Increasing the development and patient access to advanced therapy medicinal products

The European Medicines Agency (EMA) has issued ideas to improve the development of advanced therapy medicinal products (ATMPs) and their availability to patients. These ideas were developed following a multi-stakeholder expert meeting, held last year, which looked at how to drive innovation, research and development of ATMPs.

ATMPs are medicines based on genes or cells that offer ground-breaking new opportunities for the treatment of disease and injury. These types of medicines can be categorised into four main groups:

  • Gene therapy medicines which contain genes that result in a therapeutic, prophylactic or diagnostic effect. They work by inserting ‘recombinant’ genes into a patient’s body.
  • Somatic-cell therapy medicines which contain cells or tissues that have been altered to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body.
  • Tissue-engineered medicines which contain cells or tissues that have been changed so they can be used to repair, regenerate or replace human tissue.
  • Combined ATMPs which include one or more medical devices as an integral part of the medicine.

 

These kinds of treatment hopefully have a vital role to play in overcoming for severe, chronic diseases for which conventional medicines have not worked.

To read the EMA publication, please click here.