Working Group on Compassionate Use and Pre-Approval Access




Biosketches of working group members can be found here.

General inquiries:
Lisa Kearns

Media inquiries:
Beba Blagojevic

Statement from the Working Group on passage of federal “right to try” bill (June 25, 2018)

Fact Sheet and Recommendations from the Working Group on Alternative Policies to Right to Try (updated March 6, 2018)

Our fact sheet from 2017 is available here

The Working Group’s 2017 Annual Report 

Examining Patient Access to Investigational Drugs” hearing before the U.S. House of Representatives' Energy & Commerce subcommittee on Health

Official written testimony from Alison Bateman-House and Kenneth I. Moch

The High School Bioethics Project at NYU Langone Health's Compassionate Use Module

Upcoming Events

“Ethics, the Right to Try, and Access to Unapproved Drugs”
Arthur Caplan
December 14, 2018
Kyoto University, Japan
Please contact Jessica Wico for more information.

Expanded Access Summit
Speakers include Lisa Kearns, Richard Klein, and Tom Watson
January 22-23, 2019, Washington, DC
Register and learn more here.

The NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access (CUPA) was founded in 2014 to explore the ethical issues surrounding access to investigational medical products before they’ve received regulatory approval, including: the fairness and transparency of access decisions, the role of social media, and legislative efforts to sidestep regulation of pre-approval access. Today, CUPA continues to work to advance knowledge about this topic, identify best practices, and bring to the forefront important ethical issues that need further attention.

Who we are

CUPA is chaired by Arthur Caplan, PhD, founding director of the NYU School of Medicine's Division of Medical Ethics, and Alison Bateman-House, PhD, MPH, MA assistant professor in the Division of Medical Ethics. Members, listed at right, represent a wide variety of fields, including medicine, law, academia, bioethics, government, industry, and patient advocacy. The Working Group studies pre-approval access globally.

What we do

CUPA’s main responsibilities, activities, and accomplishments include:

  • Working with patient groups to help demystify pre-approval access and create community-specific resources.
  • Publishing hundreds of articles in peer-reviewed journals and mainstream publications (click on the links at right for articles and interviews, as well as resources for patients and other stakeholders). Members have received extensive coverage in major media outlets including the L.A. Times, STAT, Harper’s Magazine, Forbes, and NPR, among others, and they speak frequently at conferences and seminars.
  • Serving as the go-to resource for issues related to pre-approval access. In addition to publications and resources, CUPA’s website features an educational module on pre-approval access and information on legislative and industry activities.

Our FAQ: Our list of frequently asked questions has served as an invaluable resource learning about compassionate use/pre-approval access and associated topics:

What is “compassionate use”?

“Compassionate use” is the same thing as “expanded access,” which is the same thing as “pre-approval access.” Regardless of which name you use, it is a pathway by which patients may use a drug, device, biologic, vaccine, or other type of medical product that hasn’t been approved for sale or use in the United States by the Food and Drug Administration (FDA). Compassionate use is the term typically used by the media, and expanded access is the legal term used by the FDA. We prefer the term pre-approval access because we think it makes clear what is being discussed: access to a medical product before it is approved for use, if such approval occurs. Outside of the U.S., there are additional terms used, like “named patient programs,” “named patient supply,” or “managed access.” We recently wrote a paper arguing for one term to be used by everyone to prevent confusion. So, we would prefer everyone use the term “pre-approval access.” However, in this FAQ we will use all of the terms interchangeably, assuming that readers won’t necessarily read the entire FAQ, and we want them, no matter where they start, to know what is under discussion.

Generally, patients in the U.S. access unapproved medical products by enrolling in a clinical trial. Pre-approval access does not involve enrolling in a clinical trial. (This is a little complicated, and we discuss it more thoroughly below.) For a variety of reasons, some patients cannot participate in a clinical trial. Terminally ill patients who are not able to participate in a clinical trial and who have no other treatment options may seek pre-approval access. So might those with debilitating non-terminal diseases or conditions, such as blindness, multiple sclerosis, or chronic pain. Also, patients with serious or terminal illnesses who have no other approved treatments available may use expanded access to seek an experimental drug or device that is not yet in clinical trials or for which the clinical trials are full or have concluded. Finally, in some cases, patients can seek access to drugs that lost their FDA approval due to new information via compassionate use requests. These would be patients for whom the drug’s benefits outweigh the risks that made the FDA decide against the general public having access to the product.

