COMPASSIONATE USE
Frequently Asked Questions

To make this FAQ accessible to all audiences, some healthcare industry jargon has been omitted. For example, we say “experimental drug” to include all compounds and biologics under investigation. If you would like further technical detail, please contact us.

ABOUT
COMPASSIONATE USE


What is compassionate use?

Compassionate Use is a way that patients can access experimental medical treatments. Under compassionate use, patients with life-threatening or severe chronic health conditions can access experimental medical treatments when (1) they have no commercially approved treatment options remaining and (2) they cannot access a clinical trial. In most countries, compassionate use of a medical treatment outside of clinical trials requires government approval.

Patients may seek compassionate use of experimental treatments at any phase of clinical trial, though it is more common to pursue a treatment in clinical trials where the treatment shows some indications of efficacy. That said, the existence of efficacy or the degree of efficacy may not yet be established at the time of requesting compassionate use.

Government agencies sometimes call “compassionate use” by other names, including “expanded access,” “special access,” “compassionate access,” and “temporary use.”

How does a patient seek approval for compassionate use of experimental drug?

Compassionate use in most countries requires 2-3 steps:

  1. Drug Company Agreement
    The drug company must determine if it will provide the experimental drug to the patient, because the drug company owns the drug. Drug companies are not obligated to provide compassionate use of drugs under development. In the United States, a patient cannot submit a formal compassionate use request to FDA until the drug company first agrees to provide compassionate use of the drug.
  2. Government Approval
  3. Medical Ethics Committee
    Some countries require that a medical ethics committee approve and monitor a compassionate use treatment plan to ensure that patient welfare is appropriately considered.

The requirements for government approval differ from country to country. See our Compassionate Use by Country for a summary of compassionate use approval programs in the United States, European Union, Canada, and Australia.

Is compassionate use for individual patients or groups of patients?

Compassionate use can be provided to an individual patient or to group of patients with similar health conditions and situations.

CURe uses the term “compassionate use” to refer to access for both individuals and groups of patients. Some governments and organizations only use the term “compassionate use” for single patient approval; however, such distinctions can get murky and are not always useful.

CURe strongly believes that patients are best served when we address compassionate use for both individual patients and groups of patients together. Individual compassionate use cases can help open the door to providing compassionate use to groups of similar patients. Individual cases can increase awareness — in drug companies, regulators, doctors, patient advocates, the public—of the greater need and demand for compassionate use of a drug. They can also providing initial learning that may benefit a larger group of compassionate use recipients. In some cases, individuals pursuing compassionate use can be—by happenstance or by design—the voices of a population of patients who may not know about compassionate use or may not have the resources to seek compassionate use.

Each country determines how it manages individual and group compassionate use approvals. The United States Food and Drug Administration (FDA) break “expanded access” into three categories of patient-group size:

  • Individual Patient Access (“Single Patient IND”)
  • Intermediate-Size Patient Population Access (use by more than one patient, but not as many patients as would make up a typical clinical trial group)
  • Expanded Access for Widespread Use
How many people request and receive compassionate use each year?

No one knows the number of patients seeking compassionate use each year. The requests that receive “NO” from a drug company generally go unrecorded.

In the U.S., FDA does track the number of requests it receives and approves. Unfortunately, this tells us very little about the full population of patients seeking compassionate use. From 2011-2015, FDA received an average of 1,210 compassionate use requests annually and approved over 99% of the requests. However, FDA does not accept compassionate use requests until after the drug company has agreed to provide access to its experimental drug. In other words, the FDA data only tell us the number of requests in the United States that received a YES from drug companies. FDA data tell us nothing about the number of people who asked and received a NO from drug companies.

What we do know is that the number of people who ask drug companies for compassionate use is bigger—likely much bigger—than the number of requests FDA receives at the end of the compassionate process.

Do drug companies report on the number of requests they receive, agree to, and deny?

Many larger drug companies do not actually know how many compassionate use requests they receive each year. Until recently, most companies did not centrally coordinate and keep records of compassionate use requests. That is beginning to change due to recent social media and legislative pressure. However, there is no guarantee that companies will share the data. CURe encourages them to do so.

CURe is exploring different ways to fill the data gap, including physician surveys, self-reporting, and industry survey approaches.

INPUT: If you have good data sources on compassionate use requests volumes outside the United States, please contact us at info@compassionateusereform.org.

Is it ethical to give compassionate use when the experimental drug is not fully tested or proven effective?

The ethics of compassionate use have been a serious discussion between doctors, patient advocates, drug companies, ethicists, and regulators since the inception of compassionate use programs.

The big question is whether it is ethical to administer experimental drugs to patients before we know if the drugs are medically effective and before we understand the full set of risks associated with the drug. This article provides a good summary of the history of the ethics discussion. Lawmakers in many countries have determined that compassionate use is acceptable, given the following conditions:

  • The patient has exhausted other comparable treatment options before trying an experimental drug
  • The risks to the patient are deemed no greater than not using the experimental drug
  • The patient (or patient’s guardian) gives informed consent — i.e., the patient receives a full explanation of the treatment and associated risks and benefits, and the patient then chooses to go forward with the treatment.
  • Compassionate use of the drug does not interfere with completion of clinical trials of the drug which benefit the longer term good of future patients.

