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Productivity & Standards
In the area of better evaluation tools, the Critical Path is keenly on developing improved methods for assuring product safety before
interested in new biomarkers and disease models. Biomarkers, or human testing begins.
measurable characteristics that reflect physiological, pharmacological
or disease processes in animals or humans, are of particular value in Of course, there are obstacles on this path, including a lack of funding,
clinical trials because they can help predict adverse reactions to the difficulties competitors have sharing data with each other,
therapeutics and provide early assessments of the immunogenicity, scepticism that the initiative will lead to burdensome new
cardiac toxicity and liver or kidney toxicity of candidate drugs. requirements and the concern that the FDA may have to divert
resources from ongoing product reviews to meet Critical Path goals.
As an example of these evaluation tools, the FDA has recently However, substantial resources have already been invested in this
approved several genomic tests for drug-metabolising enzymes – important undertaking, and some successes have been realised.
assays that can identify patients who are at risk of serious toxicity from
cancer therapies because the recommended doses are too high for According to FDA materials about the initiative, one success story of the
them. However, the FDA does not have the resources to develop these Critical Path philosophy in action involves facilitating efficiency in vaccine
tools; the innovations must come from industry. manufacture to help defeat meningococcal disease in Africa. FDA science
that represents a breakthrough method for conjugation in vaccines was
provided to a manufacturer, the Serum Institute of India, through a
technology transfer agreement and through FDA training of the
institute’s personnel. Thanks also to a European manufacturer supplying
the necessary polysaccharide for the vaccine at low cost, an estimated 25
In the area of better evaluation million doses of vaccine per year will be available beginning this year at a
tools, the Critical Path is keenly
production cost of less than 50 US cents per dose.
interested in new biomarkers and
Work to gauge the efficacy of digital mammography for breast cancer
disease models.
screening is another success story touted by the agency. For this effort,
the FDA was instrumental in developing and implementing a more
flexible and innovative approach to the large clinical trials needed to
evaluate medical screening devices. The agency proposed that four
companies working on the technology each carry out a study of
10,000 patients using a common protocol, and that the propriety
Another point of concern for the overall initiative was well stated in a results be pooled for analysis without the companies’ devices being
2004 letter to the FDA from Robert J Beall, PhD, President and Chief compared with each other. Similar protocols could be developed and
Executive Officer of the Cystic Fibrosis Foundation, in response to the applied in future cases where the size of trials needed to establish a
Critical Path report: “Increasing international harmonization on device’s effectiveness is beyond the range of individual companies.
safeguards, processes, and data collection for clinical trials is critical for
efficient, timely drug approval, particularly for orphan diseases.” Indeed, As these examples amply demonstrate, industry ultimately must buy
an increase in the amount and quality of clinical trials data from outside into the idea that treading the Critical Path is a worthwhile endeavour
the US, especially in universally shared formats that make interpretation and be willing to invest the time and collaborative efforts needed to
and application of results far more efficient than is typically the case ensure its success.
now, would be a boon to many crusades against health crises that are
international in scope. A welcome inclusion among the Critical Path
Opportunities, then, is an initiative known as Clinical Data Acquisitions
Standards Harmonization, which targets improvements in the clinical
Increasing international harmonization
trial data collection environment for investigative sites, with the goal of
increasing recruitment of new investigators and subjects by
on safeguards, processes, and data
implementing data collection standards globally.
collection for clinical trials is critical
for efficient, timely drug approval,
Thus, the Critical Path Initiative is a collaborative effort. One specific
example of the many partnerships that are blazing the trail is the particularly for orphan diseases.
Oncology Biomarker Qualification Initiative, a public/private team
spearheaded jointly by the FDA, the National Cancer Institute and the
Centers for Medicare and Medicaid. The goal of this effort is to
develop project concepts, standards, nomenclature and other tools to We are all of us – manufacturers, researchers, volunteers, regulators,
improve the clinical utility of biomarker technologies in cancer reviewers – on this new path together because it became abundantly
treatments. Another example is the FDA-supported Predictive Safety clear that the path we were on for much of the past decade was not
Testing Consortium, which was founded by the Critical Path Institute taking us where we wished to be. Putting safe and effective drugs,
and includes eight major pharmaceutical companies: Bristol-Myers biological products and medical devices into widespread use more swiftly
Squibb, Johnson & Johnson, GlaxoSmithKline, Merck, Novartis, Pfizer, and efficiently is a goal that should make the many lessons learned along
Roche and Schering Plough Research Institute. The consortium focuses the way – be they hard or easy – well worth the journey. ■
8 DRUG DEVELOPMENT 2007
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