Do any clinical guidelines support the use of ketamine or esketamine for treatment-resistant depression (TRD)?

Comment by InpharmD Researcher

Few clinical guidelines address the use of ketamine or esketamine for treatment-resistant depression (TRD); the role of these agents is not yet fully elucidated. Clinical guidelines from the US Department of Veterans Affairs and Department of Defence note the lack of supportive data for ketamine administration outside of adjunct or third-line treatment in patients with depression, providing only a weak-recommendation for their use. However, numerous efficacy-related studies are available for ketamine and esketamine, generally demonstrating efficacy in treatment of depression.

Background

Guidelines for major depressive disorder (MDD) published by the United States Veterans Affairs (VA) and Department of Defense (DOD) very briefly mention ketamine/esketamine for treatment resistant depression (TRD). The guidelines make one weak recommendation suggesting ketamine or esketamine as an option for augmentation in patients with MDD who have not responded to several adequate pharmacologic trials. [1]

Additionally, Canadian Network for Mood and Anxiety Treatments (CANMAT) Task Force guidelines published in 2021, which included a systematic review, examined the efficacy and safety of racemic ketamine in adults diagnosed with TRD. Intravenous (IV) racemic ketamine administered as a single infusion demonstrated Level 1 evidence for efficacy in adults with TRD, with a rapid onset of antidepressant effects noted. However, the evidence supporting multiple infusions, which could be administered as an acute series or for maintenance treatment, was limited to Level 3, while non-IV formulations of racemic ketamine only exhibited Level 3 or 4 evidence. Adverse events primarily included transient dissociative symptoms and physiological effects like hypertension. The CANMAT task force recommended single-dose IV racemic ketamine as a third-line intervention for adults with TRD, emphasizing careful assessment of the need for repeated and maintenance infusions. The task force highlighted the limited evidence concerning efficacy and potential risks associated with misuse and diversion, advising that oral and other non-IV formulations be restricted to specialists with the requisite expertise and connections to tertiary or specialized centers. [2]

Several review articles have discussed esketamine use for treatment resistant depression along with therapeutic drug monitoring recommendations. Esketamine is shown in clinical trials to be effective for the short-term treatment of depression and reduction in suicidality rate as monotherapy and in combination with other antidepressants compared to placebo. Intranasal esketamine’s advantage over other antidepressants was identified as its ability to elicit an almost immediate clinical response in hours or days compared to weeks for other similarly indicated medications. Although both serious and non-serious adverse events have been reported with short-term esketamine use, most adverse events in clinical trials have been categorized as mild to moderate and transient. Overall, the authors suggest new generation antidepressants, including esketamine, are as effective as classical agents based on results from clinical studies. Providers are encouraged to personalize therapy and weigh the risk/benefit of antidepressant medication choices by monitoring for adverse effects and patient response to treatment. [3], [4]

In a 2021 systematic review and meta-analysis (N= 24 trials, 1877 participants), the efficacy and tolerability of racemic ketamine and esketamine were compared for unipolar and bipolar major depression. Overall, ketamine demonstrated a significant improvement in response (RR 2.0382, 95% CI 1.5748 to 2.6380) and remission rates (RR 2.0029, 95% CI 1.5005 to 2.6735) compared to control. A subgroup analysis was performed comparing ketamine and esketamine. When compared to intranasal esketamine, intravenous ketamine was again observed to demonstrate more significant overall response (RR 3.01 vs.1.38; p= 0.0023) and remission rates (RR 3.70 vs. 1.47; p= 0.0012). Additionally, drop-outs due to adverse events were significantly lower with ketamine (RR 0.76 vs. 1.37, p= 0.0331). [5]

