The US Food and Drug Administration (FDA) has published new rules to clarify the type of patients that should be eligible for receiving fast-tracked investigational drugs as well as guidelines about how much those drugs should cost.
The new rules are part of an ongoing FDA initiative to fast-track new investigational drugs, used outside of clinical trials, for seriously ill patients suffering from illnesses such as hepatitis and HIV/AIDS who have no other treatment options.
To increase awareness and use of investigational drugs in the healthcare community, the FDA has defined three categories of patient populations to whom investigational drugs could be made available.
These are: individual patients; small patient groups not usually selected for investigational new drugs (IND); and larger populations where widespread IND treatment is more appropriate.
Existing regulations only recognised emergency use for individual patients and widespread use in large populations. The new rules expand the circumstances under which access to investigational drugs is permitted to non-emergency settings and to smaller patient groups, which are recognised by the FDA as having no satisfactory approved therapy.
The FDA has also changed the changed the regulations governing when it is appropriate to charge for an investigational drug to reflect the broader range of circumstances for their use.
The new rules clarify that charging for treatment use of an investigational drug is intended to facilitate and encourage access to drugs that might not be made available for treatment use unless a manufacturer is able to recover costs.
Such charges are only permissible in a clinical trial to facilitate development of drugs with significant promise, which might otherwise not have been developed because of their high cost.
The changes make it clear that charges for an investigational drug may include only direct costs associated with the drug’s development, and that charges for investigational drugs include administrative costs of making the drug available for intermediate patient populations.
Indirect costs, including costs incurred primarily to produce the drug for commercial sale, cannot be recovered