Some high-profile cases have brought pre-approval access into public awareness. In 2001, the Abigail Alliance for Better Access to Developmental Drugs was launched in memory of Abigail Burroughs, a 21-year-old cancer patient who died while trying to access an unapproved drug. In 2013, Andrea Sloan sought compassionate use of an ovarian cancer drug. Her request was initially denied; she appealed, and she died shortly after a company agreed to provide the drug. And in March 2014, the family of seven-year-old Josh Hardy waged an aggressive media campaign to secure pre-approval use of an antiviral drug from a small manufacturer to treat an opportunistic infection that he acquired while undergoing treatment for kidney cancer that he had suffered from since infancy. Although the company refused to grant the drug to Hardy via compassionate use, it created, with the help of the FDA, a new, open-label clinical trial of the antiviral and enrolled him in it. Hardy responded well to the drug and returned home. Sadly, he died of cancer in September 2016. These examples were all single-patient cases of pre-approval access, which we describe in more detail below. There are also cases of pre-approval access for groups, which we also talk about below.

Outside the U.S., policies for pre-approval use vary by country. This FAQ pertains only to the United States.

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What is an unapproved medical product?

In the U.S., drugs, medical devices, biologics, and vaccines are normally available for sale or use outside of a clinical trial only after they have been approved by the FDA, the federal agency charged with making sure medical products have been shown to be relatively safe and effective before they can enter the market. While these items are being developed and tested, they are called unapproved medical products. When a company has a new drug or device that it wishes to market, it needs to follow the FDA’s rules about how to get a product approved. For drugs, this involves going through a series of tests, both of safety and efficacy. Only after this testing (which entails several types of clinical trials) is completed and the data collected is reviewed by the FDA will the agency decide whether to approve the medical product.

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Who is eligible for pre-approval access?

FDA regulations specify two groups of people who are eligible for expanded access when no alternative treatment is available: (1) those with life-threatening diseases or conditions for whom “there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment”; and (2) those with serious diseases or conditions associated with “morbidity that has a substantial impact on day-to-day functioning.” More information can be found in the first section here.

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I've heard that FDA approval is very slow. What about people who need access to new medical products before they are approved?

Whether FDA approval of a new medical product is slow or not is a matter of debate and will not be covered here. What we are focused on is patient access to medical products that are unapproved. For patients seeking access to unapproved medical products, there are several possible routes of access. The most common one is to enroll in a clinical trial. You can get more information about that here. The less common one is pre-approval access, also called expanded access and/or compassionate use. And in some states, it is theoretically possible to access some investigational medical products via something called “right to try” laws, which we discuss below.

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What can you ask for through pre-approval access?

Unapproved medical products: drugs (including biologics), vaccines, and devices. More information can be found on the FDA website.

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Is pre-approval access a clinical trial?

The pre-approval access scenario that we are most familiar with from the media, movies, and the like is a request from a single patient. Individual-patient compassionate use is not a clinical trial. The purpose of an individual-patient compassionate use attempt is to treat a patient, not to collect data. For more information on this type of compassionate use, see the Code of Federal Regulations, §312.310.

The type of pre-approval access that we, as a society, are less familiar with deals with groups of patients with a similar disease or condition, such as boys with Duchenne muscular dystrophy. For more information on this type of pre-approval access, see CFR §312.315. Individuals within the defined group can sometimes gain access to an unapproved medical product by means of an “expanded access program” created by the company developing the product. For example, if a new drug to treat a deadly disease is nearing the end of the clinical trials testing process, and the trial data suggest that the drug is safe and effective, the company may start an expanded access program (EAP) to make the drug available to those who will be harmed by waiting for the drug to receive FDA approval before they begin using it. Furthermore, there is frequently a time lag between FDA approval of a new drug and when it starts being stocked in pharmacies or when insurance companies agree to start paying for it. During that lag time, patients can access the (now approved) drug through expanded access programs. Expanded access programs close down once a drug is available in pharmacies.

Some expanded access programs ARE types of clinical trials. They do not have many of the features that we think of as typical of clinical trials (for example, patients are not randomized to receive other treatments or a placebo); however, they do involve the collection of clinical data about the participating patients. Patients who seek pre-approval access are typically in worse health than patients in clinical trials (because patients seeking pre-approval access are ineligible for clinical trials, often due to the severity of their condition or because they have additional complications). By collecting data on the patients in an expanded access program, the company developing the new product can get an idea of how it works on patients sicker than those on whom the clinical trials were conducted. This information can be useful in predicting how the product will work in the “real world,” after it has been approved by the FDA.

