spacer
home > ebr > autumn 2017 > risk ratio
PUBLICATIONS
European Biopharmaceutical Review

Risk Ratio

On 30 August 2017, the US FDA formally approved the application for Kymriah, a cancer immunotherapy utilising modified human T-cells that express chimeric antigen receptors (CAR-T). This momentous event follows a decade of intense work to commercialise a therapy that was first demonstrated in 2011 in a handful of terminal paediatric leukaemia patients. At this moment, many CAR variants are in development, alongside a biotechnology business frenzy.

Pre-genomic immunotherapy consisted of human immunoglobulin injections to boost immunity during disease outbreaks. Clearly, this was a blunt instrument. Half a century later, a group at the University of Pennsylvania described remission in paediatric patients with refractory chronic lymphocytic leukaemia using genetically modified immune cells. Furthermore, at the beginning of September 2017, the first ever approval of CAR-T therapy was granted by the FDA for cases of acute lymphoblastic leukaemia in children and young adults. This inverts the usual regulatory paradigm by approving paediatric indications first.

In their Harvard Business Review paper ‘How to Spot a Technological Winner’, Graham and White discuss the factors that influence the success of a transformative innovation. The present intensity of the commercial race in cancer immunotherapy and gene-editing reflects a belief within the biopharmaceutical community that the trade-off of business drivers and technical hurdles is favourable. In just a few years, the number of groups working in this field has expanded from just a handful to over 200.

Read full article from PDF >>

Rate this article You must be a member of the site to make a vote.  
Average rating:
0
     

There are no comments in regards to this article.

spacer
Emile Bellott is a member of the EBR Industry Advisory Board and an industry consultant with experience in drug discovery, development and pharmaceutical outsourcing.
spacer
Emile Bellott
spacer
spacer
Print this page
Send to a friend
Privacy statement
News and Press Releases

Saudia Cargo launches state-of-the-art pharmaceutical cold storage facility at KAIA

MAY 2018 – Saudia Cargo has recently inaugurated one of the Region’s newest and most modern cold storage facilities of pharmaceuticals and medicines at King Abdulaziz International Airport (KAIA) to meet the surged demand on highly-sensitive and temperature-controlled shipments.
More info >>

White Papers

Points to Consider When Developing a TMF (Trial Master File) Strategy

Phlexglobal Ltd

Many organizations are currently outsourcing clinical trial activities to one or more contract research organizations (CROs). This strategy enables companies to leverage specialized expertise and take advantage of flexible resourcing throughout the conduct of a clinical trial. Outsourcing minimizes the costs of recruiting experts, building a team and maintaining an infrastructure. However, it can also add complexity as the organization looks to meets its compliance obligations regarding clinical trial documentation. The documentation referred to in Article 15(5) of Directive 2001/20/EC as the trial master file shall consist of essential documents, which enable both the conduct of a clinical trial and the quality of the data produced to be evaluated.1 This essential study specific documentation is also known as the TMF. As organizations try to minimize their reliance on paper files, the electronic TMF (eTMF) has emerged. A current industry initiative to standardize the organization of this content is known as the TMF Reference Model. This model is helping standardization efforts across paper and electronic systems. As companies implement outsourcing strategies, CROs and sponsor organizations look for a common foundation on which to build their TMF capabilities. The following paper outlines some of the challenges organizations face when outsourcing clinical trial activities to multiple contract research organizations and a strategy to facilitate partnering and management of trial information between sponsors and CROs.
More info >>

 
Industry Events

Clinical Innovation Partnerships 2018

3-4 July 2018, Radisson Blu Hotel, Zurich Airport

Clinical Innovation Partnerships is the only event for senior clinical operations and procurement professionals driving innovation in their clinical trials. It is where the pharma, CRO and vendor clinical trials ecosystem meet to drive innovation in digitalisation, tech and partnering to improve patient experience and reduce trial cost and time.
More info >>

 

 

©2000-2011 Samedan Ltd.
Add to favourites

Print this page

Send to a friend
Privacy statement