I. Introduction
In May of 2018,
the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right
to Try Act of 2017 (“Right to Try Act”) was signed into law in order to
“authorize the use of unapproved medical products by patients diagnosed with a
terminal illness” as an “alternative pathway alongside, existing expanded
access policies of the Food and Drug Administration [(“FDA”)]. . . .”[1] While drafting the law, questions arose over
what types of diseases and conditions would be covered by the Right to Try Act
and whether those differed from existing laws allowing access to
investigational drugs.[2]
Given that nearly
45,000 Americans die of suicide each year,[3]
and that people with severe mental illnesses have a twenty-times higher risk of
suicide than the general population,[4]
such questioning by the drafters begs another: Does the Right to Try Act allow
patients with severe mental illnesses access to experimental drugs absent a
comorbid physical condition?
II. Background
Although
the Right to Try Act expressly states that eligible patients are those with
“life-threatening disease[s] or condition[s]” as described by 21 C.F.R. § 312.81,[5]
the Right to Try Act also specifically includes terminally ill patients as
eligible.[6] This ambiguity regarding the eligibility of a
disease led to discussions about the proper scope of the Right to Try Act. The consensus among lawmakers and the FDA was
that the Right to Try Act applied more broadly than past programs allowing
access to investigational drugs.[7] After one analyzes the various terminology
used by legislature and the FDA to define the scope of the Right to Try Act,
five reasons that a patient with a severe mental illness should be eligible
under Right to Try Act emerge.
III. Discussion
The
first of these reasons for concluding that a patient with severe mental illness
can be eligible is that legislatures acknowledged that “life-threatening
diseases” as defined by the Right to Try Act could include chronic conditions.[8] Furthermore, multiple definitions of “chronic
conditions” in the medical field include severe mental illnesses.[9] Thus, if the Right to Try Act includes chronic
conditions, it should include severe mental illnesses as “life-threatening
diseases.”
Second,
because studies have shown that patients diagnosed with
some severe mental illnesses commit alarming rates of suicide[10] and
several medical journals have noted that these patients
desire a treatment outcome of survival,[11] it
is clear that the characteristics of at least some
severe mental illnesses can easily fit within the Right to Try Act’s definition
of “life-threatening.”[12]
Third, the Right to Try Act expressly
describes eligible patients as those with “terminal illness[es].”[13] The Right to Try Act does not define or make
reference to another law defining “terminal illness,” however, earlier versions
of the Right to Try Act allowed state law to control how the term was defined.[14]
Of the forty-one states who have their own right to try legislation, two
definitions of “terminal illness” appeared most commonly among the varying
definitions.[15] The first, adopted nearly verbatim by twelve
states, describes the qualifying illness as:
[a] progressive disease or medical or
surgical condition that (i) entails significant functional impairment, (ii) is
not considered by a treating physician to be reversible even with
administration of available treatments approved by the United States Food and
Drug Administration; and (iii) will soon result in death without
life-sustaining procedures.[16]
If
the Right to Try Act adopted the definition of “terminally ill” commonly used
among these states, severe mental illnesses would likely fit the criteria of
that three-prong description because many severe mental illnesses are treatment
resistant,[17] entail
a “significant functional impairment,”[18]
and are associated with a high rate of suicide shortly after diagnosis.[19]
Fourth,
there is evidence demonstrating that the medical community increasingly recognizes
situations where severe mental illnesses can and should be considered terminal.
This view is based on doctors’ own
experience with mentally ill patients whose suicides seemed “inevitable” and
the notion that the amount of suffering is the same regardless of whether the patient’s
illness is physical or mental.[20]
Lastly,
the legislatures and regulators have said that the Right to Try Act is more
broad than preceding legislation.[21] Indeed, the Right to Try Act itself says that
it “expands the scope of individual liberty and agency among patients” and is
“consistent with . . . existing expanded
access policies of the [FDA].”[22] This broad language supports the conclusion
that the Right to Try Act encompasses the FDA’s Expanded Access program, which
covers patients with “serious diseases or conditions.”[23] Characteristics of severe mental illnesses
meet the description of “serious diseases or conditions” described in 21 C.F.R. § 312.300(b).[24] Further, the federal reporter’s discussion
that the term was meant to be flexible and consistent with an Institute of
Medicine report that specifically named psychotic illnesses as examples of
serious diseases or conditions.[25] Thus, because the Expanded Access program falls
within the scope of the Right to Try Act and some severe mental illnesses are
“serious diseases or conditions,” it follows that the Right to Try Act covers
some forms of severe mental illnesses, that constitute serious conditions.