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Are patients in expanded access programs in a clinical trial or are they receiving the drug through pre-approval access?

Expanded access program have elements of both clinical trials and compassionate use programs. As in clinical trials, there are rules about who is eligible to enroll, and patients’ physicians must report to the company certain information about the drug, including any side effects and how well it is working. However, the rules about who is allowed in an expanded access program are frequently not as strict as those about who may be allowed to join a clinical trial, and the main goal of the expanded access program is to help the patients in it, rather than the development of knowledge. If, for whatever reason, a patient is not able to access a drug through an expanded access program, he or she may then ask to get it through single-patient compassionate use.

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Who is in charge of pre-approval access decisions?

In the U.S., the companies developing the new medical product are the key decision-makers about whether to allow access to an investigational drug or device. Physicians channel requests to the appropriate company and provide information about the requesting patient. If the company agrees to provide a product to a physician for a particular patient, two more approvals must be secured: that of the FDA and, unless use of the investigational medical product in a single patient is deemed an emergency, that of an institutional review board's (IRB). (For emergency requests, physicians have five days to notify, and submit paperwork to, their institutions’ IRBs after the treatment attempt has started.)

The FDA reviews proposed use of experimental medical products to make sure there is sound medical reasoning behind the proposal and no obvious indications that the product will harm the patient. The FDA does not "approve" pre-approval proposals; rather, it allows them to proceed or not. More than 99% of expanded access proposals are allowed to proceed. However, in some circumstances a proposal is altered in response to feedback from the FDA reviewers. Jarow et al. looked at a random sample of 150 proposals and found that 11% had been altered in response to FDA input. Such alterations were along the lines of changing the proposed dosing schedule and altering informed consent forms.

Congress has been looking at streamlining the IRB approval process for individual patient expanded access. Pursuant to congressional legislation, in the fall of 2017 the FDA announced new rules on this topic.

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If it’s “compassionate,” why does the issue generate so much controversy?

Several reasons. Access to investigational medical products is not always fairly granted. For example, those with the financial resources or special medical or political connections may seek and gain preferential access. Those who know how to utilize social media may have an advantage over those who don’t. (The Josh Hardy case is a good illustration of this.) Similarly, not every doctor, much less patient, knows about compassionate use or how to request it.

Another key issue is the risk that pre-approval access poses to the clinical trial process and the timeline for FDA approval. Granting individuals access to investigational medical products can jeopardize an ongoing clinical trial that may eventually produce a treatment that could help many future patients. If patients had a choice between gaining access to a desired experimental medical product via either expanded access or a clinical trial, they would likely choose expanded access, especially if the trial involves the possibility of being randomized to a placebo or some other treatment. So, there must be clear rules about who would be allowed to access a drug via expanded access and who would not. Additionally, diverting some of a scarce drug from clinical trials to compassionate use may result in an insufficient amount of the product to permit the clinical trial to be done in a timely manner. Delaying the trial would delay possible FDA approval of the new product, and, ultimately, the availability of the new drug for patients who need it.

Finally, because the investigational medical products being sought by patients have not been thoroughly tested (unless someone is seeking access to a drug that is in Phase 3 clinical trials or for which trials have concluded), their efficacy and side effects are often unknown, especially in the very sick people who often would not be eligible to participate in a clinical trial. As such, the experimental treatments may cause further harm and suffering, hasten death, or do absolutely nothing. Very bad outcomes (like deaths) in patients who try an unapproved product via expanded access could have serious repercussions for the investigational medical product, which could be blamed—even if the poor outcome happened because the patient was seriously ill. The FDA says it does not hold adverse events associated with compassionate use against a drug unless it is reasonable to do so, but some companies are afraid to take this risk and, as a result, avoid compassionate use. The FDA has recently conducted audits of its processes and is using that data to try to assure pharmaceutical manufactures that the likelihood of an experimental drug being derailed by bad outcomes in the compassionate use setting is extremely unlikely.

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How else could granting a dying person access to an unapproved medical product harm an ongoing clinical trial?

