United States: Developments in Compassionate Use and Right To Try Laws

Last Updated: June 9 2016
Article by James Beck

We blogged a couple of years ago about the beginnings of what has become a wave of state "Right to Try laws" – laws that purport to give terminal patients with no other medical options the right to seek investigational drugs for their conditions from manufacturers who have yet to obtain full FDA approval.  Mostly, these laws are motivated by the unwieldy nature of the FDA's "compassionate use" regulations, which are directed at the same problems.  Given that these laws originated with a "states' rights group" called the Goldwater Institute, we strongly suspect that a second motive of gratuitously poking the FDA in the eye was also at work.

Whatever their provenance, we were deeply skeptical of their practicality. Three large obstacles loomed.  Number one:

For one thing, there's the FDA. States can pass all the laws they want, but unless the FDA gives its okay to programs more expansive than its compassionate use ("expanded access") program, nothing's going to happen.

Number two:

The reason, as is the case for so many things these days, is the threat of liability. . . . You won't induce a manufacturer to participate in a voluntary program by painting a target on its back.

Number three:

There's no upside. These statutes are for use by very ill people, and if (as is unfortunately likely) the statutory participant died, then there's an adverse event that must be reported to the FDA.  Companies investigate drugs in the hope of obtaining approval.  Adverse events definitely don't help get approval.

Indeed, despite a couple dozen Right to Try statutes enacted over the last few years, we are unaware of even a single instance in which anybody successfully obtained treatment with an investigational drug under any of these state laws.

A couple of recent developments might favorably affect this bleak scenario, however. One is newly introduced legislation (as of May 10, 2016), called the "Trickett Wendler [an ALS victim] Right to Try Act," and more pedantically known as S. 2912, which seeks to remove some of the obstacles identified above that hamstring the states' efforts.  S. 2912 is quite short, but it does two things.  First, it grants full immunity from suit to those acting under a state Right to Try statute:

Notwithstanding any other provision of law, no liability shall lie against a producer, manufacturer, distributor, prescriber, dispenser, possessor, or user of an experimental drug, biological product, or device for the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device that is in compliance with subsection (a) [pertaining to Right to Try statutes].

Id. §2(b)(1).  Getting the plaintiffs' lawyers out of the way might allow lives to be saved.

Second, the statute removes the disincentives created by FDA reporting requirements for investigational drugs. S. 2912 prohibits use of adverse events involving Right to Try participants from negatively affecting possible FDA approval:

Notwithstanding any other provision of law, the outcome of any . . . use of an experimental drug, biological product, or device that was done in compliance with subsection (a) shall not be used by a Federal agency . . . to delay or otherwise adversely impact review or approval of such experimental drug, biological product, or device.

Id. §2(b)(2).  Ideally, we'd like to see this protection extend to inadmissibility at trial, since some plaintiff expert could still assert such an adverse event as a supposed "red flag" in other civil litigation, but this bill would be a big step forward on the liability front.  A federal-law umbrella like S. 2912 would eliminate the patchwork of liability provisions in the state statutes that we discussed at some length in our prior post.

That leaves the third piece of the puzzle – the FDA.

There's been movement on this front as well. In what we doubt is a coincidence, the FDA released – this month – two new guidance documents that bear on the general issue of compassionate use of investigational drugs.  The first, entitled "Expanded Access to Investigational Drugs for Treatment Use − Questions and Answers," can be found here.  The second, entitled "Charging for Investigational Drugs Under an IND − Questions and Answers," is available here.  These are, of course, "nonbinding recommendations," but they do indicate that the FDA is at least responding to the dissatisfaction with the current state of affairs that has been driving the Right to Try movement over the last several years.

We learn from the first guidance that the term "compassionate use" is out, replaced by the dryer phrase "expanded access." Expanded Access Guidance, at Q1.  "Expanded access" is therapeutic, not investigational, in its purpose, and the more formal "treatment IND" is only available if the drug's manufacturer ("sponsor") is pursuing eventual approval of the particular use involved. Id. Not only does the guidance cover drugs for which approval is being sought, but also drugs that have "been withdrawn for safety reasons," if they might help particular patients. Id.

