Technology Forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption

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Research areas: High tech medicine, HTA-methods & steering instruments

Project leaders: Claudia Wild

Project team: Claudia Eder
Duration: August 2018 – November 2018
Language: English
Publication: Article

Background:
Advanced Therapy Medicinal Products "(ATMP) is the umbrella term for three drug product classes, somatic cell therapies, gene therapeutics and engineered tissue preparations [also called tissue engineering products (TEP)], as well as combination products. These drugs usually contain or consist of living cells or tissues and are therefore characterized by a high degree of complexity. The cells used are often taken from a patient, processed in the laboratory (eg, replicated or genetically engineered), and then re-administered to the same patient. The field of work of the ATMP is still young and there is still limited regulatory experience in the approval: The individual production in often small quantities leads to a more complex situation in the licensing and corresponding clinical studies (designs). The development of ATMPs is mostly characterized by a strong experimental nature and often originates in the academic research environment such as universities and their clinics.

Project objectives:
The project objective is to give an overview of applications of ATMP products shortly before or after market launch.

Methods:
Systematic literature search incl. “grey literature” and database reviews as well as manual search following pre-defined search term. Searches (selection) in

Presentation of results acoording to

  • Approved by the EMA
  • In late clinical trial
  • Applied via hospital exemption

Inclusion criteria:

  • Application: Human medicine
  • Interventional product classified as ATMP or allegedly classified as ATMP
  • Language of publication English or German
  • Intervention falls under ATMP definition
  • Intervention takes place within an EU member state
  • In case of clinical trials: Phase III or IV

Exclusion criteria:

  • Publications on basic research without direct clinical application
  • Articles targeting animal experiments without direct clinical application
  • Articles in languages other than English or German
  • Articles not publicly available

Result: Short report of methods and results, in form of a publication

Milestones: Manuscript written by end November 2018

References:

  1. Falkner, E., Wild, C. (2008): Clinical application of Tissue Engineering: An overview of International and Austrian Areas of Research with a critical analysis of selected applications. LBI-HTA Projektbericht 013. https://eprints.aihta.at/807/1/HTA_Projektbericht_013.pdf
  2. Directive 2001/83/EC amended by the ATMP Regulation 1394/2007: REGULATION (EC) No 1394/2007 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004