100% reimbursed or covered by the health care system). In some cases, the marketing authorisation holder creates a financial assistance programme for patients who do not have enough revenue. This is not compassionate use; it is a marketing tool to open access to a larger group of patients.
     
  • A way to place a product on the market prior to marketing authorisation: some companies can be tempted to provide a medicine they develop to a large group of patients, prior to its authorisation. This is to make sure the medicine is already largely used, at an early stage, to “capture” a large market share and make it more complex for competitor products to make their own market share. This is why a CUP needs to be properly authorised and monitored by national competent authorities. A CUP cannot be used for investigational purposes or commercial pre-authorisation activities. Promotion of the medicinal product in question, or the CUP itself, is not permitted.
     
  • A “favour” or a “gift” to clinicians who achieve their objectives in recruiting for clinical trials: completing enrolment of patients in a clinical trial is a key factor of success for a clinical trial, and thus for the product development and evaluation. Clinicians who are also investigators of clinical trials are precious to the trial sponsor, e.g. a pharmaceutical company, as they can ask their own patients to participate in a clinical trial. In a very competitive world, a company can be tempted to gain the favours of clinicians by offering them access to the medicine on a compassionate basis if they manage to enrol high numbers of patients. This practice is not official but does exist, and is not recommended as it creates inequity in accessing the medicine on a compassionate basis.

Page created: 30/10/2013
Page last updated: 07/11/2014
 
 
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