Right-to-Try is legislation that allows terminally ill patients to access investigational treatments which have passed Phase I testing through the Food and Drug Administration (FDA), but are not available yet to the public because they have not yet been approved by the FDA. Int his webinar, Professor Bateman-House, Assistant Professor in the Division of Medical Ethics at New York University Langone Medical Center, discusses the legislation, breaks down the language, and explains how Right-to-Try can affect cancer patients.

1. Medical Ethics and Right-to-Try

2. TODAY’S WEBINAR
 SPEAKER(S)
 Allison Bateman-House, PhD, MPH, MA
 QUESTIONS
 Ask a question in the panel on the RIGHT SIDE of your
screen
 WEBINAR ARCHIVE
 FightCRC.org/webinar
 TWEET ALONG
 Follow along via Twitter – use the hashtag #CRCWebinar

3.  We are using LogMeIn GoToWebinar platform
 The side control panel can be adjusted using the
orange arrow
 Questions are asked by opening the “Questions”
tab – the arrow opens the box
 Not all questions are addressed during the
presentation depending on time and quantity, but if
necessary will be followed up individually
 If you are new to GoToWebinar and experience
streaming problems, shut down other high
bandwidth services such as Facebook, IM, or
hangout systems during presentation
 The “Audio” tab allows you to select either your
computer or phone to listen in
WEBINAR TECH

4. RESOURCE
S TABOO-TY PODCAST MINI MAGAZINES YOUR GUIDE IN THE FIGHT

5. FIGHTCOLORECTALCANCER
DISCLAIMER The information and services provided
by Fight Colorectal Cancer are for
general informational purposes only.
The information and services are not
intended to be substitutes for
professional medical advice,
diagnoses or treatment.
If you are ill, or suspect that you are
ill, see a doctor immediately. In an
emergency, call 911 or go to the
nearest emergency room.
Fight Colorectal Cancer never
recommends or endorses any specific
physicians, products or treatments for
any condition.

6. Dr.AlisonBateman-House
Alison Bateman-House, PHD, MPH, MA, is an Assistant Professor in
the Division of Medical Ethics at New York University Langone
Medical Center. She has advanced training in bioethics, public health,
and history, and she specializes in the ethics and history of human
subjects
research and the ethics and history of public health. Alison Bateman-
House serves as the non-voting, nonpaid deputy chairperson of the
Compassionate Use Advisory Committee (CompAC), an external,
expert panel of internationally recognized medical experts,
bioethicists, and patient representatives formed by NYU School of
Medicine, which advises the Janssen division of Johnson and Johnson
about requests for compassionate use of some of its investigational
medicines. She also co-chairs the NYU School of Medicine Working
Group on Compassionate Use and Pre-Approval Access.

7. Q
&
A
SNAP A #STRONGARMSELFIE
In 2018, up to $55,000 will be donated thanks to our
sponsors: Bayer, Fujifilm, Myriad Genetics and Taiho
Oncology!
Flex a “strong arm” & post it to Twitter or Instagram using the
hashtag #StrongArmSelfie

8. CONTACT US
CALL TOLL FREE
1.877.427.2111

9. Alison Bateman-House, PhD, MPH, MA
Assistant Professor, Division of Medical Ethics
Co-Chair, Working Group on Compassionate Use and Pre-
Approval Access (CUPA)
Alison.Bateman-House@nyumc.org @ABatemanHouse
Medical Ethics and
“Right to Try”

14. “patient-centricity”
+ “regulatory innovation”
+ transparency initiatives
+ fear of public pressure
more willingness to provide pre-approval
access

17. 17

18. Minimum # of Patients Granted Access in 2017 = 1632
Maximum # of Patients Granted Access in 2017 = ???

19. Best way to get access to investigational drugs
(and other treatments):
clinical trial

20. BUT…not all patients can participate in
clinical trials
* limited slots
* patient may not fit enrollment criteria
*geographic/financial/social barriers
* Some patients may not WANT to participate in a clinical trial
(I’m not talking about these patients; if you want to – let me
know)