Two of the processes described in the first guidance, the "expanded access protocol" and the "new treatment IND" must be initiated by the manufacturer and thus means more work for it – with no immediate benefit – not a good combination for what are, after all, for profit entities. Id. at Qs 2-5.  They also require use of institutional review board review. Id. at Q6.  These processes thus are not particularly relevant to what the state Right to Try statutes are seeking to accomplish, which is patient- or doctor-initiated individualized therapy.  The guidance also includes information on how patients who are enrolled in a clinical trial may seek to continue treatment with the investigational drug after the trial ends or something happens that puts them outside of the study's inclusion criteria. Id. at Qs 27-29 (discussing "open-label" studies).

More promising from this point of view is "individual patient expanded access," in which the "sponsor/investigator" is the doctor of the patient for whom all other therapies have failed. Id. at Q9.  It uses a form (FDA 3926) "created specifically for individual patient IND submissions, including those for emergency use." Id. While this route can be used by a manufacturer, id. at Q10, it can also be initiated by a treating physician:

A physician can submit an individual patient expanded access IND for his/her patient. In this scenario, when the patient's physician submits an expanded access IND, the physician is both the sponsor and the investigator − in other words, he or she is considered a sponsor-investigator.

Id. This method can even be used for an investigational drug where the manufacturer does not have an active IND (assuming that the manufacturer is willing to provide the drug). Id.

In individual patient expanded access, the patient's treater "must determine that the probable risk to the patient from the investigational drug is not greater than the probable risk from the disease or condition." Id. at Q11.  In a Right to Try situation where the condition is fatal, this criterion would ordinarily be satisfied.  The FDA, however, retains the final say.  "FDA must determine, based on the information available to FDA, that the potential benefit justifies the potential risks." Id. One potential reason the FDA could do so is if the expanded access would somehow "interfere" with the ongoing approval process for the drug. Id. at Q24.

Further, "individual patient expanded access is generally limited to a single course of therapy for a specified duration," id. at Q13, although more than one investigational drug may be involved. Id. at Q31..  That effectively means one and done, except in rare instances of a "chronic disease or condition that requires extended treatment" where "the circumstances of the treatment are well defined and reasonable in light of the available evidence." Id. at Q13.  In Right to Try situations, this is unlikely to be the case.  Whether expanded access is available for many investigational drugs can be determined at clinicaltrials.gov, since all sponsors who register their trials on the site are asked for their positions (not all ongoing studies are on the site, however). Id. at Q30.

Just like all of the Right to Try statutes are voluntary – no manufacturer can be ordered to supply investigational drugs – so is the FDA's new program. Congress has not given the FDA the power to order participation.  "FDA cannot compel a company to provide expanded access to its drug. When a company provides expanded access to its drug, it does so voluntarily." Id. at Q23.  The FDA's guidance does not create, or even recommend, any form of tort immunity for participants.

Similarly, the guidance retains FDA power to use any adverse events against the manufacturer in its ultimate determination whether to approve the investigational drug. Any time a treater initiates individual patient expanded access, s/he assumes responsibility to "provide to FDA a written summary of the results of the expanded access use, including adverse effects." Id. at Q25.  Those adverse events may adversely affect FDA evaluation of the drug:

From a public health perspective, early identification of important adverse events is beneficial. For example, a relatively rare adverse event might be detected during expanded access use, or such use might contribute safety information for a population not exposed to the drug in clinical trials.  There are a small number of cases in which FDA has used adverse event information from expanded access in the safety assessment of a drug.

Id.  Thus, the FDA's guidance does not address either of the two main drawbacks to manufacturer willingness to participate in expanded access – possible adverse effect on approval and exposure to litigation over investigational drugs.

Next, a bit on the FDA's second guidance, on charging money for investigational drugs.  Ordinarily manufacturers/sponsors are prohibited from "commercializing" investigational products.  21 C.F.R. §312.7(b).  Since 1987, however, the FDA has allowed them to recover costs in some situations. See 21 C.F.R. §312.8.  This new guidance addresses such charges are calculated, and also expands the ability to charge into expanded access situations.  Charging for Investigational Drugs Guidance at Q3.  Since this post is directed only to the expanded access/Right to Try situation, we're only looking at that part of the guidance.  To charge a fee for investigational drugs provided through an expanded access process, the manufacturer must:

  • Provide "reasonable assurance" to the FDA that the charges will not interfere with drug development.
  • Provide the FDA with "calculations" used to arrive at the amount charged that are consistent with the requirements in §312.8(d) limiting recovery to direct costs (those specifically and exclusively attributable) of making the drug available to these patients – that is, the incremental manufacturing and shipping costs, not costs of development. Such submissions consist of invoices and a accountant's statement.

