Compassionate Use – help is at hand!
When all approved therapies have been exhausted and no satisfactory or alternative therapy options remain for a patient with a serious or immediately life-threatening medical condition, the patient’s last path available could be to use an unapproved treatment. Receiving this treatment without participating in a specific clinical trial can be achieved through the Expanded Access or Compassionate Use program, although it is also referred to by many other names throughout the US and Europe.
Access to treatments that do not have a marketing authorisation is handled on a country-by-country basis. The FDA has provided regulations and the EMEA has issued guidelines for national regulatory agencies to follow. However, as these are only EU guidelines, each European country has its own access laws which differ from each other.
It can be difficult to find the regulatory pathway relevant to a patient´s needs but the one thing that remains constant among the differing requirements is that the authorities will need several documents to be written and submitted. These documents are time-consuming to be both written and reviewed and, in the case of patients with an immediately life-threatening condition, this is valuable time that cannot be spared.
In order to be eligible to receive an IMP through expanded access, patients need to fulfil strict criteria. Sufficient information also needs to be provided on the treatment being requested and the patient’s medical condition so that the regulatory authorities are able to judge whether the possible benefits of the drug or device outweigh any risks.
In cases like these, where the time constraint is acute, hiring a medical writing service provider accelerates the mechanism to receive the go-ahead. Writing professionals know how to research the information required, how to synthesize the essential elements into the appropriate forms, letters, dossiers and other documents (the requirements of which differ by country) and prepare it for submission as efficiently as possible.
EMEA: COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP). GUIDELINE ON COMPASSIONATE USE OF MEDICINAL PRODUCTS, PURSUANT TO ARTICLE 83 OF REGULATION (EC) No 726/2004. Ref: EMEA/27170/2006. Click to read [PDF] (Accessed: 25 November 2019).
FDA: Individual Patient Expanded Access Applications: Form FDA 3926 Guidance for Industry. 2017. Click to read [PDF] (Accessed: 25 November 2019).
Documents need to be written | Europital can write them for you
Contact our Senior Medical Writer, to learn more about how we can help you with all your Medical Writing needs.