21. For patients who can’t enter a clinical trial,
several ways to try “experimental” products
* self-experimentation
* use approved products in a new way or for a condition other
than what the drug was approved to treat (“off-label” use)
* personal importation from another country
* pre-approval access

22. What is “pre-approval access”? Access to a
medical product before it is approved by a
regulator AND outside of a clinical trial
* Also known as “compassionate use” (& myriad other terms,
especially ex-US)
* In the US, 2 legal pathways
“Expanded Access” – under FDA oversight (Code of Federal
Regulations, Title 21, Chapter 1, Subchapter D, Part 312)
“Right to Try” – 1 federal law (currently no use); 41 state laws

23. Expanded Access
* no comparable or satisfactory alternative therapy to diagnose,
monitor, or treat the patient's disease or condition
* 2 groups eligible for expanded access:
(1) those with life-threatening diseases/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
treatment” &
(2) those with serious diseases/conditions associated with
“morbidity that has a substantial impact on day-to-day
functioning”

24. use of an investigational medical product
to try to treat (aka, not for research, though data
may be collected – growing trend, which I
support)
Several different types of Expanded Access
* emergency versus non-emergency
* individual patient EA / group EA

25. • Large majority of requests that go to FDA are permitted
• ~ 11% of requests modified by FDA
• 1-4 day median response time
• FDA WAS NOT THE ROADBLOCK
Data Points re EA

26. • Identify willing physician / Identify promising drug
• Identify how to contact company
• Physician contacts company to request drug
• Physician contacts FDA to complete a form (w/ company’s help)
• FDA reviews form, allows proposal to proceed or not
• Physician seeks IRB approval at institution where drug will be used
• Report serious/unexpected adverse events to FDA (and, as required,
data to the company)
The Process

27. Right To Try
* Use of unapproved investigational drugs by individual patients
diagnosed with a terminal illness
??????????????????????????????????????????????????????????
“diagnosed with terminal illness” = diagnosed with a life-
threatening disease or condition

28. Patient must:
1) have exhausted approved treatment options,
2) provide written informed consent, &
3) be unable to participate in a clinical trial involving the drug, as
certified by a physician who…
a) is in good standing with the physician’s licensing
organization/board &
b) will not be compensated directly by the manufacturer for so
certifying

29. “Eligible investigational drug”:
1) a Phase 1 clinical trial has been completed,
2) has not been approved or licensed,
3) is the subject of an IND, &
4) ‘‘the active development or production of which is ongoing
and has not been discontinued by the manufacturer or placed
on clinical hold”

30. • Identify willing physician / Identify promising drug
• Identify how to contact company
• Physician contacts company to request drug
• Physician contacts FDA to complete a form (w/ company’s help)
• FDA reviews form, allows proposal to proceed or not
• Physician seeks IRB approval at institution where drug will be used ??
• Report serious/unexpected adverse events to FDA (annually except
where deemed public health issue) and, as required, data to the
company)
The Process (federal law has not yet been translated into
regulations; 41 different state laws)

31. • Goldwater Institution targeted state legislators: currently law in 41
states
• Federal RTT bill passed by Senate (in order to permit necessary FDA
User Fee vote);
• Alternate version passed by House;
• House version went to the Senate but was not voted on;
• Subjected to Koch-funded advertising campaign + executive
pressure, House passed version originally passed by Senate
Right to Try Saga

33. Access via EA v. Access via RTTEA
RTT

35. • FDA not obstacle to access
• Companies unlikely to provide product without FDA sign-off
• FDA review is useful & pro-patient (improves treatment plan)
• IRB review may be useful & pro-patient (improves informed consent?)
• EA has meaningful informed consent provision
• RTT opens door to bad actors
• Multiple state RTT laws have bad provisions
• EA designed for maximum flexibility
• EA designed to inform FDA of SAEs rapidly
• RTT liability waiver too wide
Why is EA better than RTT?