Id. at Q13.  Only a years' worth of the drug can be charged without the FDA reevaluating. Id. at Q14.  Costs of drugs provided under individual patient expanded access may be recouped in this fashion, but specified "indirect" costs may not be recovered. Id. at Q17.

Finally, our take on the situation. We think that the proposal in S. 2912 is recognition of what we thought before – that individual state Right to Try statutes are unlikely to achieve their goal of broadening access to investigational drugs because they don't (and indeed cannot) alter manufacturers' risk/benefit ratios enough to induce them to participate in these voluntary programs.  The proposed statute would eliminate two of the biggest obstacles.  The FDA's new guidance documents help somewhat, but as long as adverse events from use of investigational drugs outside clinical trials can imperil the whole point of the exercise – eventual FDA approval (or expanded approval) – voluntary participation remains unlikely.  The FDA's guidance documents also pointedly ignore even the existence of these state statutory initiatives, so it is unclear what, if any, role state law is able to play.  For example, all of the state statutes include informed consent provisions, but whether they would satisfy the FDA's informed consent requirements is not addressed.  The Gordian Knot of compassionate use of investigational drugs thus remains tied around the medicine cabinet.

This article is presented for informational purposes only and is not intended to constitute legal advice.

To print this article, all you need is to be registered on Mondaq.com.

Click to Login as an existing user or Register so you can print this article.

James Beck
In association with
Related Video
Up-coming Events Search
Font Size:
Mondaq on Twitter
Register for Access and our Free Biweekly Alert for
This service is completely free. Access 250,000 archived articles from 100+ countries and get a personalised email twice a week covering developments (and yes, our lawyers like to think you’ve read our Disclaimer).
Email Address
Company Name
Confirm Password
Mondaq Topics -- Select your Interests
 Law Performance
 Law Practice
 Media & IT
 Real Estate
 Wealth Mgt
Asia Pacific
European Union
Latin America
Middle East
United States
Worldwide Updates
Check to state you have read and
agree to our Terms and Conditions

Terms & Conditions and Privacy Statement

Mondaq.com (the Website) is owned and managed by Mondaq Ltd and as a user you are granted a non-exclusive, revocable license to access the Website under its terms and conditions of use. Your use of the Website constitutes your agreement to the following terms and conditions of use. Mondaq Ltd may terminate your use of the Website if you are in breach of these terms and conditions or if Mondaq Ltd decides to terminate your license of use for whatever reason.

Use of www.mondaq.com

You may use the Website but are required to register as a user if you wish to read the full text of the content and articles available (the Content). You may not modify, publish, transmit, transfer or sell, reproduce, create derivative works from, distribute, perform, link, display, or in any way exploit any of the Content, in whole or in part, except as expressly permitted in these terms & conditions or with the prior written consent of Mondaq Ltd. You may not use electronic or other means to extract details or information about Mondaq.com’s content, users or contributors in order to offer them any services or products which compete directly or indirectly with Mondaq Ltd’s services and products.


Mondaq Ltd and/or its respective suppliers make no representations about the suitability of the information contained in the documents and related graphics published on this server for any purpose. All such documents and related graphics are provided "as is" without warranty of any kind. Mondaq Ltd and/or its respective suppliers hereby disclaim all warranties and conditions with regard to this information, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. In no event shall Mondaq Ltd and/or its respective suppliers be liable for any special, indirect or consequential damages or any damages whatsoever resulting from loss of use, data or profits, whether in an action of contract, negligence or other tortious action, arising out of or in connection with the use or performance of information available from this server.

The documents and related graphics published on this server could include technical inaccuracies or typographical errors. Changes are periodically added to the information herein. Mondaq Ltd and/or its respective suppliers may make improvements and/or changes in the product(s) and/or the program(s) described herein at any time.


Mondaq Ltd requires you to register and provide information that personally identifies you, including what sort of information you are interested in, for three primary purposes:

  • To allow you to personalize the Mondaq websites you are visiting.
  • To enable features such as password reminder, newsletter alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our information providers who provide information free for your use.

Mondaq (and its affiliate sites) do not sell or provide your details to third parties other than information providers. The reason we provide our information providers with this information is so that they can measure the response their articles are receiving and provide you with information about their products and services.

If you do not want us to provide your name and email address you may opt out by clicking here .

If you do not wish to receive any future announcements of products and services offered by Mondaq by clicking here .

