Title |
Innovative approaches to clinical development and trial design.
|
---|---|
Published in |
Annali dell'Istituto Superiore di Sanità, January 2011
|
DOI | 10.4415/ann_11_01_03 |
Pubmed ID | |
Authors |
John J Orloff, Donald Stanski |
Abstract |
Pharmaceutical innovation is increasingly risky, costly and at times inefficient, which has led to a decline in industry productivity. Despite the increased investment in R&D by the industry, the number of new molecular entities achieving marketing authorization is not increasing. Novel approaches to clinical development and trial design could have a key role in overcoming some of these challenges by improving efficiency and reducing attrition rates. The effectiveness of clinical development can be improved by adopting a more integrated model that increases flexibility and maximizes the use of accumulated knowledge. Central to this model of drug development are novel tools, including modelling and simulation, Bayesian methodologies, and adaptive designs, such as seamless adaptive designs and sample-size re-estimation methods. Applications of these methodologies to early- and late-stage drug development are described with some specific examples, along with advantages, challenges, and barriers to implementation. Because they are so flexible, these new trial designs require significant statistical analyses, simulations and logistical considerations to verify their operating characteristics, and therefore tend to require more time for the planning and protocol development phase. Greater awareness of the distinct advantages of innovative designs by regulators and sponsors are crucial to increasing the adoption of these modern tools. |
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Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 3 | 150% |
United Kingdom | 1 | 50% |
India | 1 | 50% |
Brazil | 1 | 50% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Researcher | 11 | 550% |
Student > Ph. D. Student | 7 | 350% |
Student > Bachelor | 5 | 250% |
Other | 4 | 200% |
Student > Doctoral Student | 2 | 100% |
Other | 6 | 300% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 20 | 1000% |
Agricultural and Biological Sciences | 6 | 300% |
Pharmacology, Toxicology and Pharmaceutical Science | 3 | 150% |
Business, Management and Accounting | 2 | 100% |
Engineering | 2 | 100% |
Other | 1 | 50% |