Charging for Investigational Drugs: Current Application of 21 CFR Part 312 and the Expanded Access Rules

FDA's final rule revising the 21 CFR Part 312 regulation concerning charging patients for investigational new drugs clarifies the circumstances in which charging for an investigational drug may be appropriate and which costs can be recovered. This rule permits charging for a broader range of uses than was previously allowed. Two new categories of expanded access, individual patients and intermediate-size patient populations, are now included. Three circumstances must be met. First, the promising new drug might not otherwise be developed or safety information might not otherwise be obtained without charging for the drug. Second, charging is only permitted for a trial that is necessary for the further development of the drug. Third, charging must be necessary to fund the conduct of the clinical trial.

Although the three conditions seem straightforward, the devil, as usual, is in the details. And what a devil! In this webinar we will dissect the regulations for charging for an investigational drug. We will start with FDA's rationale for the expansion, so that a lot of these tricky requirements make more sense. We'll discuss the differences between indirect and direct costs and how these are viewed in terms of charging for the drug. Then there are time limits, population size limits, requirements for progress of the clinical trial, rules for charging for the sponsor's own drug vs. charging for an approved drug obtained from another entity for use as an active control and we'll review the guidelines and provide some practical advice for moving forward in each of these areas.

Once you have got the rules down or at least have your list to follow, there are supporting document requirements. We'll describe those documents with some examples. And just when you think you are ready to support your clinical study by charging for your investigational drug, we'll step back for a moment and consider price setting and the documentation that will require, followed by some advice on how to navigate collaboration with various payment systems (CMS and Insurance).

We'll also need to discuss promotion limitations, liability, interim labeling and overall economic impacts that FDA will consider before final authorization. Detail by detail, this webinar will introduce you to the process of gaining approval to charge for your investigational drug.

• Determining if your investigational drug is a candidate for this program.
• Developing the supporting documentation for FDA review
• Different paths for approval when charging for sponsor's own drug vs. charging for an active control obtained from another entity.
• Duration of charging authorization and how it's determined.
• Recoverable cost limitations, indirect and direct costs. How to figure.
• Effects on other payment systems.
• Promotion, liability, and labeling for this stage of drug development.
• Some ethical considerations

• Sponsors
• Company CEOs, CFOs and even COOs
• Clinical Research Professionals
• Clinical Protocol Developers
• Clinical Study Managers
• Regulatory Managers
• Clinical Studies Directors

Learning objectives:

• Participants will understand why FDA has expanded the conditions under which a sponsor may charge for an investigational drug.
• Participants will have an understanding of the complexity of the approval process.
• Participants will know where to find the resources provided by FDA to facilitate implementation of the expanded access rule