Developing Cell Therapies: Enabling cost prediction by value systems modeling to manage developmental risk.
Requires Subscription or Fee PDF

Keywords

Commercialisation
Cellular Therapeutics
Regenerative Medicine
New Product Development

Abstract

This work quantifies the highest risk activities and interdependencies in cell therapy new product development (NPD).  A simulation model based upon an activates based and information driven  approach of the Design Structure Matrix (DSM), using Latin Hypercube sampling methods with discrete event simulation evaluated the interdependencies between critical development tasks.  Input data was collected from quarterly financial reports of cell therapy developers and developmental milestones as reported in company press releases and publications.  .

Successfully planning and managing development processes is problematic in an emerging industry lacking precedents and standardised technology platforms.   Methods of understanding and reducing developmental uncertainty and risk are needed to aid resourcing decisions.  A particular requirement is to understand the impact of process and clinical development, in this highly regulated sector.

Results from the model quantify the probability and impact of process iterations and failures that impact cost and duration of cell therapy NPD.  High impact areas quantified are the interdependence of Phase 1 clinical trials and investment, the scaling of the manufacturing process from Phase 1 to Phase 2 and Phase 2 to Phase 3.  The model also allows for the calculation of the probability of NPD success for given resource levels, time constraints and market conditions.  An application comparing alternative regulatory approaches indicates that the current favoured strategy of targeting an orphan indication gives little benefit for the tested clinic al indication because of reduced clinical trial recruitment rate.  While specifically developed for cell therapy NPD this modelling approach has potential application across the wider biotechnology industry.

https://doi.org/10.5912/jcb585
Requires Subscription or Fee PDF

Unless specified by prior arrangement, the author agrees to the following terms and assurances:

  1. For myself and on behalf of the other authors listed on this work, I assign to thinkBiotech LLC the copyright* in the contribution for the full term throughout the world.
  2. I/we further give to the following assurances
    1. I am the sole author of the contribution, or, if not, I have the written authority of the other authors to transfer the copyright* to thinkBiotech LLC and give these warranties;
    2. I and (where appropriate) the other authors are entitled to transfer the copyright to thinkBiotech LLC and no one else would be entitled to prevent us from publishing the contribution;
    3. To the best of my/our knowledge, all the facts in the contribution are true and accurate;
    4. The content of the contribution is entirely original to me (and where appropriate to the other authors) or, if not, the written permission of the owner of the copyright in any material copied from elsewhere has been obtained for all media (all such permissions to be attached to the contribution as supplementary files);
    5. Nothing in the contribution is obscene or libellous;
    6. Nothing in the contribution infringes any duty of confidentiality which I/or the other authors may owe to anyone else.
    7. I and/or the other authors have obtained the appropriate clearances from my/our employer(s) or other concerned institution(s).
* Works by US government employees prepared as part of official duties are in the public domain and the authors are therefore exempt from copyright assignment.