Your doctor may contact the company to verify if the medicine is available for compassionate use

The process to follow when no one has requested the medicine on a compassionate use basis before

Your doctor may be aware that a new medicinal product for your disease is being tested via clinical trial. Depending on the criteria to participate in the clinical trial and/or your health status, your doctor may think it is best for you to receive the experimental treatment on a compassionate basis.

Patients can also initiate the process to access a Compassionate Use Programme by informing their doctor that a new medicinal product could provide a promising alternative to treat their condition. Thoughnot yet fully evaluated, if it fulfils the criteria, the treatment could be eligible for a CUP. This type of scenario often occurs when patients’ advocates are working with the companies that develop medicines for their disease and are well informed of research in progress.

Your doctor may then contact the National Competent Authority and ask if there is already a Compassionate Use Programme for this medicine for your disease. If there is none, someone must initiate the discussions with the company developing the medicine: it is often a treating physician who contacts the company asking if and how a treatment could be made available on this basis.

  • The company calculates how much product is manufactured (it has to be a commercial batch, that is, no longer a pilot product produced in small quantities in a laboratory but batches of product of the same quality as batches that will be placed on the market, should a marketing authorisation be granted. Therefore manufacturing sites have to be active and validated. There are exceptions to this, e.g. some advanced therapies or for ultra-compassionate use).
     
  • The company then calculates how much product is needed for current and future clinical trials, and secures some supply for trial participants who may ask to access the product when the clinical trial is over, if they wish to continue receiving the treatment, or start receiving it if they were on the placebo arm or treated with a comparator regimen.
     
  • The product that remains can be allocated to a Compassionate Use Programme. Based on the average daily dose, the company estimates how many patients could be treated on a compassionate basis and decides how many treatments can be allocated to different countries.
     
  • The decision to organise a Compassionate Use Programme is not based only on the availability of a certain stock. It is a strategy decision. Not all companies are willing to satisfy the demand from patients in greatest need of new options. For example, the company might fear that the most advanced patients will not greatly benefit from the product: if the risk of imminent death is already high, the product may not help increase survival and the Compassionate Use Programme might give the product a poor “reputation”.
     
  • If the company does elect to make available a product for compassionate use,  the company can respond to the doctor: “We can provide treatment for X many patients in your country, of which we can provide Y to you”.
     
  • The company then contacts the National Authority and submits a dossier to obtain a Compassionate Use Programme authorisation (sometimes it is up to the treating physician to submit this request and then the national authority contacts the company in question).
     
  • This usually means an import authorisation for a not yet authorised product needs to be decided by regulatory authorities.

 

Example
Total production/year From 250 to 1000 kg
Average dose / patient  1mg / day or 365 mg /year
Maximal number of patients enrolled in clinical trials 600

 

simulation of the calculation of the amount of product available for a Compassionate Use Programme over the next four years when production capacity is largely increased

Simulation of the calculation of the amount of product available for a Compassionate Use Programme over the next four years when production capacity is largely increased. This information is important for regulatory authorities and also for patients’ organisations who engage in discussions with a company to understand when a CUP could be started.

  • Year 0: Discussions for a CUP start. Production capacity: 250 kg or enough to treat 685 patients for one year. At this stage, 200 patients are enrolled in clinical trials receiving the medicinal product (or 73 kg). Therefore, 612 kg are available but not used (representing 485 treatments). They are stock-piled for new clinical trial participants to be enrolled the next year. No patients are enrolled in the CUP as it has not yet started.

  • Year 1: Production capacity unchanged (250 kg). With this production and the stock available from the previous year, a total of 600 patients receive the product in clinical trials, 330 are enrolled in a CUP, and enough treatment for 240 patients is stock-piled for the next year.
     
  • Year 2: Production increases to 500 kg. With this production and the stock available from the previous year, a total of 900 receive the product in clinical trials or their follow-up, 90 new patients can be enrolled in the CUP (total number in CUP: 420 patients), and enough treatment for 290 patients is stock-piled for the next year.
     
  • Year 3: Production remains at the same level, 500 kg. With this production and the stock available from previous years, a total of 1100 receive the product in clinical trials or their follow-up, 140 new patients can be enrolled in the CUP (total number treated in CUP 560 patients), and no more treatment is stock-piled for the next year. This means year 3 is the most difficult year; everything that is produced or that has been stock-piled from previous years is used, no with stock remaining or carried forward.
     
  • Year 4: Production increases to 1 tonne. With this production and the stock available from previous years, a total of 1200 receive the products in clinical trials or their follow-up, 140 new patients can be enrolled in the CUP (total number in CUP: 700 patients), and enough treatment for 839 patients is stock-piled for the next year. If more than 140 new patients asked to be enrolled in the CUP, this now becomes possible: all clinical trials are now ended, and all trials’ volunteers are receiving treatment in a follow-up phase. All stock that remains can now be allocated to the CUP.

 

This is the general framework. Sometimes the patients' organisation first talks with the national authority asking  "What would you think of a CUP for this product? We think it is most needed". The agency evaluates the needs and the product, and can decide to authorise the CUP, and then informs all relevant doctors that the CUP is open.

Further reading: example of an application form to create a Compassionate Use Programme (from the French Agency, in English).

 

Form to submit an A.T.U application
http://ansm.sante.fr/Activites/Autorisations-Temporaires-d-Utilisation-ATU/ATU-Avis-aux-demandeurs-formulaires/(offset)/6

 

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