A 2019 review of reviews, meta-analyses, and randomized controlled trials (not included in the other reviewed literature) found that low-dose intravenous (IV) ketamine provides a rapid antidepressant effect at 24 hours. Validated depression rating scales showed that administration of ketamine changed scores in as little as 40 mins with effects lasting at least 1-2 weeks. Still, there is limited and inconsistent data for ketamine having efficacy in depressive patients after 2 weeks. Remission symptoms have been observed at 80 mins, 1 day, and up to 2 weeks. Two studies showed a reduction of suicidal thoughts after 1-2 days after a single dose of ketamine, but this effect was not seen after 3 weeks in a third study. Studied doses of ketamine included 0.1 mg/kg, 0.2 mg/kg, 0.5 mg/kg, 1 mg/kg, and 2 mg/kg. Most infusion times reported were over 40 minutes, with one study delivering six 0.5 mg/kg infusions over 45 mins thrice weekly for three weeks (no efficacy was observed in this study). [6], [7], [8], [9]

Ketamine as an antidepressant was shown to be well tolerated and adverse events usually occurred with very high doses administered over an extended period of time. Some of the most common adverse events were dizziness, dissociation, nausea, headaches, vertigo, and dysgeusia which usually resolve spontaneously. No long-term psychotomimetic side effects have been reported in any of the studies, but one isolated case of suicide attempt was reported in one study. The dose, mode of administration, and long-term effects of ketamine need to be further explored to optimize its antidepressant effects. [6], [7], [8], [9]

An international group of mood disorder experts provided a synthesis of the literature regarding the safety, efficacy, and tolerability of ketamine and esketamine in adults with treatment-resistant depression. It was recommended to start the administration of IV ketamine at a dose of 0.5 mg/kg with an infusion time of 40 minutes. While the efficacy at lower doses (0.1-0.2 mg/kg) may be inferior, doses may be lowered in cases of intolerability. The upper dosing limit for ketamine infusions in this patient population is not established, but studies show no evidence of superiority of 1 mg/kg over 0.5 mg/kg. The panel recommended the utilization of ideal body weight in patients who are overweight or obese, as this patient population may show a higher response rate to ketamine. A follow-up assessment should be conducted after completing four to six IV infusions to determine the intervention's overall efficacy. If the individual presented with a minimal response (i.e., ≤20% improvement from baseline in total depression symptom severity) after four to six infusions, that individual would be considered non-responsive and further infusions would not be warranted. There is insufficient data regarding the dosing and frequency of the administration of IV ketamine as a maintenance treatment, but studies have also shown no difference in efficacy between twice-weekly and thrice-weekly 0.5 mg/kg infusions over 40 minutes. It was suggested that practitioners perform periodic evaluations of the need for ongoing treatment on a monthly to bi-monthly basis. [9]

A 2020 meta-analysis evaluated the effects of IV ketamine infusion in patients with treatment-resistant depression using data from 19 studies (7 randomized controlled trials and 12 open-label trials; N= 818). All included studies utilized a single dose of IV ketamine infusion of 0.5 mg/kg, with outcomes measured up to 7 days post-infusion. The overall effects of ketamine were noted as early as 4 hours, peaked at 24 hours, but gradually diminished from days 2 to 7. Subgroup analysis of depression scores in patients receiving ketamine compared to placebo demonstrated a standard mean difference (SMD) of 0.77 (95% CI 0.46 to 1.08) at 24 hours and 0.49 (95% 0.20 to 0.78) at 7 days. Overall, IV ketamine was associated with a significantly improved SMD in depression scores (0.68; 95% 0.46 to 0.90). Similarly, better clinical response and clinical remission were also observed in ketamine recipients. Though a quantitative analysis was not performed for multiple versus single ketamine infusions, results demonstrated that multiple infusions (0.5 mg/kg, 3 times/week over 2 weeks) led to enhanced effectiveness and delayed clinical relapse. This was also observed when ketamine was used in a maintenance schedule with patients who responded to the initial treatment. However, long-term data are still required to investigate the safety and efficacy of ketamine in patients presenting with resistant symptoms. [10]

References:

