Leading the Way in Life Science Technologies

GEN Exclusives

More »


More »
October 15, 2009 (Vol. 29, No. 18)

Update on Evolving Regulatory Guidance

Companies Need to Be Cognizant of and Continually Adjust to Changing Guidelines

  • New Access to Investigational Drugs

    In mid-August, FDA published two guidance documents that allow expanded patient access to investigational drugs/biologics outside of a clinical trial. The documents also defined the circumstances under which individual patients, as well as intermediate and larger size patient populations, can obtain unapproved investigational drugs.  

    Additionally, the FDA clarified the circumstances under which companies can charge for access to an unapproved investigational drug/biologic and what type of costs can be recovered. (Additional information can be found at www.fda.gov)

    As a result, companies developing promising new therapies are now likely to have more inquiries from patients who have failed approved therapies. Companies should anticipate such requests with policies regarding access. They should also consider how to best use the opportunity that these two new guidances present; specifically, companies with orphan drugs should look at the utility of the guidance to facilitate clinical exposure beyond their clinical study.

  • Foreign vs. Domestic Inspections

    Earlier this year, the FDA was criticized for the lack of technical strength in its foreign inspection program. As a consequence, FDA recently announced that it is doubling the number of overseas facility GMP inspections that it conducts. The FDA also reported that these could be unannounced inspections.

    I am aware of one non-U.S. manufacturer of OTC drugs that did have a surprise inspection, but it was merely to validate that the non-U.S. company is an active OTC drug manufacturer. Nonetheless, unannounced FDA inspections present a new challenge to non-U.S. product manufacturing facilities. 

  • Cancer Vaccines

    A shift in CBER’s stance on therapeutic cancer vaccines was evident in the organization’s recent “Draft Guidance for Industry: Clinical Considerations for Therapeutic Cancer Vaccines” (September 2009).

    FDA has recognized that testing cancer vaccines using the conventional model in metastatic cancer patients may not allow time for development of an antitumor immune response needed for demonstration of potency/effectiveness.

    The agency concluded that the administration of an investigational vaccine in patients with metastatic cancer, who are receiving multiple treatments that are detrimental to the immune system, likely minimizes the potential responsiveness to the cancer vaccine being tested.

    However, testing cancer vaccines in patients with no evidence of residual disease or who have minimal burden of disease, as discussed in this guidance, may provide adequate time for the immune response elicited by the cancer vaccines to develop and manifest. 

    This new position focuses the initial clinical effort on safety and demonstration of an immune response, as well as on determining the optimal-vaccine dose, dosing regimen, and maximum-tolerated dose.  FDA previously stated that the demonstration of an immune response is not considered an indicator of potential efficacy.

    The interesting question is whether investors will accept an immune response as a sufficient proof of principle and support the continued development of the experimental vaccine.

Related content