These conditions provide the framework for government decision-making rules for compassionate use.

But these are not straightforward questions. For example, how can you compare the risks of the experimental drug to the risks of the disease itself when the drug is too new to know its risks? Or, how can you ensure informed consent when there is evidence that both patients and doctors regularly consider new treatments to be more promising than existing treatments, even when the data do not support such conclusions?

These ethics questions continue to be discussed, and other important questions arise while answering them. In particular, how do we fairly decide who receives compassionate use? Visit NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access to read more about the ethics questions compassionate use stakeholders continue to discuss.

Does compassionate use stop people from joining the clinical trials?

In theory, patients might try to obtain compassionate use instead of enrolling in a clinical trial, because they want to avoid a placebo or “standard of care” treatment that they may receive in a clinical trial. In practice, most patients pursue compassionate use as a last resort, because they are ineligible or unable to enroll in a clinical trial (due to the status of clinical trial or the criteria for trial enrollment, such as patient age, prior health profile, and stage of disease). Under U.S. regulation, FDA cannot approve a compassionate use case if a patient is eligible and able to enroll in a clinical trial of the experimental drug.

Who pays for compassionate use treatment?

The costs of making experimental drugs and the costs of the medical care associated with administering experimental drugs to patients can be very high. Travel to facilities where compassionate use treatment is available can add costs.

In the U.S., FDA allows drug companies to charge for the direct costs of compassionate use (for example cost of manufacturing, shipping, etc.) and some related administrative costs (for example, reporting costs), but they are not permitted to profit from compassionate use. Hospitals may charge for administration of the experimental treatment.

Who pays for compassionate use varies from case to case. Sometimes the drug company absorbs the costs; sometimes a disease foundation will fund it; sometimes the patient must find a way to foot the whole bill.

In the U.S., insurance companies rarely cover the direct costs of the experimental drug, although they may cover some of the tests associated with the compassionate use care. Coverage depends on the patient’s policy.

What is the difference between compassionate use and off-label use?

Off-label use is the unapproved use of an approved drug. In other words, the drug is used to treat a different condition or population (such as children) than it was originally approved to treat. Healthcare providers can prescribe off-label use of an approved drug when they determine it is medically appropriate for their patients. They do not need special government approval. Off-label use is often covered by insurance plans.

In contrast, compassionate use involves a drug that has not yet been granted government approval for any health condition, so a doctor cannot prescribe the drug. For compassionate use of experimental drugs, government approval is required.

THE CHALLENGE


Why isn’t the compassionate use system working well for patients?

Patients face two major challenges in seeking compassionate use: (1) information and (2) drug company agreement:

Lack of Information
There are very few resources that explain to patients how to step through the process to request compassionate use. Most resources focus on the government approval stage, but are silent on the critical first stages. There is little information out there about the hard, confusing, and often frustrating part: asking a drug company for compassionate use. The resources that do exist are not written or designed for patients and families who are sick, emotionally strained, tired, stressed, and critically pressed for time.

And it isn’t just patients who struggle with the process. How-to information is also scarce for doctors and small drug companies.

CURe Info Hub will be a patient-centric online guide to the whole compassionate use process— from the first step to talk to your doctor about compassionate use to asking for help from a drug company to the formal request for government approval. Learn more below.

Drug Company Agreement
The drug company developing the experimental drug must agree to provide compassionate use of the drug, because the experimental drug is the private property of the drug company. Drug companies often deny access, even if patients meet the government criteria and their doctors’ medical judgement for compassionate use.

There are several reasons why drug companies deny compassionate use to a patient, depending on the company’s size, mission, culture, and precedent, on manufacturing limitations, and on clinical trial processes. These barriers and disincentives can include:

  • Financial—The smaller companies, often the developers of cutting-edge treatments, are often cash strapped and unable to divert their funds away from clinical trials
  • Profit—Companies cannot profit from compassionate use of their experimental drugs
  • Regulatory—Some companies are concerned that a harmful side effect in a compassionate use case may give government regulators grounds to deny approval of the drug
  • Human Resources & In-House Knowledge—Some smaller companies do not know how to conduct compassionate use and/or cannot afford the additional staff and staff time to manage the process
  • Manufacturing—Sometimes a drug company has challenges supplying its clinical trials with the drug and cannot spare drug supplies for compassionate use
  • Cultural—Some drug companies have not made the decision—yet— that from the top-down their company will make compassionate use work for the patient when it is medically appropriate and logistically possible.

We strongly believe that there are practical win-win solutions to lower those barriers. What compassionate use needs is the right thinkers and doers to work together and identify and build solutions. Some solutions are at our fingertips; others can be built with a willingness to work together. CURe will make that work happen with Solution Hub.

Why would drug companies be interested in finding solutions to make compassionate use work?

First, drug companies are made up of people, and many many of those people work for drug companies, because they want to help other people. Many drug company employees have fought doggedly inside their companies for a patient’s compassionate use, and they have been deeply saddened and frustrated when their companies say no. There is a mountain of good will and human hearts in the drug development industry. We want to work with that good will to find a workable way to provide compassionate use as needed.