[1] VA/DoD Clinical Practice Guideline. (2022). The Management of Major Depressive Disorder. Washington, DC: U.S. Government Printing Office.
[2] Swainson J, McGirr A, Blier P, et al. The Canadian Network for Mood and Anxiety Treatments (CANMAT) Task Force Recommendations for the Use of Racemic Ketamine in Adults with Major Depressive Disorder: Recommandations Du Groupe De Travail Du Réseau Canadien Pour Les Traitements De L'humeur Et De L'anxiété (Canmat) Concernant L'utilisation De La Kétamine Racémique Chez Les Adultes Souffrant De Trouble Dépressif Majeur [published correction appears in Can J Psychiatry. 2021 Dec;66(12):1102. doi: 10.1177/07067437211035276.]. Can J Psychiatry. 2021;66(2):113-125. doi:10.1177/0706743720970860
[3] Swainson J, Thomas RK, Archer S, et al. Esketamine for treatment resistant depression. Expert Rev Neurother. 2019;19(10):899-911. doi:10.1080/14737175.2019.1640604
[4] Protti M, Mandrioli R, Marasca C, Cavalli A, Serretti A, Mercolini L. New-generation, non-SSRI antidepressants: Drug-drug interactions and therapeutic drug monitoring. Part 2: NaSSAs, NRIs, SNDRIs, MASSAs, NDRIs, and others. Med Res Rev. 2020;40(5):1794-1832. doi:10.1002/med.21671
[5] Bahji A, Vazquez GH, Zarate CA Jr. Comparative efficacy of racemic ketamine and esketamine for depression: A systematic review and meta-analysis [published correction appears in J Affect Disord. 2020 Nov 20;:]. J Affect Disord. 2021;278:542-555. doi:10.1016/j.jad.2020.09.071
[6] Corriger A, Pickering G. Ketamine and depression: a narrative review. Drug Des Devel Ther. 2019;13:3051‐3067.
[7] Serafini G, Howland R, Rovedi F, Girardi P, Amore M. The Role of Ketamine in Treatment-Resistant Depression: A Systematic Review. Curr Neuropharmacol. 2014;12(5):444-461.
[8] Fond G, Loundou A, Rabu C, et al. Ketamine administration in depressive disorders: a systematic review and meta-analysis. Psychopharmacology (Berl). 2014;231(18):3663-76.
[9] McIntyre RS, Rosenblat JD, Nemeroff CB, et al. Synthesizing the Evidence for Ketamine and Esketamine in Treatment-Resistant Depression: An International Expert Opinion on the Available Evidence and Implementation. Am J Psychiatry. 2021;178(5):383-399. doi:10.1176/appi.ajp.2020.20081251
[10] Marcantoni WS, Akoumba BS, Wassef M, et al. A systematic review and meta-analysis of the efficacy of intravenous ketamine infusion for treatment resistant depression: January 2009 - January 2019. J Affect Disord. 2020;277:831-841. doi:10.1016/j.jad.2020.09.007

Literature Review

A search of the published medical literature revealed 7 studies investigating the researchable question:

Do any clinical guidelines support the use of ketamine or esketamine for treatment-resistant depression (TRD)?

Level of evidence

B - One high-quality study or multiple studies with limitations  Read more→



Please see Tables 1-7 for your response.


 

Efficacy and Safety of Flexibly Dosed Esketamine Nasal Spray Combined With a Newly Initiated Oral Antidepressant in Treatment-Resistant Depression: A Randomized Double-Blind Active-Controlled Study

Design

Randomized, double-blind, active-controlled, multicenter, phase 3 study

N= 223

Objective

To compare the efficacy and safety of switching patients with treatment-resistant depression from an ineffective antidepressant to flexibly dosed esketamine nasal spray plus a newly initiated antidepressant or to a newly initiated antidepressant (active comparator) plus placebo nasal spray

Study Groups

Esketamine (n= 114)

Placebo (n= 109)

Inclusion Criteria

Age 18 to 64 years, single-episode (> 2 years) or recurrent major depressive disorder without psychotic features, Mini International Neuropsychiatric Interview, score > 34 on the Inventory of Depressive Symptomatology–Clinician Rating (IDS-C), moderate to severe depression, treatment-resistant depression, medically stable, able to self-administer

Exclusion Criteria

Recent suicidal behavior or ideation, psychotic disorder, bipolar or related disorders, antisocial histrionic or narcissistic personality disorder, obsessive-compulsive disorder, intellectual disability, autism, uncontrolled hypertension, seizures, history of moderate or severe substance use disorder, positive urine test results for drug of abuse (e.g. cannabinoids, methadone, etc.)

