Introduction
Most regulatory affairs careers are built around the standard development pathway: preclinical work, an IND or clinical trial application, phased clinical trials, and eventually a marketing application. But a growing slice of regulatory work happens outside that pathway entirely, for patients who cannot wait for it or do not qualify for an ongoing trial. Expanded access, sometimes called compassionate use, is the mechanism that lets a patient receive an investigational drug or biologic before it is approved. As sponsors build deeper pipelines in oncology, rare disease, and cell and gene therapy, more of them are standing up formal expanded access programs, and that is creating a distinct, specialized lane within regulatory affairs.
This is not a large hiring category compared to core submissions work, but it is a real one, and it rewards a specific mix of regulatory judgment and patient-facing communication that not every regulatory professional has had the chance to build. Understanding what the work actually involves is useful whether you are considering it as a specialty or simply want to recognize it when it shows up in a job posting or a cross-functional project.
What Expanded Access Actually Covers
In the United States, FDA's expanded access framework (sometimes called Treatment IND or Treatment Protocol) allows a patient with a serious or immediately life-threatening disease to receive an investigational product outside of a clinical trial, when there is no comparable or satisfactory alternative therapy available. FDA regulations describe three categories: individual patient expanded access for a single patient, intermediate-size population expanded access for a defined group with the same condition, and widespread treatment use for larger populations once a drug is far enough along in development.
A separate but related pathway is the federal Right to Try Act, signed into law in 2018, which allows eligible patients to request access to certain investigational drugs that have completed a Phase 1 trial without going through FDA's expanded access process at all. Right to Try did not replace expanded access; it created a second route with its own eligibility criteria and its own documentation expectations, and companies that receive requests need to know which pathway a given request falls under and what each one requires of them.
The sponsor is not obligated to grant any individual request. Deciding whether to make an investigational product available, and under what conditions, is a company decision that regulatory affairs is almost always asked to help shape, because the decision has direct regulatory consequences: safety data from expanded access use is reportable, and the way a program is designed can affect the ongoing clinical development program if it is not handled carefully.
Why This Work Is Growing
Several forces are pushing more companies to formalize expanded access rather than handle requests ad hoc. Pipelines in rare disease, oncology, and cell and gene therapy have grown substantially, and these are exactly the therapeutic areas where patients and physicians are most likely to ask about access before approval. Patient advocacy organizations have also become more organized and more vocal about expecting sponsors to have a stated access policy, and some companies now publish their expanded access policy publicly, as FDA guidance recommends for products intended to treat serious conditions.
There is also a practical driver: an unmanaged expanded access request, handled inconsistently or without proper documentation, can create real problems, from inconsistent safety reporting to reputational damage if a company is seen as unresponsive to a dying patient's request. Building a formal program with clear criteria, a defined regulatory pathway, and a cross-functional review process reduces that risk, and someone in regulatory affairs typically owns building and running it.
What the Regulatory Affairs Role Looks Like Day to Day
The work is a mix of writing, process design, and cross-functional coordination. On the writing side, someone has to draft or review the treatment protocol or individual patient IND submission, prepare the informed consent language in coordination with legal and clinical, and keep the safety reporting plan current as adverse events come in from expanded access use, which follows different timelines than a standard clinical trial.
On the process side, the role often means building the intake and review workflow: how a physician's request reaches the company, who evaluates eligibility, how quickly a decision gets communicated, and how the decision gets documented in a way that would hold up if FDA or an inspector asked about it later. That includes coordinating with medical affairs, clinical operations, quality, and often legal and communications, since a request for compassionate use is frequently urgent and emotionally charged for everyone involved.
Regulatory affairs is also the point of contact with the health authority itself. An individual patient IND typically requires FDA authorization, and while the agency can act quickly in emergencies, the request still has to be filed correctly and the follow-up reporting obligations still have to be met. Getting this right under time pressure, without cutting corners on documentation, is the core skill the role is built around.
Skills That Differentiate a Candidate for This Work
Strong regulatory writing and a solid grasp of safety reporting requirements are the baseline. What tends to separate someone who does this work well from someone who merely can is comfort operating under emotional and time pressure without letting that pressure erode documentation discipline, and the ability to explain a regulatory constraint to a treating physician or a patient's family in plain, respectful language rather than agency jargon.
Cross-functional fluency matters more here than in many other regulatory roles. A person running an expanded access program is regularly the bridge between medical affairs, which is fielding the physician relationship; clinical development, which is worried about any effect on the ongoing trial program; legal, which is thinking about liability and consent; and communications, which may need to prepare for public interest in a high-profile case. Being able to hold that group together and keep the regulatory pathway moving is the differentiator.
How Expanded Access Interacts With the Ongoing Clinical Program
One of the trickier parts of this work is that expanded access does not happen in isolation from the company's registrational trials. Investors and clinical leadership sometimes worry that safety events from expanded access use, gathered outside a controlled trial and often in sicker patients than a trial's eligibility criteria would allow, could complicate the interpretation of the pivotal safety database or raise questions from FDA during review. Regulatory affairs has to design the program so that it serves patients without creating avoidable noise in the data package the company will eventually submit, which often means being deliberate about eligibility criteria, monitoring requirements, and how expanded access safety data is analyzed and presented separately from trial data.
Supply is the other constraint regulatory affairs has to manage honestly. A company running a small clinical program may simply not have enough drug product to support expanded access requests without threatening its own trial timelines, and part of the regulatory role is helping leadership make, document, and communicate that constraint clearly, rather than letting an ad hoc yes turn into a promise the company cannot keep for the next request.
How Other Regions Handle This
The United States is not the only place this work happens, and companies operating globally need a regulatory affairs function that understands the differences. The European Union has its own compassionate use provisions, allowing member states to authorize access to a medicine that is not yet approved for patients with a chronic, seriously debilitating, or life-threatening condition, though the specific procedures are implemented at the national level and vary by country. Many other countries, including Canada, Australia, and Japan, run their own named-patient or special access programs with their own eligibility rules and submission formats.
For a regulatory affairs professional supporting a multinational program, this means tracking a patchwork of country-specific pathways rather than a single global standard, and knowing early which markets can support an urgent request and which cannot, so that a physician's inquiry does not stall while the company works out which regulatory framework even applies.
How This Shows Up in Job Postings
It is uncommon to see an entry-level job titled specifically for expanded access. More often the work appears as a named responsibility inside a broader regulatory affairs role, particularly at companies with concentrated rare disease or oncology portfolios, or it shows up as a defined specialization once a company's pipeline matures enough to justify a dedicated person or small team. Titles to watch for include Expanded Access Program Manager, Regulatory Affairs Manager - Patient Access, or simply a regulatory affairs role where the posting explicitly lists Treatment IND or compassionate use experience as a plus.
If this work interests you, the fastest way in is usually lateral: volunteering to support an expanded access request inside a broader regulatory role, or moving from clinical operations or medical affairs into regulatory with that experience already in hand. It is a specialty built more on demonstrated judgment than on a credential, which makes it accessible to people who have not followed the standard submissions-first career path.
Conclusion
Expanded access and compassionate use work will never be the largest category of regulatory affairs hiring, but it is a growing one, and it sits at an unusually direct intersection of regulation, patient need, and company reputation. For regulatory professionals who want work that combines technical rigor with a very human stakes, it is worth understanding closely, whether the goal is to specialize in it or simply to be the person on a cross-functional team who already knows how the process works when a request comes in.