The 21st Century Cures Act: Putting Patients First, Literally and … Substantively (And FDA’s New Expanded Access Form)

By James E. Valentine* & Jim C. Shehan –

The 21^st Century Cures Act’s focus on patients is inescapable.  Title I of this nearly 400 page bill is entitled, “Putting Patients First By Incorporating Their Perspectives Into The Regulatory Process and Addressing Unmet Needs.”  When we reported on the 21^st Century Cures Initiative hearing on incorporating the patient perspective (see our previous coverage here), it was clear from stakeholder testimony and engagement by Energy & Commerce Committee members that such a legislative proposal would be included.

So what exactly do patients get in the proposed legislation?  Well, considering the entire document is devoted to the discovery, development, and delivery of new treatments and cures, potentially quite a lot (see our broader coverage of the Act here and its proposed expansion of clinical trials data sharing here).  But specifically, we’ve identified two provisions that are patient centric: (1) Patient-Focused Drug Development and (2) Expanded Access.  Neither are new concepts.  In fact, both are current programs at FDA.  So let’s break down what’s new.

Patient-Focused Drug Development

This provision, Section 1001, drafted by Health Subcommittee Chairman Joe Pitts (R-PA) and Representative Cathy McMorris Rodgers (R-WA), would build off FDA’s Patient Focused Drug Development.  FDA would be required to implement the structured benefit-risk framework it took on in PDUFA V, and develop processes for incorporating “patient experience data” into the framework to be used in its regulatory decision-making with respect to the benefits and risks of new drugs.

“Patient experience data” broadly includes information about the experience of patients with a disease, including specifically the impact of the disease on their lives and their caregivers.  This information can take the form of complete data and summaries and analysis of the data, and may be incorporated into “draft guidances” that patients groups submit to the agency. 

To facilitate “patient experience data” collection, the provision would have FDA publish guidance on (a) methodological considerations for collecting this information, (b) assessing patients’ beliefs of benefits and risks in the management of their disease, and (c) experimental designs for patient-reported outcomes.  FDA is also asked to include timeframes for review of documents containing this information and how it will use the documents and data.  Interestingly, the guidance would also cover establishing and maintaining patient registries designed to improve understanding the natural history of a disease.  A pre-draft workshop and post-draft public meeting would facilitate guidance development.

So in summary, this one provision endorses three tools for fostering patient participation in the regulatory process: patient registries, patient-reported outcomes, and “patient experience data,” including benefit-risk preferences.

Expanded Access

Another heavily patient centric section of Title I of the 21st Century Cures Act is Subtitle G, entitled “Expanded Access.”  This subtitle almost replicates H.R. 8505, the Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act, introduced at the end of last year by Representative Michael McCaul (R-TX) and blogged on by us previously.  Among changes worth noting, Subtitle G makes some slight changes to the scope of products covered, adding designated orphan drugs and dropping drugs approved under accelerated approval and drugs whose sponsors receive priority review vouchers.  Also, the task force assigned to look for ways to improve expanded access has grown from nine to thirteen members and is specifically asked to look at compliance with adverse event reporting requirements in expanded access programs.

There was another significant development in expanded access last week – FDA issued a draft guidance containing its new form for individual patient expanded access INDs.  Checking in at two pages and containing only eight sections that the patient’s physician needs to complete, the, new draft form, designated Form FDA 3926, appears to accomplish the FDA’s stated purpose  to “greatly simplify and accelerate the process by which a physician can request” expanded access.  The most significant pieces of information that physicians will need to provide are:

1. The patient’s initials;
2. Clinical information, including indication, brief clinical history of the patient, and the rationale for requesting the proposed treatment, including an explanation of why the patient lacks other therapeutic options
3. Treatment information, including the investigational drug’s name and treatment plan, that is, the planned dose, route and schedule of administration, planned duration of treatment, monitoring procedures, and planned modifications to the treatment plan in the event of toxicity.
4. A letter of authorization from the investigational drug’s manufacturer
5. Information about the physician’s qualifications;
6. The physician’s name, address and contact information;
7. A check box authorizing use of the form, which amounts to a request for a waiver of additional requirements under 21 C.F.R. Part 312; and
8. The physician’s signature and certification that treatment will not begin until 30 days after FDA receives the application unless there is earlier notification from FDA; that the physician will obtain informed consent; that IRB review of the expanded access use will be obtained; and that in the case of an emergency request treatment may begin without prior IRB approval provided the IRB is notified of the emergency treatment within 5 working days of treatment.

According to a post on FDA Voice, FDA expects that the new form can be completed within 45 minutes.  Comments on the guidance and the draft form may be submitted to FDA within 60 days.

*Admitted only in Maryland. Work supervised by the Firm while D.C. application pending.