Methods

Patients were randomized (1:1) to receive esketamine 56 or 84 mg nasal spray or placebo. Both administered twice weekly combined with newly-initiated oral antidepressants selected by investigators.

Duration

28 days

Outcome Measures

Primary: change in Montgomery Asberg Depressing Rating Scale (MARDS) score from baseline to day 28

Secondary: > 50% decrease in MARDS score by day, early response defined as > 50% decrease n MARDS score by day 2, safety

Baseline Characteristics

 

Esketamine (n= 114)

Placebo (n= 109)

 

Age, years

44.9 46.4  

Female

65.8% 57.8%  

Race

Asian

Black/African American

White

Multiple

 

0.9%

5.3%

93.0%

0.9%

 

0.9%

4.6%

93.6%

0.9%

 

Concomitant antidepressant

Duloxetine

Escitalopram

Sertraline

Venlafaxine extended-release

 

52.6%

18.4%

14.0%

14.9%

 

56.0%

15.6%

14.7%

13.8%

 

Results

Endpoint

Esketamine (n= 114)

Placebo (n= 109)

Difference between groups (95% confidence interval [CI]; p-Value) or p-Value

Change in MARDS score from baseline to day 28

-21.4

-17.0

-4.0 (-7.31 to -0.64; p= 0.02)

> 50% decrease in MARDS score by day

7.9%

4.6%

0.321

Early response defined as > 50% decrease n MARDS score by day 2

18/109 (16.5%)

11/102 (10.8%)

--

Adverse Events

Treatment-emergent adverse events (TEAEs) in the esketamine group were dizziness, dissociation, dysgeusia, verigo, and nausea (incidence ranging from 20.9% to 26.1%). Incidence was 2- to 10-times lower in the placebo arm versus the comparator.

Study Author Conclusions

Current treatment options for treatment-resistant depression have considerable limitations in terms of efficacy and patient acceptability. Esketamine is expected to address an unmet medical need in this population through its novel mechanism of action and rapid onset of antidepressant efficacy. The study supports the efficacy and safety of esketamine nasal spray as a rapidly acting antidepressant for patients with treatment-resistant depression.

InpharmD Researcher Critique

Patients with significant concomitant psychiatric illness were excluded. While generally observed as safe when the primary condition was treatment-resistant depression, the effectiveness and safety in these subpopulations remain uncertain as patients are likely to exhibit multiple psychosocial conditions.



References:

Popova V, Daly EJ, Trivedi M, et al. Efficacy and Safety of Flexibly Dosed Esketamine Nasal Spray Combined With a Newly Initiated Oral Antidepressant in Treatment-Resistant Depression: A Randomized Double-Blind Active-Controlled Study [published correction appears in Am J Psychiatry. 2019 Aug 1;176(8):669]. Am J Psychiatry. 2019;176(6):428-438. doi:10.1176/appi.ajp.2019.19020172

 

Efficacy of Esketamine Nasal Spray Plus Oral Antidepressant Treatment for Relapse Prevention in Patients With Treatment-Resistant Depression

Design

Phase 3, multicenter, double-blind, randomized, withdrawal study

N= 297

Objective

To assess the efficacy of esketamine nasal spray plus an oral antidepressant compared with an oral antidepressant plus placebo nasal spray in delaying relapse of depressive symptoms in patients with treatment-resistant depression (TRD) in stable remission after an induction and optimization course of esketamine nasal spray plus an oral antidepressant.