Consequently,
whether viewed through the lens of a “life-threatening disease,” a “terminal
illness,” or a “serious disease or condition,” it is apparent that severe
mental illnesses make patients eligible to access experimental drugs under the
Right to Try Act of 2017.
-
Josh Baumgart
[1] Press
Release, White House, President Donald J. Trump Signs S. 204 into Law (May 30,
2018), https://www.whitehouse.gov/briefings-statements/president-donald-j-trump-signs-s-204-law/. See
generally Pub. L. No. 115-176, 132 Stat. 1372 (codified as amended at 21
U.S.C. § 360bbb-0a (2018)).
[2] See
Rose Beltz, What the Right to Try
Bill Means for Terminially Ill Patients, Krem2
(May 30, 2018), https://www.krem.com/article/news/health/what-the-right-to-try-bill-means-for-terminally-ill-patients/293-560084864;
Mark Terry, Right to Try Law and FDA Face
Criticism from Law’s Author, BioSpace
(June 1, 2018), https://www.biospace.com/article/right-to-try-law-and-fda-face-criticism-from-law-s-author/.
[3] Suicide
Statistics, Am. Found. for Suicide
Prevention, https://afsp.org/about-suicide/suicide-statistics/ (last
visited Feb. 3, 2019).
[4] See
Elizabeth Sinclair, Research Weekly:
Serious Mental Illness and Suicide, Treatment
Advocacy Ctr. (June 13, 2018), http://www.treatmentadvocacycenter.org/fixing-the-system/features-and-news/4011-research-weekly-serious-mental-illness-and-suicide
(finding individuals with schizophrenia are twenty-four-times more likely, individuals
with twenty-three-times more likely, and individuals with depression twelve-times
more likely to commit suicide).
[5] See 21 C.F.R. § 312.81(a) (2018). “For purposes of this section, the term ‘life-threatening’
means: (1) Diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted; and (2) Diseases or conditions with potentially fatal
outcomes, where the end point of clinical trial analysis is survival.” Id.
[6] 21 U.S.C. § 360bbb-0a.
[7] See
e.g., 164 Cong. Rec. H4355-01 (May 22, 2018) (statement of Rep. Pallone)
(“Now, supporters of this bill would have you believe that this legislation is
targeted at those with terminal illnesses, but that is simply not the case. S.204 would, in fact, apply to a much broader
range of patients diagnosed with life-threatening diseases or conditions.”).
[8] See
U.S. Dep’t. of Health and Human
Servs., Expanded Access to Investigational Drugs for Treatment Use – Questions
and Answers (Oct. 2017), https://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm351261.pdf;
see also Shannon Firth,
FDA Head Expresses Doubt About ‘Right To
Try’, MedPage Today (Oct. 4,
2018), https://www.medpagetoday.com/washington-watch/fdageneral/68310
(discussing removing “immediately” from “life threatening” in the Right to Try
Act opened up the eligibility to patients with “advanced diabetes”).
[9] E.g., World
Health Org., Preventing Chronic
Diseases 35 (2005),
http://www.who.int/chp/chronic_disease_report/full_report.pdf (“Mental and
neurological disorders are important chronic conditions . . . .”); Chronic Conditions, Ctrs. for Medicare & Medicaid Servs., https://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/Chronic-Conditions/CC_Main.html
(last updated May 10, 2017) (listing examples of nineteen chronic conditions);
Carmel M. Martin, Chronic Disease and Illness
Care, 53 Can. Fam. Physician 2086,
2086 (2007), https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2231531/#b4-0532086) (“Chronic
disease . . . includes . . . depression.”); Jean Yoon & Adam Chow, Comparing Chronic Condition Rates Using
ICD-9 and ICD-10 in VA Patients FY2014–2016, BMC Health Serv. Res. tbl.1 (2017), https://bmchealthservres.biomedcentral.com/articles/10.1186/s12913-017-2504-9
(listing depression, schizophrenia, PTSD, bipolar, and dementia as chronic
conditions in its analysis); U.S. Dep’t.
of Health and Human Servs., Guidance for Industry Information Program on
Clinical Trials for Serious or Life-Threatening Diseases and Conditions 5 (2002), https://www.fda.gov/downloads/regulatoryinformation/guidances/ucm126838.pdf
(“[M]any chronic illnesses that are generally well managed by available therapy
can have serious outcomes. For example .
