2009-08-17
The following is a message from the FDA’s Office of
Oncology Drug Products Director, Dr. Richard Pazdur:
On August 12, 2009, the Food and Drug Administration
(FDA) published two new rules related to investigational drugs.
One new rule, “Expanded Access to Investigational
Drugs for Treatment Use,” amends existing regulations on expanded access by
clarifying current rules and adding new types of expanded access for treatment
use. The second rule, “Charging for Investigational Drugs Under an
Investigational New Drug Application,” clarifies the specific circumstances and
the types of costs for which a manufacturer can charge a patient for an
investigational drug, either as part of a clinical trial, or for treatment use
outside the scope of a clinical trial through expanded access.
Expanded Access
The changes are meant to consolidate and clarify the
requirements and facilitate making investigational drugs and biologics more
widely available to seriously ill patients, including those with cancer, who
have no other treatment options.
The new expanded access rule defines three categories of patient populations
(individual patients, small groups of patients and larger groups of patients
under a treatment IND) to whom investigational drugs could be made available
for the purpose of treatment outside of a clinical trial through expanded
access, when there is no satisfactory alternative therapy.
To authorize these expanded access treatment uses, FDA generally must be
satisfied that the patient’s serious or immediately life-threatening disease or
condition has no satisfactory approved therapy; that the potential benefit for
the patient justifies the potential risks; and that providing the therapy will
not interfere with the drug’s development.
Charging for Investigational Drugs
The new rule revises the regulation regarding recovery of the costs of an
investigational drug to clarify that charging may be permitted for any category
of treatment use - individual patient and intermediate-size patient population
INDs as well treatment INDs. Charging for treatment use is intended to
facilitate and encourage access to drugs that might not be made available
unless a manufacturer is able to recover its costs. It also clarifies that charging is
permissible in a clinical trial only to facilitate development of drugs that
promise significant advantages over existing therapies, and might not otherwise
be developed because of their high cost.
The change simplifies the cost recovery calculation by making clear that
charges for an investigational drug used in a clinical trial may include only
direct costs associated with the drug’s development. Charges for
investigational drugs for treatment use under intermediate patient population
and large scale treatment INDs may also include administrative costs associated
with maintaining the IND (e.g., costs to
monitor, costs to comply with IND
reporting requirements). Indirect costs, including costs incurred primarily to
produce the drug for commercial sale, cannot be recovered.
For additional information, please see: http://edocket.access.gpo.gov/2009/pdf/E9-19004.pdf and http://edocket.access.gpo.gov/2009/pdf/E9-19005.pdf.
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