|
|
home > ebr > winter 2022 > pooling efforts to drive gene therapies for rare diseases |
 |
 |
PUBLICATIONS |
European Biopharmaceutical Review
|
When multiple organisations pool their efforts, it promotes affordability and return on investment. Gene therapies, which are expensive and complex to develop and study, and whose target populations are very small, are prime candidates for reaping the benefits of collaboration.
To facilitate cooperation, the US-based Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021, designed to encourage the delivery of more gene therapies for rare diseases. The consortium is headed and funded by the FDA and the National Institutes of Health. Other stakeholders range from large and small biotechs and pharma brands to non-profit organisations.
The Potential to Reduce Costs and Accelerate Approvals
The potential benefits of multiple stakeholders banding together could be significant and provide a model for other markets to follow. If a standardised platform can be agreed, with each drug developer sharing findings and building on what has gone before, the potential to reduce costs and accelerate approvals could be significant. As barriers crumble, new momentum might build as more pharma teams are able to see a clearer path to market. The ultimate beneficiaries will be patients, who could find themselves with access to breakthrough treatments to cure their condition, extend their life expectancy, or improve their quality of life.
|
Read full article from PDF >>
|
 |
 |
 |
Rate this article |
You must be a member of the site to make a vote. |
|
Average rating: |
0 |
| | | | |
|
|
 |
News and Press Releases |
 |
Clinerion adds Patient Network Explorer Coverage to three new Hospitals in Brazil
Basel, Switzerland, April 19th, 2022: Clinerion has extended its
patient coverage in Brazil to three new hospitals with the help of
partner iHealth Group. This raises the footprint of Clinerion’s Patient
Network Explorer to 22 healthcare organisations and over 8 million
patients in Brazil.
More info >> |
|

 |
White Papers |
 |
Clinical Trials in Russia Orange Paper: Summary of Year 2013
Synergy Research Group
Russia remains a very popular geography for local, regional, and global pharmaceutical companies to conduct clinical trials.
Sponsors mention the following reasons for conducting studies in Russia:
1. Fast patient enrollment due to the centralized medical infrastructure.
2. Nearly 100% patient retention
3. GCP trained and certified Investigative Sites generating high quality data
4. Low cost: Average per patient cost is 60% to 70% below US and European prices due to the low cost of Investigators and the high concentration of patients in therapeutically aligned medical centers
More info >> |
|
|