Several ways. If patients learn that pre-approval access is a possibility, they may not be willing to enroll in a clinical trial, fearing they’ll receive a placebo or standard-of-care treatment instead of the desired product. If people had a choice to receive the investigational medical product they wanted through a compassionate use program or by entering a clinical trial, it is not surprising that they would choose the compassionate use program—after all, they would know for sure what they were receiving. While that may be the most rational choice for an individual, it could imperil the availability of patients for clinical trials, which would have dire consequences for the drug development process and for future patients.

Many manufacturers have limited supplies of their investigational products, and granting compassionate use access can threaten those supplies. If they run out of supplies, manufacturers may have to scale back or suspend clinical trials. Especially with biologics, it is no easy task to “just make more drug.” Some biologics can take more than a year to produce, and there are limited numbers of manufacturing facilities that can perform these complex tasks. Other drugs are hugely costly to make.

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What are “right-to-try” laws?

Legislation introduced in the U.S. at the state and federal levels that purports to grant terminally ill people access to investigational drugs and devices without FDA oversight. The movement was created by the Goldwater Institute, a libertarian think tank; it created a model right to try bill for states to use in crafting their own right to try laws, and most of the laws and bills follow the model bill closely, although there are differences. For example, one state doesn't allow inpatients to request drugs under right to try, something not specified in the model bill, and many bills' definition of "terminal illness" differs from the model's. Additionally, 19 states allow insurers to deny hospice coverage for patients using a drug obtained via right to try, 7 allow coverage for home health aides to be denied, and 4 states may deny health insurance benefits to patients who try an investigational drug obtained via right to try. S.204 — The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 was signed into law on May 30, 2018.

Right to try laws have generated a lot of attention since the first one was enacted in Colorado in May 2014. They are now on the books in 40 states. Although advocates praise these laws for ostensibly throwing a lifeline to terminal patients, the laws don't require a manufacturer to provide investigational medical products upon request, so, in actuality, they are no more of a guaranteed lifeline than is the FDA program that right to try seeks to undercut. An article by Working Group members Lisa Kearns and Alison Bateman-House analyzed the variations in provisions among the first 32 state laws and found that these laws offer few tangible benefits to patients, despite their patient-centric rhetoric.

The laws' backers claim that the legislation speeds up the process of getting investigational medical products to patients by removing the FDA from the pathway to access. In the assessment of the Working Group, this is misleading, as FDA review is quick (within 24 hours for emergencies and with a median time of 4 days for non-emergency requests). Furthermore, cutting the FDA out of the picture by means of state laws is likely unconstitutional, which is why right to try advocates are now pushing for federal legislation. Too, although companies may wish to fulfill patient requests, they may see no upside to providing their investigational products without the FDA’s permission. If the products work, companies want to be able to use that information when they seek marketing approval from the FDA, and if the products do not work, companies want to be able to say that there was oversight of the use of their investigational product on a sick or dying patient.

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How many patients have been helped by right-to-try laws so far?

According to the Goldwater Institute and Dr. Ebrahim Delpassand, the founder of Excel Diagnostics and Nuclear Oncology Center, between 80 and 100 patients have gained access to an agent called Lu-177 Dotatate under the Texas right to try law. Yet because this substance was already available via an expanded access program set up by the company, we do not accept this claim as evidence of one or more patients receiving otherwise unavailable drug(s) because of a right to try law. To the best of our knowledge, no patients have been spared from death by right to try laws.

People in states with right to try laws may be receiving access to investigational medical products, but it is done via the FDA's "expanded access" provisions, which predate right to try laws. And the FDA isn't the problem right to try supporters make it out to be. As mentioned above, over the past six years the agency has granted more than 99% of all pre-approval requests, and approval for drugs to treat rare or life-threatening diseases are, as the New York Times put it, "the rule, not the exception" (see the article, and a chart showing the agency's approval trajectory since 1990, here). Not only does the FDA respond to individual patient pre-approval access requests very quickly; in June 2016 the agency released a significantly streamlined expanded access application, reducing the time needed to complete it to less than an hour. The FDA also lends important expertise to the process, including alterations to submitted protocols such as altering dosing schedules.

Right to try laws raise an important ethical concern: Because drug makers aren’t required to grant access to their investigational products, the laws create the false hope in desperately ill people that they can obtain something they may, in fact, not be able to get. And, some state right to try laws strip patients who use right to try of important benefits, such as health insurance or access to home healthcare.

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Who could possibly object to dying people trying any means available to save their lives?