Information Collection and Use

We require site users to register with Mondaq (and its affiliate sites) to view the free information on the site. We also collect information from our users at several different points on the websites: this is so that we can customise the sites according to individual usage, provide 'session-aware' functionality, and ensure that content is acquired and developed appropriately. This gives us an overall picture of our user profiles, which in turn shows to our Editorial Contributors the type of person they are reaching by posting articles on Mondaq (and its affiliate sites) – meaning more free content for registered users.

We are only able to provide the material on the Mondaq (and its affiliate sites) site free to site visitors because we can pass on information about the pages that users are viewing and the personal information users provide to us (e.g. email addresses) to reputable contributing firms such as law firms who author those pages. We do not sell or rent information to anyone else other than the authors of those pages, who may change from time to time. Should you wish us not to disclose your details to any of these parties, please tick the box above or tick the box marked "Opt out of Registration Information Disclosure" on the Your Profile page. We and our author organisations may only contact you via email or other means if you allow us to do so. Users can opt out of contact when they register on the site, or send an email to unsubscribe@mondaq.com with “no disclosure” in the subject heading

Mondaq News Alerts

In order to receive Mondaq News Alerts, users have to complete a separate registration form. This is a personalised service where users choose regions and topics of interest and we send it only to those users who have requested it. Users can stop receiving these Alerts by going to the Mondaq News Alerts page and deselecting all interest areas. In the same way users can amend their personal preferences to add or remove subject areas.


A cookie is a small text file written to a user’s hard drive that contains an identifying user number. The cookies do not contain any personal information about users. We use the cookie so users do not have to log in every time they use the service and the cookie will automatically expire if you do not visit the Mondaq website (or its affiliate sites) for 12 months. We also use the cookie to personalise a user's experience of the site (for example to show information specific to a user's region). As the Mondaq sites are fully personalised and cookies are essential to its core technology the site will function unpredictably with browsers that do not support cookies - or where cookies are disabled (in these circumstances we advise you to attempt to locate the information you require elsewhere on the web). However if you are concerned about the presence of a Mondaq cookie on your machine you can also choose to expire the cookie immediately (remove it) by selecting the 'Log Off' menu option as the last thing you do when you use the site.

Some of our business partners may use cookies on our site (for example, advertisers). However, we have no access to or control over these cookies and we are not aware of any at present that do so.

Log Files

We use IP addresses to analyse trends, administer the site, track movement, and gather broad demographic information for aggregate use. IP addresses are not linked to personally identifiable information.


This web site contains links to other sites. Please be aware that Mondaq (or its affiliate sites) are not responsible for the privacy practices of such other sites. We encourage our users to be aware when they leave our site and to read the privacy statements of these third party sites. This privacy statement applies solely to information collected by this Web site.

Surveys & Contests

From time-to-time our site requests information from users via surveys or contests. Participation in these surveys or contests is completely voluntary and the user therefore has a choice whether or not to disclose any information requested. Information requested may include contact information (such as name and delivery address), and demographic information (such as postcode, age level). Contact information will be used to notify the winners and award prizes. Survey information will be used for purposes of monitoring or improving the functionality of the site.


If a user elects to use our referral service for informing a friend about our site, we ask them for the friend’s name and email address. Mondaq stores this information and may contact the friend to invite them to register with Mondaq, but they will not be contacted more than once. The friend may contact Mondaq to request the removal of this information from our database.


This website takes every reasonable precaution to protect our users’ information. When users submit sensitive information via the website, your information is protected using firewalls and other security technology. If you have any questions about the security at our website, you can send an email to webmaster@mondaq.com.

Correcting/Updating Personal Information

If a user’s personally identifiable information changes (such as postcode), or if a user no longer desires our service, we will endeavour to provide a way to correct, update or remove that user’s personal data provided to us. This can usually be done at the “Your Profile” page or by sending an email to EditorialAdvisor@mondaq.com.

Notification of Changes

If we decide to change our Terms & Conditions or Privacy Policy, we will post those changes on our site so our users are always aware of what information we collect, how we use it, and under what circumstances, if any, we disclose it. If at any point we decide to use personally identifiable information in a manner different from that stated at the time it was collected, we will notify users by way of an email. Users will have a choice as to whether or not we use their information in this different manner. We will use information in accordance with the privacy policy under which the information was collected.

How to contact Mondaq

You can contact us with comments or queries at enquiries@mondaq.com.

If for some reason you believe Mondaq Ltd. has not adhered to these principles, please notify us by e-mail at problems@mondaq.com and we will use commercially reasonable efforts to determine and correct the problem promptly.