Turning the genomics vision into reality
|
In April, scientists worldwide celebrated the 50th anniversary of the discovery of the double helix by announcing the successful
completion of the Human Genome Project (HGP). As they unveiled the nearly finished version of the human genome sequence, leaders
of this landmark project outlined plans for tackling the next stage: translating genomic research into medical treatments
to improve public health. This will involve identifying variations in DNA sequences that contribute to disease and defining
how the proteins produced by each gene control cellular operation.
HGP accomplished a great deal beyond producing a 99.9% accurate sequence. It mapped the genome of comparative organisms (rat
and mouse) and developed new technologies that will facilitate further exploration and development. The project identified
more than 1400 disease genes and studied the ethical, legal and social issues raised by expanded knowledge regarding human
genetic make-up. The next stage of utilizing genome-based discoveries in product development will involve pharmaceutical manufacturers
more directly and will require the US Food and Drug Administration (FDA) and other government agencies to address pertinent
regulatory and legal issues.
New vision In marking the completion of HGP's stated task, Francis Collins, director of the National Human Genome Research Institute
(NHGRI) at the National Institutes of Health (NIH), declared it was time to move from large-scale DNA sequencing to more specific
research projects. Collins and other HGP leaders outlined new research and development (R&D) opportunities in a recent article.1
This blueprint for future genomics research describes the resources and technological developments critical to developing
"powerful new therapeutic approaches to disease" (see sidebar "Turning the genomics vision into reality").
Patent protection and public access to data
|
An ongoing issue is the need to balance timely access to new discoveries with protection of intellectual property (IP). Without
an understanding of how complex patent and licensing policies influence private sector investment in new technologies, many
diagnostic and therapeutic advances based on genomics may never reach the clinical setting where they can benefit patients
(see sidebar "Patent protection and public access to data").
Pharmacogenomic policies A major concern for manufacturers is that gathering more genomic data will affect policies governing the testing and approval
of new drugs and medical products. Industry is already investing more resources in pharmacogenomics (PG) to better understand
how and why individuals respond differently to pharmaceuticals, a key issue in identifying candidate compounds and in testing
them for safety and efficacy. This information promises to streamline animal and human studies by
- developing new sets of biomarkers for toxic responses in animals and humans
- predicting who will respond to a drug based on genetic differences in pathogenesis
- predicting who will have serious side-effects based on toxicogenomic analysis
- rationalizing drug dosing by using genetic/phenotypic tests for metabolizer status.
Such analyses have the potential to revolutionize drug development processes and lead to the production of more effective,
less toxic drugs more quickly and efficiently, commented Janet Woodcock, director of FDA's Center for Drug Evaluation and
Research (CDER), during the April meeting of FDA's Science Board. However, FDA officials are concerned that companies are
not presenting PG analysis to the agency because of fears that it may lead to requests for even more data and tests, and further
delay new product approvals. Woodcock wants to clarify FDA regulatory policies to encourage PG analysis and to gain access
to information that could advance scientific discovery.
In a sense, this concern among manufacturers that innovative research will complicate product regulation is similar to the
"don't use" and "don't tell" attitude hindering industry adoption of new manufacturing technologies. Manufacturers worry that
installing new online methods to control pharmaceutical production and drug quality will raise additional questions from plant
inspectors and FDA staffers who review chemistry, manufacturing and controls data. To overcome these obstacles, FDA has launched
the Process Analytical Technology (PAT) initiative, which aims to encourage manufacturers to install more efficient production
systems, which are able to reduce costs and better ensure product quality.
Similarly, Woodcock seeks to develop several PG guidances to clarify agency approaches for using PG information. The aim is
to overcome industry reluctance to disclose data from exploratory PG studies used by researchers to identify target compounds
and to evaluate cellular and animal responses to drug candidates. The guidances will address
- when PG data will have a "regulatory impact"
- co-development of drugs and diagnostic tests
- general PG standards and techniques.
The first step will be to develop "concept papers" regarding these topics, followed by draft and final guidances, which FDA
hopes to issue by the end of 2004.
Achieve optimum FTIR performance, sensitivity, reliability and value with Shimadzu spectrometers.
Introducing the ACQUITY UPLC H-Class System, the new UPLC system for HPLC users
Celebrating 35 years of processing solutions! Join us, May 4-6, in Rosemont, IL.
Introducing the ACQUITY UPLC H-Class System, the new UPLC system for HPLC users
See the latest news from Optima Group pharma.
Buy new and used pharmaceutical equipment, used process equipment and laboratory equipment at LabX. Auctions, classifieds, and post FREE wanted ads to locate equipment.
Try a complimentary sample of the new ultra-pure gasket from Gore PharmBIO Products
Major Biotech & Pharmaceutical Equipment Auction from Pfizer, Merck & More
Special offers on new and improved products from Restek.