. . depression, psychoses, and many other diseases can be serious. . . .”).
[10] Among the specific subtypes of
depression, studies illustrate that patients with major depressive disorder
have a lifetime suicide risk of 3.5%. G.W.
Blair-West et al., Down-Rating Lifetime Suicide
Risk in Major Depression, 95 Acta
Psychiatrica Scandinavica 259, 259 (1997), https://onlinelibrary.wiley.com/doi/epdf/10.1111/j.1600-0447.1997.tb09629.x. Additionally, between 10% and 19% of patients
with bipolar depression die of suicide. Meliha
Zengin Eroglu et al., Bipolar Disorder
and Suicide, 26 J. of Psychiatry and
Neurological Sci. 139, 140 (2013), https://www.researchgate.net/publication/260419495_Bipolar_disorder_and_suicide.
Patients diagnosed with schizophrenia
illustrated similar numbers, with studies reporting a lifetime suicide risk
between 4.9% and 15%. Kayhee Hor &
Mark Taylor, Suicide and Schizophrenia: A
Systematic Review of Rates and Risk Factors, 24 J. of Psychopharmacology 81, 84, https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2951591/
(estimating the rate to be approximately 5%).
[11] See, e.g., John March et
al., Fluoxetine, Cognitive-Behavioral
Therapy, and Their Combination for Adolescents with Depression, Jama Network (Aug. 18, 2004), https://jamanetwork.com/journals/jama/fullarticle/199274
(studying how various combination methods of treatment for MDD in adolescents
were aimed at preventing suicide and suicide attempts).
[12] See
21 C.F.R. § 312.81(a) (2018).
[13] Trickett Wendler, Frank Mongiello,
Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, Pub. L. No.
115-176, 132 Stat. 1372 (codified as amended at 21 U.S.C. § 360bbb-0a (2018)).
[14] H.R. 2368, 115th Cong. § 2 (2017)
(“The term ‘terminal illness’ has the meaning given to such term in the State
law . . . .”).
[15]
Roseann B. Termini, The Latest “Federal
Movement” in the Food and Drug Law Arena: The Federal Right-to-Try or Rather
Right-to-Know and Thus Request Investigational Therapies for Individuals with a
Life-Threatening Disease or Condition,
16 Ind. Health L. Rev. 1, 2
(2018) (forthcoming Nov. 2018); Right
to Try in Your State, Goldwater
Instit., (last vistited Feb. 20, 2019), http://righttotry.org/in-your-state/.
[16] Ala.
Code 1975 § 22-5D-2 (West 2019);
Fla. Stat. Ann. § 499.0295 (West 2019); Idaho Code Ann § 39-9403 (West 2019); Iowa Code
Ann. § 144E.2 (West 2019); Kentucky
S.B. 21, 2017 Sess., (Ky 2017) (enacted); Mont. Code Ann. § 50-12-102 (West 2019); N.C. Gen. Stat § 90-325.1 (West 2019); Nebraska 2017 NE. L.B. 117 (2017) (enacted);
S.C. Code Ann. § 44-137-10 (West 2019); Tenn. Code Ann. § 63-6-302 (West 2019).
[17] Some mental illnesses are categorized
as incurable. See Sally Burgess & Keith Hawton, Suicide, Euthanasia, and the Psychiatrist, 5 Philosophy, Psychiatry, & Psychol. 113,
122 (1998) (referencing severe depression and schizophrenia as “untreatable
mental illnesses”); Catherine S. Shaffer et al., A Conceptual Framework for Thinking About Physician-Assisted Death for
Persons with a Mental Disorder, 22
Psychol. Pub. Pol’y & L. 141, 143 (2016) (using Schizophrenia and
Bipolar Disorder as examples of “irremediable mental disorder[s].”); Candice T.
Player, Article, Death with Dignity and
Mental Disorder, 60 Ariz. L. Rev.