No one, but you can certainly object to falsely raising patients' hopes that they have a “right” to investigational medicine, when the truth is that the drug company gets to decide whether or not to provide it. You can object to patients experiencing increased suffering or possibly an earlier death after not being fully informed of the risks they were taking or aware of conflicts of interest that might be influencing the advice they were given. Also, many right to try laws and bills stipulate that if patients receive access to an unapproved medical product, they are at risk of losing hospice care, home healthcare, or even insurance coverage. These provisions are not well publicized, so patients may not understand what they risk.

Many people might also object to granting individual requests at the expense of enrolling participants in clinical trials, which could delay or even halt the development of treatments that have the potential to help larger numbers of future patients. PhRMA (Pharmaceutical Research and Manufacturers of America), a trade group representing companies in the pharmaceutical industry in the United States, has expressed concerns about the legislation, both because it threatens the integrity of clinical trials and because it considers FDA oversight of investigational products to be in the best interest of patients.

It is also the case that unscrupulous health care providers might try to take advantage of right to try laws to sell nostrums and quack cures to desperate patients and their families. Right to try legislation requires only that a Phase 1 safety study has been done, and that's flimsy protection from shysters and crooks. Despite the fact that Phase 1 studies are often called "safety studies," the completion of a Phase 1 study does not guarantee that an experimental drug is safe. Most Phase 1 studies use paid, healthy volunteers to determine the appropriate dosage for future studies, without conducting a long-term follow-up. As such, there is little information available about how these experimental drugs may work in sick patients. While patients, particularly the terminally ill, may be understandably willing to take on risk to try a new treatment, we need to ensure that they understand that Phase 1 studies are no guarantee of safety and that prior to the completion of Phase 2 studies, there is no information about a drug's efficacy. Using a drug after just Phase 1 testing is truly in a leap into the unknown.

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Still, given the length of time it takes the FDA to approve new therapies, why not remove the middleman—the FDA—from the compassionate use equation?

First, there's a real reason for FDA oversight. In 1962, the thalidomide incident spurred legislation that forces drug companies to prove to the FDA that their products are safe and effective before they can be approved. And bear in mind that there will always be hucksters and snake oil salesmen peddling anything as a cure to desperate people. This sadly exists in the unregulated arena of stem cell "therapies," which have been portrayed as cures for nearly everything in some nations. The FDA helps prevent an increase in attempts to manipulate and exploit those desperate for a cure or treatment for their ailments.

Importantly, the FDA review can be helpful to patients. Jarow et al. looked at a random sample of 150 proposals and found that 11% were altered in response to FDA input, including, for example, changing the proposed dosing schedule. When trying an investigational drug, a patient's doctor is working with very limited knowledge, so the more insight provided about how to use the drug and how to treat any side effects, the better. And once the investigational treatment has been given to the patient, the doctor has an obligation to notify the drug company and the FDA of any serious, unanticipated, adverse outcomes suffered by that patient.

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If the FDA permits more than 99% of compassionate use requests to proceed, do we really need it to review these proposals?

Yes. While the FDA almost always permits a compassionate use request to go forward, FDA personnel weigh in on aspects of the proposal, such as the dosage to be used, the dosing schedule, and the like. Based on the FDA's knowledge of the experimental agent and how it worked in any previous usage in people, the agency can help the physician to adjust the proposed treatment plan. This is an important step in trying to help patients because the FDA employs expert scientists and physicians who specialize in clinical research protocols, while a patient's treating physician may not have this experience. So, FDA approval is not a “rubber stamp”; it is a necessary collaboration between experts in the administration of investigational products and a patient's treating physician.

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If right-to-try laws don’t help get treatments to sick patients, then why have supporters been pushing them so hard?

The prevailing theory is that interested parties wanted to try to force federal action—that is, federal legislation that would limit the FDA’s oversight of the entire drug approval process through generating a groundswell of state activity. The Yale Law Journal made just this case early on, in December 2014. A federal right to try bill was first introduced in July 2015, lending more weight to this theory.

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Why do so few people in the U.S. get access to experimental drugs via pre-approval access? Just over a 1,000 people per year in our entire country versus thousands in France, a much smaller country?

