Developments, news and strategies for drug development specific to phase I through Phase III global clinical trial management, execution, project management and outsourcing. Go→
News, articles and issues specific to clinical trial practice and implementation at the investigative site level. Go→
News, developments and strategies related to eClinical, data management, data collection, ePRO, and more information technology used in the drug development chain. Go→
News, articles and issues specific to laboratories role in the clinical trial, including ECG, imaging, genotyping, tissue samples and more. Go→
News, developments and strategies for clinical trials conduct in relation to the FDA, EMEA and other global regulatory authorities overseeing the drug development industry. Go→
News, articles and strategies related to clinical trial design which impact postmarketing studies, therapeutic areas, adaptive trials, statistics, protocols and more. Go→
Biopharmaceutical companies are touting their huge investment in R&D, which has filled the drug pipeline with more potential first-in-class medicines, including orphan drugs, personalized medicines and new therapies based on novel scientific strategies. A report by the Analysis Group for the Pharmaceutical Research and Manufacturers of America (PhRMA) documents more than 5,000 new medicines in the pipeline globally, many for untreated diseases and life-threatening conditions. The promise is that this more robust pipeline will lead to more new critical therapies for patients.
Yet, the data also reveals that most of these therapies are in early stages of development: less than 1000 of the 5400 products in clinical development have reached stage III, and only 82 are headed for market following approval by the Food and Drug Administration. The attrition from phase II to phase III remains very high despite a range of scientific advances and regulatory improvements. Of nearly 3000 new molecular entities (NMEs) to treat cancer, only 288 have reached stage III clinical trials, and only a handful make it to market. Therapies for infectious diseases seem to have a higher success rate, with about 700 investigational projects yielding 22 recent approvals. Certainly a higher “early kill” rate may be a sign of progress in the risky world of pharma R&D, where a key goal is to avoid costly phase III studies that won’t pass muster with FDA. The current study doesn’t provide the historical data that could tell more about whether pharma R&D is becoming more productive, but there doesn’t seem to much evidence of progress.
A more telling sign is the recent rise in FDA’s approval numbers for NMEs, reaching almost 40 in 2012 and still providing steady good news for sponsors. The promise is that more INDs eventually will yield more new approved medicines. “There are no guarantees” from a stronger and more diverse pipeline, but the study reveals the “depth of possibilities,” observed Genia Long of the Analysis Group in a PhRMA webinar. However, it also is important for pharma to reduce the overall cost of developing new drugs, and that will require more informative early stage research strategies that efficiently separate potential winners from likely losers. Researchers are making progress in developing treatments for Alzheimer’s disease, pointed out Eli Lilly CEO John Lechleiter, acknowledging that success “will require longer, more expensive studies to show benefit for patients.”
The PhRMA report aims to demonstrate to the public and policymakers the high value of biopharmaceutical R&D and the importance of continuing government support for FDA programs and research funded by the National Institutes of Health. It also highlights the value – and the need to pay for – new therapies to treat rare diseases and conditions that currently lack effective treatment. Ultimately, these new, costly research endeavors could lead to cures and preventives for cancer, Alzheimer’s and other devastating illnesses that affect millions. But the costs for individual patients may be enormous, and it remains to be seen if public and private health programs will pay for them.