115, 148 (2018) (describing a study conducted by the National Institute of
Mental Health which found that some patients with major depressive disorder do not
respond to treatment).
[18] Professionals in the medical field
commonly classify mental illnesses and disorders as conditions that entail
significant functional impairments, specifically major depression. See,
e.g., Patrick E. McKnight & Todd B. Kashdan, 29 Clinical Psychol. Rev. 243, 244 (2009) (“Mental disorders .
. . produce functional impairment.”); Glorisa
Canino, Nat’l Academies of Sci., Engineering, & Medicine, The Role of Measuring Functional Impairment in
Assessing Serious Emotional Disturbance in Children 6, https://sites.nationalacademies.org/cs/groups/dbassesite/documents/webpage/dbasse_171793.pdf
(“Functional impairment can result from psychiatric . . . illness . . . .”);
Judit Balázs et al., Adolescent
Sub-Threshold Depression and Anxiety: Psychopathology, Functional Impairment,
and Increased Suicide Risk, 54 J. of
Child Psychol. & Psychiatry 670, 670 (2013) (“Subthreshold-depression. . . . ha[s] been associated
with significant impairment in adults.”).
[19] John Bostwick et al., Affective Disorders and Suicide Risk: A Reexamination,
157 Am. J. Psychiatry 1925, 1926
(2000). See generally Jason R. Randall et al., Acute Risk of Suicide and Suicide Attempts Associated with Recent
Diagnosis of Mental Disorders, 59 Can.
J. of Psychiatry 531, 535 (2014) (studying the rates of suicide within
the first year of diagnosis in patients with depression, schizophrenia,
anxiety, and dementia).
[20] See,
e.g., Constance E. George, When Is
Depression a Terminal Illness? Deliberative Suicide in Chronic Mental Illness,
18 AMA J. of Ethics 594, 597
(2016); see also Michael F. Myers, The Physician as Patient: A Clinical Handbook
for Mental Health Professionals 9 (2008) (“[S]ome psychiatric illnesses
are terminal and will not respond to any kind of treatment.”).
[21] See, e.g., 164
Cong. Rec. H4355-01 (May 22, 2018) (statement of Rep. Pallone) (“Now,
supporters of this bill would have you believe that this legislation is
targeted at those with terminal illnesses, but that is simply not the
case. S.204 would, in fact, apply to a
much broader range of patients diagnosed with life-threatening diseases or
conditions.”); Scott Gottlieb, Examining
Patient Access to Investigational Drugs, U.S.
Food & Drug Admin. (Oct. 3, 2017), https://www.fda.gov/NewsEvents/Testimony/ucm578634.htm.
[22] Trickett Wendler, Frank Mongiello,
Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, Pub. L. No.
115-176, 132 Stat. 1372 (codified as amended at 21 U.S.C. § 360bbb-0a (2018)).
[23] See
Expanded Access, U.S. Food & Drug
Admin.,
https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm
(last visited Feb. 3, 2019).
[24] See 21
C.F.R. § 312.300 (2018). Serious disease
or condition is defined as
a disease or condition associated with
morbidity that has substantial impact on day-to-day functioning. Short lived
and self-limiting morbidity will usually not be sufficient, but the morbidity
need not be irreversible, provided it is persistent or recurrent. Whether a
disease or condition is serious is a matter of clinical judgment, based on its
impact on such factors as survival, day-to-day functioning, or the likelihood
that the disease, if left untreated, will progress from a less severe condition
to a more serious one.
Id.; For a discussion of
these characteristics, see supra, notes
19–22.
[25] The FDA adopted this definition
because it intended the phrase “serious disease or illness” to be “flexible in
its interpretation” and felt that it was “conceptually consistent with the
criteria in the IOM report,” which listed examples of such diseases, including
“[c]onditions that may require frequent monitoring, such as schizophrenia and
other psychotic illness.” Expanded Access to Investigational Drugs for
Treatment Use, 74 Fed. Reg. 40900, 40909 (Aug. 13, 2009) (to be codified at 21
C.F.R. pt. 312) (emphasis added); Guidance for Industry on Fast Track Drug
Development Programs: Designation, Development, and Application Review;
Availability; Collection of Information, 63 Fed. Reg. 64093 (proposed Nov. 18,
1998) (to be codified at 21 U.S.C. § 356).