Comparing the numbers of patients treated with experimental agents in the U.S. and France is an apples-to-kumquats comparison. It is inappropriate to compare these two countries for one simple reason: France has a national healthcare system and the US does not. Not all Americans have access to centers of clinical excellence and/or research specialty care. It would be a miracle for a patient in a safety net hospital to be given the option of trying an unapproved drug via compassionate use. These hospitals rarely participate in clinical trials, do not have the infrastructure necessary to treat patients with experimental drugs, and, more to the point, see a tremendous volume of patients, even for complex specialties like oncology. There is no institutional support for anything but standard care, and patients have no option to leave there and go to a private hospital. As such, an entire economic class is ruled out of access to investigational drugs. This has nothing to do with FDA rules about access to experimental drugs; it has to do with a system that was never set up to provide every patient with cutting edge, including experimental, treatment. It is not fair that access to unapproved drugs is only for those who know about the possibility; can afford the "right" physician and hospital; and can pay costs associated with the process that insurance may not pay or are lucky/appealing enough to find themselves the beneficiaries of a charitable endeavor. Thus, a sensible way to help patients would be to deal with these systemic inequalities.

Other reasons that comparing the number of patients treated with experimental agents in the U.S. and France is an apples-to-kumquats comparison include the facts that 1) the French health system pays for investigational agents while most private and public insurance systems in the U.S. do not, 2) drugs are approved faster in the U.S. than in France (which means some of the agents obtained through pre-approval access in France are available in pharmacies in the U.S.), and 3) many patients travel to France from all over Europe to get pre-approval access to experimental medicines.

Despite the fact that large numbers of Americans are not situated to be able to seek experimental drugs for compassionate use, there are still many patients who both seek this access and receive it. The FDA gets more than 1,000 requests a year, but a request can be a protocol for one patient or many patients. A recent report from the U.S. Government Accountability Office on the FDA’s expanded access program shows that in fiscal years 2012 through 2015, the FDA approved 5,697 compassionate use protocols. 5,452 of these approved protocols were for single patients, but 245 were for multiple patients. Of these 245 multi-patient protocols, 194 of the approved protocols were deemed "intermediate" (defined as more than one but less than many) in size, and 51 were deemed large. Without knowing the names of the drugs, we cannot know the number of patients involved in these intermediate and large requests; however, it is not unheard of to have more than 1,000 patients in a large compassionate use program. So, nobody knows exactly how many patients receive pre-approval access in our country, but it is certainly more than just the number of pre-approval requests permitted by the FDA.

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Who is on the NYU Langone Health's Working Group on Compassionate Use and Pre-Approval Access, and what do they hope to accomplish?

Members of the Working Group are listed here. The Group works to raise awareness of the many ethical issues surrounding compassionate use and other efforts to facilitate access to investigational medical products prior to their approval by the FDA. In order to do this, and to understand the perspectives of all of those involved in this issue, the Group works closely with patient advocacy organizations, large and small pharmaceutical and biotechnology companies, venture capitalists who fund such companies, and a host of other organizations, including the FDA and various trade organizations. The Group is currently working to develop connections with physicians and IRB professionals in order to better understand compassionate use and pre-approval access from the perspectives of those groups.

Where it sees room for improvement, the Working Group seeks to effect policy changes that will make the process more realistic, more equitable, and more transparent for those seeking access to investigational medical products.

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Does the Working Group talk with legislators or other policy makers?

The Working Group talks with anyone who has insight into pre-approval access or ideas about how to improve the process for those seeking access to investigational medical products. We have communicated with both state and federal legislators and FDA leadership. We answer questions, share what we have learned, and seek to provide education. Neither these conversations nor this document represents an official view of NYU or any employer or other institutional affiliation of any Working Group member. However, Working Group members are not precluded from speaking their own minds in an individual capacity or from writing editorial or position papers.

The Working Group has also provided input and advice on pre-approval access to patient advocacy groups and pharmaceutical companies, as well as legislators and others. Some of those whom we have advised include: State Sen. Ted Kennedy Jr. (CT), U.S. Sen. Tom Carper (DE), the ALS Emergency Treatment Fund, Janssen Pharmaceuticals, the Hyundai Hope on Wheels Thought Leader's Summit, Assemblywoman Linda Rosenthal (NY), U.S. Rep. Michael McCaul (TX), BIO, the Florida Dept. of Health, and the Energy and Commerce Committee of the U.S. House of Representatives.

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Has the Working Group found any positive proposals to make for those seeking compassionate use?

