Set up an Expanded Access Program in a few weeks (II)
Welcome to Part 2 of a 3-part blog post about how pharmaceutical companies can prepare to offer Expanded Access. In case you missed it, see Part 1 (The Philosophy of Expanded Access).
A Checklist to Get Up and Running
For a more-detailed review of operational requirements from a U.S. perspective, please see the MedaSystems white paper Expanded Access in the United States: Planning and Management Considerations. Here I will offer just a summary, focusing on the main areas of preparation needed to set up a simple Expanded Access program in just a few weeks.
Here is a checklist of six key things to consider (for full disclosure, items 2 & 3 are areas where MedaSystems helps our customers):
For a more-detailed review of operational requirements from a U.S. perspective, please see the MedaSystems white paper Expanded Access in the United States: Planning and Management Considerations.
Policy. You'll need to develop your Expanded Access policy, and publish this on the company website. (In the U.S., this is required even for companies not offering Expanded Access.) For more detail on what to put in the policy, please see our whitepaper U.S. Regulatory Requirements for Expanded Access Policies. In short, the policy should say whether you accept requests for Expanded Access, how you decide each case (and how quickly), and instructions for how physicians can make a request. Notably, a recent paper entitled Implementation of 21st Century Cures Act Expanded Access policies requirements found that only one-third of a sample of biotechnology companies who should have a policy, actually have one. Moreover, those policies that were available were often incomplete, missing information such as the criteria used to evaluate requests. For those looking for a simple solution, there are free examples available online, including via the Reagan-Udall Foundation.
Request process. You'll need a way for healthcare providers to make requests. This can be via an email address (some companies even list a phone number), but it will be clearer for physicians if you present an online form with specific questions you want them to answer in order to initiate a request. This will also help you to receive requests in a more digestible format. The gold standard is to offer a portal (such as the one pictured below) where, if needed, healthcare providers can log in to initiate a request, update information on an ongoing basis, track progress of the request, and even loop in their administrative staff to help. At the minimum, make sure there's a clear and easy way for physicians to reach you.
Internal tools. You'll also need a reliable process and system to track all cases (whether approved or not), including the follow-up communications with physicians, and the internal decision process, and any related documentation such as regulatory forms. Not to mention shipping and resupply. Many companies are able to successfully do this by repurposing tools such as spreadsheets, email and the proverbial hand-written note on a post-it or whiteboard. However, this approach can result in a poor workflow beset by repetitive manual steps, such as constantly downloading and re-uploading files and attachments between systems, or preparing the same email or chart over and over. This is not to mention concerns with security and auditability, or potential for misplacement of information during the process.
Personnel. You'll need one or more people designated to receive incoming requests, gather any needed follow-up information from healthcare providers, and make sure cases are reviewed by the appropriate colleagues. (Biopharma companies who use MedaSystems can access our template checklists and workflows to support this, which we've previously blogged about here and here.) Smaller companies without dedicated staff for Expanded Access often leverage external consultants to help with the timely triage and management of cases. Experienced consultants can also assist with program design and oversight. Ideally, if a request arrives from a jurisdiction where relevant local regulations are already understood, most of the internal work can focus on the medical risk/benefit analysis, and vetting that the requesting physician is qualified to administer the therapy.
International guidance. If you don't already have the expertise in-house, you'll need a way to navigate local regulations in each country from which you accept requests. You'll need to understand how regulators approve cases (if applicable), as well as rules about labeling, shipping/customs and reporting. One example resource is the Global Guide to Compassionate Use Programs. Some biopharma companies decide upfront which countries and which therapeutics to consider, whereas others choose to see what mix of requests come in, and make decisions as they go. Note that in some geographies, once you provide investigational medicines under Expanded Access, you're required to continue supply until the patient stops treatment or the product becomes commercially available in that geography.
Document package. You may choose to make an information package available to requesting physicians, in order to help them craft their proposed treatment plan and statement of expected benefit vs. risk. (Some companies provide multimedia or even live training sessions.) You'll also want to collate templates of any documents you envisage using frequently—such as a confidentiality and/or other agreement to use with physicians. For those looking for creative inspiration, there are several good examples of physician attestations, etc. available online, including here and here.
An example of an Expanded Access intake portal
Key in all this is to keep things simple for the physicians who are making requests. Their compensation structure may not adequately recognize the time they spend on Expanded Access, and the process is for them often a lengthy and confusing one, especially for those who do it infrequently. I would go as far as to argue that the single weakest link in the entire global Expanded Access pathway is the quicksand of paperwork in which physicians get bogged down. The clearer you can make your instructions to them, the faster things will move—and the less follow-up you'll need to do to gather all the information you need in order to approve patient treatment.
In Part 3, we will conclude by reviewing the opportunity to collect Real-World Data from patients treated under Expanded Access.