The global biopharmaceutical industry currently has a total of 5,408 new medicines in clinical development, of which 70% are potentially first-in-class treatments, says a new report.The present pipeline also includes nearly three times as many drugs for rare diseases and conditions as was the case a decade ago, averaging 140 per year in the last 10 years from 64 in the previous decade, says the study, from Analysis Group. And researchers are now actively studying diseases and conditions with no recent approvals, including 61 projects for amyotrophic lateral sclerosis (ALS), 158 for ovarian cancer, 19 for sickle cell disease, 41 for small cell lung cancer and 26 for septic shock, it adds.
Personalised medicines are a growing proportion of the pipeline, Analysis Group also points out. Biomarkers were found to be employed in 155 clinical trials initiated before January 2009, it says, and cites a 2010 survey of biopharmaceutical companies by the Tufts Center for the Study of Drug Development (Tufts CSDD) which found that 12%-50% of drugs were taking this approach and forecast a sharp expansion in this area going forward, as respondents to the survey anticipated a 50% increase in investment in personalised medicine R&D during 2010-2015."With three new personalised medicines approved in 2011 and 2012, personalised medicine is beginning to bear fruit and offers great promise for patients," says Analysis Group.
Of the 5,408 new products currently in clinical development worldwide, 2,164 are in Phase I, 2,329 are in Phase II and 833 in Phase III, estimates the report, which was supported by the Pharmaceutical Research and Manufacturers of America (PhRMA).
Potential first-in-class treatments account for 78% of all products currently in Phase I, 69% in Phase II, 45% in Phase III and 51% of those now undergoing regulatory review, it goes on, noting that there are particularly high percentages of potential first-in-class medicines in neurology (84%), psychiatry (80%), cancer (80%) and diabetes (79%).
As of December 2011, the total number of "projects" in development worldwide was close to 8,000; the report defines such “projects” as new molecule-indication combinations, which may be new molecular entities (NMEs) or new indications for medicines previously approved by the US Food and Drug Administration (FDA). There are currently 3,070 such projects for new cancer treatments, while for other therapeutic areas, the projects total: infections - 750; neurology - 610; immunology - 298; cardiovascular - 450; diabetes - 281; psychiatry - 240; and HIV/AIDS -185.
The study also examines novel scientific strategies now being used in R&D, focusing on the following innovative platforms:
- antisense RNA interference (RNAi), a new strategy that targets RNA in order to silence gene expression. While most drugs target proteins such as enzymes and cellular receptors, this new approach opens up RNA, which carries genetic information to create proteins, as a new potential target for drugs. Thus far, two RNAi therapeutics have been approved and 127 more are in development;
- therapeutic cancer vaccines, which harness the immune system to fight off disease that is already underway. The first therapeutic cancer vaccine was approved in 2010, and 20 more are currently in development; and
- there are 245 projects in development using cell therapy, 99 using gene therapy and 102 utilising conjugated monoclonal antibodies to target and kill tumours while sparing nearby healthy cells, it says.