While FDA has allowed charging for investigational drugs since the 1987 “Charging Rule” went into effect, the Agency has been responding to questions from industry ever since. In 2009, the FDA revised the Charging Rule itself. The revised guidance (here) discusses these changes and notes that the “FDA’s regulations on charging for investigational drugs under an investigational new drug application (IND) for the purpose of  either clinical trials or expanded access for treatment use (21 CFR 312.8), which went into effect on October 13, 2009” has continued to generate questions relating to the implementation and charging for investigational or compassionate use products, and in 2016, the Agency issued a Q&A document to address many of those questions.

The Federal Register notice (here) announces the availability of the revised draft guidance that includes answer to questions received since the 2016 guidance issued. It also says, “ In addition to editorial changes for clarity, significant changes from the 2016 version of the guidance include additional recommendations related to (1) submission of a copy of the receipt or invoice from the manufacturer as documentation when the expanded access sponsor intends to charge only the amount the manufacturer charged for the investigational drug and (2) distribution of the manufacturing, administrative, or monitoring costs from the first year over the expected duration of the expanded IND or protocol.”  The criteria for charging for investigational drugs are provided in 21 CFR 312.8(a) through (d).

The Q&A document explains how and when the agency permits charging for the investigational drug, but the Agency notes that the mechanism for charging or whom to charge is not within the purview of the FDA.  With the recent controversy surrounding accelerated approvals and or the high cost of such drugs, this Q&A guidance may take on more significance to the industry. Enjoy the read!