Early in its life, the Working Group stated that companies should make their pre-approval access policies publicly accessible. Companies should provide a telephone number, email address, or other designated point of contact to which requests for compassionate use should be directed. Also, companies should set a reasonable time frame for making a decision on a request, to convey this time frame to requesters, and to report decisions to requesters in a timely manner. These recommendations were adopted by legislators and incorporated into the 21st Century Cures legislation passed in December 2016.

In April 2017, the Working Group published a fact sheet about right to try laws and a list of proposals for helping those patients who wish to try unapproved medical products outside of clinical trials. This document identified key areas for legislators to address. In response to this proposal, the 2017 FDA User Fee Renegotiation Act mandated a public meeting with the National Institutes of Health and the FDA to discuss potential barriers to clinical trial and expanded access participation. The Act requires that the FDA publish a report on the topics discussed at this meeting and issue new draft guidance on broadening clinical trial and expanded access eligibility. Another component of the Act also stemmed from the Working Group’s proposal: a requirement that the FDA revise and streamline federal regulations that govern IRB approval for single-patient expanded access protocols. Several months after the Working Group released its proposals, the FDA announced that a single IRB member—rather than the full committee, as federal regulations previously required—may review and approve a single-patient expanded access protocol, as assembling the full board to review these requests may cause delays for those patients waiting to be treated with an unapproved medical product. The Working Group updated its fact sheet in March 2018 prior to the House vote on H.R.5247, which ultimately did not pass. 

The Working Group also called for legislators to clarify the FDA's authority over experimental drugs, as state right to try laws have unintentionally, but not unforeseeably, created confusion for healthcare professionals and organizations about whether to follow state or federal rules governing access to experimental drugs outside of clinical trials. These issues were discussed at a U.S. House of Representatives’ Energy & Commerce Committee’s Subcommittee on Health hearing on October 3, 2017, on the proposed federal right to try bills, where CUPA members Alison Bateman-House and Kenneth I. Moch testified.

The Working Group has also called for legislators to explore, with the pharmaceutical and biotechnology industries, ways to make pre-approval access a more appealing prospect to large and small companies. Companies are concerned that making experimental drugs available for expanded access could threaten their drug development timeline, turn off investors, and/or get them in trouble with the FDA. These areas are actionable for legislators and have yet to be undertaken.

The working group continues to evaluate new solutions to extending pre-approval access to investigational medical products and to making access a more appealing prospect for the pharmaceutical/biotechnology industry.

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Who pays for the Working Group?

Working Group members receive no compensation for their work on this committee. Overhead costs and other expenses are paid by the Division of Medical Ethics at NYU Langone Health, which receives income from a variety of sources, including private gifts and contracted projects.

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What publications have Working Group members issued?

Members of the Working Group have written and spoken extensively about compassionate use, pre-approval access, and related subjects, including FDA regulations, right to try laws, and more. You’ll find a link to these articles here.

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The Working Group’s co-chairs, Dr. Arthur Caplan and Dr. Alison Bateman-House, are part of NYU Langone Health’s Compassionate Use Advisory Committees (CompAC). Is this a conflict of interest?

CompAC, which was formed by the NYU School of Medicine, is independent from the Working Group. The idea for CompAC came from discussions that occurred in part among Working Group members, but the two are separate entities.

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If I’m interested in gaining compassionate use access to a drug or device, what should I do?

Talk to your physician. Although you can research investigational medical products online, you will need your physician to make the request.

If you or your physician need guidance about the process, you can call the FDA's Expanded Access Program Liaison at 301-796-8460.

The Reagan-Udall Foundation for the FDA has developed an online Expanded Access Navigator that provides step-by-step instructions on how to identify available investigational medical products and how to apply for individual access. Currently, the Navigator lists only oncology agents, but it will be growing to include rare disease agents.

If you or your physician need someplace to get started, search on using the keywords “expanded access,” “compassionate use” and “pre-approval access.”

If you are interested in a particular experimental medical product, search the website of the manufacturer of that product for the company's access policy. By law, many drug companies are now required to make such policies public.

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The Working Group sends out occasional updates about compassionate use and pre-approval access. To receive these, please contact Beba Blagojevic. For media inquiries, please contact Beba Blagojevic (Division of Medical Ethics) or Lisa Magid (Institutional Communications).

If your patient advocacy group or organization would like to host an “Ask an Expert” session on any of these topics, please contact Lisa Kearns.