Medical Device Daily Washington Editor

WASHINGTON – New guidance released Wednesday by the FDA is part of a agency-wide move to speed the development of new medical products using pharmacogenomics.

The guidance, titled "Pharmacogenomic Data Submissions," outlines how pharmacogenomic data will be evaluated by the FDA. It also outlines the kind of data needed during the marketing application review process, the format for submissions, and the data that will be used during regulatory decision-making.

The promise of using pharmacogenomic data is that it would allow healthcare providers to profile how an individual responds to certain drugs and therapies and create a specific therapeutic option for that person, according to the agency.

Because it can take multiple attempts to find the right drug and dose, doctors ideally would be able to analyze a patient's genetic profile and prescribe the best available drug therapy and dose from the start.

Genomic tests, for example, have been used to identify cancers that may be most likely to respond to a particular medication or regimen. This technology also has helped the development of targeted therapies, like those for metastatic breast cancer, chronic myeloid leukemia and metastatic colorectal cancer.

The FDA also recently approved the first laboratory test, the Amplichip Cytochrome P450 Genotyping Test, from Roche Molecular Systems (Pleasanton, California), which provides physicians with genetic information to select the right doses of certain medications for cancer and cardiac and psychiatric diseases.

Janet Woodcock, MD, the FDA's acting deputy commissioner for operations, said the agency hopes the guidance will bring researchers a step closer to truly personalizing medical treatment. She said it would allow medicines to be "uniquely crafted" to maximize benefits and reduce risk.

The agency's recommendations in the guidance also explain a new mechanism for industry to voluntarily submit research data to further the scientific exchange of information. The voluntary data, which will be reviewed by an internal FDA group – and not available for regulatory decision-making – is intended to help the agency and industry gain valuable experience as the field continues to evolve, FDA said.

"We hope ultimately to bring pharmacogenomics, a way in which to foster the personalizing of medicine, to every healthcare professional's prescription pad for the benefit of their patients and U.S. consumers," said Woodcock.

She said she hopes the guidance will save time and resources and eliminate possible delays in the application review process.

The Personalized Medicine Coalition (PMC; Washington) this week issued a statement saying it fully supports the FDA's new guidance and that the move will lead to "new generation of diagnostics and therapeutics based on an understanding of disease at the molecular level."

PMC said it hopes the new process will allow product sponsors to have broad technical discussions with the FDA about research data without undue concern that the submitted data will be used for regulatory decision-making.

The coalition's membership includes diagnostic and therapeutic companies, information technology organizations, academic medical centers, patient advocacy organizations and healthcare insurers and providers.

The agency web page related to this guidance is available at, and includes information on submitting genomic data, such as a decision tree to simplify data submissions, relevant regulatory information, and FDA contact information.

FDA pats itself on the back

Also on Wednesday, the FDA released a report on its accomplishments for 2004 – a list of devices approved and new agency initiatives and programs. On the device side, the Center for Devices and Radiological Health (CDRH) reported approving 52 devices that use new technologies via premarket approval applications, and cleared 3,365 devices through 510(k)s, meaning equivalence with existing technologies.

Some CDRH product approvals included:

  • The Pediatric DeBakey VAD from MicroMed Technology (Houston), the first miniaturized heart pump approved for children between five and 16 years old who need temporary mechanical blood circulation while awaiting a heart transplant;
  • The HeartStart Home OTC Defibrillator from Philips Medical Systems (Bothell, Washington), the first automatic external defibrillator for use without a prescription;
  • The ImageChecker CT CAD Software System from R2 Technology (Sunnyvale, California), a new technology to help radiologists analyze computed tomography images of the chest; and
  • The ExAblate 200 System by InSightec (Haifa, Israel), a non-invasive device that uses magnetic resonance image-guided focused ultrasound to destroy non-cancerous uterine fibroids.
  • CDRH also cleared several important new in vitro devices for marketing, including:
  • The AmpliChip Cytochrome P450 Genotyping Test (referred to above), a DNA microarray test that provides genetic information to aid in selecting patient medications and optimizing the dose;
  • The NeoGram Amino Acids and Acylcarnitine Tandem Mass Spectrometry Kit from PerkinElmer (Boston), the first blood test cleared for screening infants for certain inherited diseases; and
  • The QuickELISA Anthrax-Pa Kit by Immunetics (Boston), the first rapid serum antibody test used for detecting antibodies triggered by infection with anthrax.
  • Other notable devices cleared for marketing by the FDA were:
  • The CellSearch Epithelial Cell Kit/CellSpotter Analyzer from Veridex (Warren, New Jersey) for monitoring the effectiveness of cancer treatment; and
  • The Pathway Anti-c-KIT (9.7) Primary Antibody from Ventana Medical Systems (Tucson, Arizona), the first immunohistochemical marker used to help in the selection of patients for treatment with a rare gastrointestinal cancer.