37. Once the federal RTT law passed…
* ALS/ stem cell frenzy
* Ability to profit?
* Goldwater/Koch disappear
* Increased EA requests?
* Bad actors

38. Should Patients Have the Right to Try?
Yes
(They did, before the RTT law was passed, via
Expanded Access. )
RTT movement has led to improvements;
RTT law has not.
More complex issue than a single law can fix!

39. Thank You
Check out the FAQs & resources on CUPA’s webpage –
just search for “NYU Compassionate Use” or contact us
to sign up for our monthly email update
Alison.Bateman-House@nyumc.org
@ABatemanHouse

40. So, is Expanded Access perfect? NO!
common concerns; some real, some not…
• Lack of knowledge = unequal access
• Concern about paperwork burden
• Fear of litigation
• Fear SAE(s) will cause problems for development/approval
• Ancillary costs not covered by insurance
• IRB review might cause problems (time, money)
• Within FDA, divisions vary on receptivity to expanded access
• Fear that expanded access will hurt trials
• Fear that patients are unable to make free & informed decision to
try an investigational product

41. • Lack of knowledge = unequal access
• FDA charged the Reagan Udall Foundation of the FDA to
create an “Expanded Access Navigator” website
• ClinicalTrials.gov revised to make it possibly to more easily
search for expanded access opportunities
• 21st Century Cures bill (passed Dec 2016) mandates
companies to provide their expanded access policy to the
public when any of the products move to Phase II testing
• FDA released updated guidance documents
• FDA revised their webpage dealing with expanded access

42. • Concern about paperwork burden
• FDA introduced streamlined form for single patient requests
• Released updated guidance documents
• Has dedicated staff you can call for help
• Maybe the problem wasn’t the FDA paperwork after all????

43. • Fear of litigation
• 2 reviews found no evidence of expanded access-related
lawsuits
• RTT has (overly broad) liability shield
• If true concern, Congress may be convinced to pass limited
liability shield for EA?

44. • Fear SAE(s) will cause problems for development/approval
• FDA audit reviews found no evidence of this a problem
• RTT tried to address by preventing SAEs from being reported
to FDA
• Can FDA convince industry?

46. • Ancillary costs not covered by insurance
• Varies by insurer, but generally true – insurance does not pay
for investigational therapies or ancillary costs incurred outside
of clinical trials (including doctors’ time)
• Makes some doctors/institutions unwilling to participate in
expanded access
• Covering these costs may help with the unequal access issue
• Growing problem re “flipping the switch” from clinical trial to
expanded access

47. • IRB review might cause problems (time, money)
• October 2017, FDA updated guideline for FDA review of
expanded access – intended to make it faster (allows for
single, designated reviewer rather than full board review)
• Clinical Research Pathways
(Though maybe the IRB isn’t really a problem??)

48. • Within FDA, divisions vary on receptivity to expanded access
• Problem that FDA administration must fix
• FDA can only approve what it gets, so if there is less
expanded access in Neurology than in other divisions, it may
be that neurologists submit less requests than, say, their peers
in oncology and/or that drug companies in the neurological
space are declining to provide product more frequently than
companies in the oncological space….

49. • Fear that expanded access will hurt trials
• FDA restricts expanded access to those who have “no
comparable or satisfactory alternative therapy to diagnose,
monitor, or treat the patient's disease or condition”
• Companies must restrict expanded access to those who are
ineligible for their trials (physically, geographically, socially).
Mere unwillingness to participate in a trial should not suffice.
• That said, companies should try to make the trials as appealing
as possible (with regard to study design, procedures, etc.)

50. • Fear that patients are unable to make a truly free & informed
decision to try an investigational product
• If true, no (ethical) clinical trials!
• Capacity for free and informed decision-making must be
individually assessed, but there is no ethical reason to assume
that patients who have no other options are incapable of
making treatment or research decisions.

51. Equally important - must work with companies to
identify incentives/disincentives for them to make
their products available re expanded access
Example: “real world data”