Second, social media and press spotlights have turned up the pressure on drug companies to improve their compassionate use records and to play a leading role in making compassionate use work for patients.

Third, some companies have taken steps to work with experts and patient stakeholders to make their compassionate use process work better. For example, in 2015 Johnson & Johnson announced that they would work with NYU School of Medicine to pilot an external committee to fairly determine which patients should receive compassionate use of the limited supply of one experimental drug. In 2016, the company announced that the program was expanding to cover all of its experimental medical products. It’s a good start.

CURe will take the next step forward by helping companies and stakeholders identify and solve the barriers strategically.

Is government the problem?

Our short answer: No

In the U.S., the FDA Expanded Access program has gone through many revisions and improvements since it was formalized during the 1980s AIDS crisis. FDA now has a strong program that is medically sensible, appropriate for the greater public good, and compassionate towards patients. Unfortunately, until drug companies improve their compassionate use participation, patients and doctors cannot use the FDA program to its full potential.

Government still has an important problem-solving role to play. Drug companies face barriers to participating in compassionate use. To bring down those barriers, we need government participation in building win-win solutions that will work in current and future regulatory environments.

What is Right To Try (RTT)? Will it solve the problem?

Right To Try (RTT) legislation has gained momentum and has passed in states across the United States in the last few years. While they differ from state to state, RTT laws generally say that drug companies can bypass FDA Expanded Access approval and provide (and often charge for) compassionate use. There is currently a movement to pass similar national legislation in the U.S. Congress.

Unfortunately, RTT laws miss the boat. They fail to recognize that the biggest problem is drug company reluctance — and sometimes unwillingness — to provide fair, consistent, and regular compassionate use. RTT laws fail to offer workable solutions to that problem.

Instead, RTT laws assume that government is the problem. But FDA approves over 99% of the requests it receives. RTT laws assume that by bypassing FDA, drug companies will be willing to provide the experimental drugs for compassionate use. But drug companies are focused on getting their new drugs approved by FDA and to market. They have little to no incentive to provide compassionate use to patients without FDA’s approval.

For a succinct discussion of why RTT laws will not solve the compassionate use problem, see melanoma patient Jonathan Friedlaender’s blog post on national RTT legislation. For further details, visit the publication and resources list at NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access.

ABOUT CURe


How will CURe help patients make sense of the compassionate use process?

CURe Info Hub will be a patient-centric online guide, written and designed for the patients and families facing compassionate use choices. Info Hub will

  1. Meet the needs of stressed out, sick, time-pressed, and exhausted patients and primary caregivers. It will offer good visual design, easy navigation, well-ordered information, and patient-friendly language.
  2. Educate on the whole process — from the first step to talk to your doctor about compassionate use to asking for help from a drug company to the formal request for FDA approval. This will include the “why’s” that underlie doctor, drug company, and FDA decision-making.
  3. Guide patients through considerations when making a series of difficult choices.
How will CURe get drug companies to say YES to compassionate use more consistently and fairly?

CURe Solution Hub will combine cutting edge design methods with traditional community building to bring together drug companies, patient advocates, clinical researchers, health care providers, insurance providers, policymakers, and scholars. Together, we’ll develop innovative and conventional win-win solutions to the barriers that drug companies face in providing compassionate use. We will harness policy, business models, and clinical research models to build practical and effective solutions.

The program will consist of four components:

  • White Paper to lay out risks and barriers faced by large and small drug companies
  • Solution Hack Workshops that bring together curated sets of leaders and innovators across stakeholder groups to work in small teams to develop the seeds of practical solutions around key barriers and risks that drug companies face
  • Build Out Promising Solutions through collaboration with stakeholders and by contracting expertise and analysis as needed to ensure a practical, impactful, and implementable solution
  • Implementing and Advocating our best solutions via pilot programs with partnered drug companies, discussions with influencers or policymakers, and other solution-appropriate means
Is CURe focused on compassionate use in the United States or globally?

Both, but we’re starting with the United States. Here’s why:

Because so many drug companies are global companies, their compassionate use policies are also global. The United States and Europe are mega-markets for drug companies, so changes made to drug company compassionate use policies in the U.S. and EU are likely to cascade to other regions.

INPUT: If you are working on collaborative problem-solving for compassionate use outside the United States, we want to hear from you. The more we can work together, the better job we’ll do at solving this challenge.

Does CURe run social media campaigns for patients seeking compassionate use?

We believe that social media campaigns have played an important role in raising public awareness and drug company awareness of the devastating problems with today’s compassionate use system. Granted, we are biased: CURe’s founders experienced the flaws in the compassionate use system first hand when we campaigned for compassionate use for our friends.

That said, we see compassionate use social media campaigns as a sign of patient and family desperation — a sign that the system is not working.

We decided we wanted to fix that system, so families don’t get to the point where they have to turn to social media as a last resort.

We do want to maintain public awareness of the frustration, anxiety, and devastation that compassionate use denials cause families. We are building a web page that will provide information on current and past campaigns. If you or someone you know of is running a current compassionate use campaign, please let us know.