Study Groups

Stable remission 

Esketamine (n= 90)

Placebo (n= 86)

Stable response:

Esketamine (n= 62)

Placebo (n= 59)

Inclusion Criteria

Age 18 to 64, recurrent or single-episode MDD, > 34 score on Clinician-rated Inventory of Depressive Symptomatology, total MARDS score > 28, non-responders to at least 1, but no more than 5, antidepressants in current depressive episodes

Exclusion Criteria

History of psychotic disorder, suicidal behavior, current or recent homicidal or suicidal ideation or intent, diagnosis of MDD with psychotic features, moderate or severe substance or alcohol use disorder within 6 months

Methods

Patients were randomized to receive esketamine 56 or 84 mg nasal spray or placebo. After 16 weeks of treatment, the remaining patients (n=297) were randomized into the withdrawal phase to either continue esketamine nasal spray or discontinue treatment by being switched to a placebo spray along with oral antidepressants. Patients who achieved either stable remission (MADRS score ≤12 for ≥3 of the last 4 weeks, with 1 excursion [MADRS score >12] or 1 missing MADRS assessment permitted at week 13 or 14 only) or stable response (defined as ≥50% reduction in MADRS score from baseline in the last 2 weeks of the optimization phase but without achieving remission) were measured separately for analysis.

Duration

 

Outcome Measures

Relapse in patients who received stable remission and response group, safety
    Stable remission Stable response

Baseline Characteristics

 

Esketamine (n= 90)

Placebo (n= 86)

Esketamine (n= 62)  Placebo (n= 59)

Age, years

45.4 46.2 47.2 46.7

Female

64.4% 68.6% 61.3% 71.2%

Race

Black

White

Other

Multiple

Not reported

 

4.4%

88.9%

2.2%

1.1%

3.3%

 

7.0%

88.4%

1.2%

0

2.3%

 

3.2%

91.9%

4.8%

0

%

 

1.7%

1.7%

93.2%

1.7%

1.7%

Suicidal ideation in the past 6 months

20.0%

16.3%

32.3%

23.7%

Results

Endpoint

Stable remission

 

Stable response

 

Esketamine (n= 90)

Placebo (n= 86)

Esketamine (n= 62)

Placebo (n= 59)

Relapse

24 (26.7%)

39 (45.3%)

16 (25.8%)

34 (57.6%)

Hazard ratio comparing stable remission and response groups (95% confidence interval [CI])

p-Value

0.49 (0.29 to 0.84)

0.003

0.30 (0.16 to 0.55)

< 0.001

 

Adverse Events

Common adverse events for esketamine-treated patients included transient dysgeusia, vertigo, dissociation, somnolence, and dizziness (incidence, 20.4% to 27.0%), each reported in fewer patients (<7%) treated with an antidepressant and placebo.

Study Author Conclusions

Continued treatment with esketamine nasal spray plus an antidepressant can sustain antidepressant effects among patients with treatment-resistant depression to a greater extent than an oral antidepressant alone.

InpharmD Researcher Critique

Patients with more severe psychotic behaviors were excluded, limiting the generalizability of the study.



References:

Daly EJ, Trivedi MH, Janik A, et al. Efficacy of Esketamine Nasal Spray Plus Oral Antidepressant Treatment for Relapse Prevention in Patients With Treatment-Resistant Depression: A Randomized Clinical Trial. JAMA Psychiatry. 2019;76(9):893-903. doi:10.1001/jamapsychiatry.2019.1189

 

Efficacy and Safety of Fixed-Dose Esketamine Nasal Spray Combined With a New Oral Antidepressant in Treatment-Resistant Depression: Results of a Randomized, Double-Blind, Active-Controlled Study (TRANSFORM-1)

Design

Phase 3, double-blind, multicenter study

N= 346

Objective

To compare the efficacy and safety of fixed doses of esketamine nasal spray plus a newly initiated oral antidepressant to a newly initiated oral antidepressant (active comparator) plus a placebo nasal spray in adult patients with treatment-resistant depression (TRD)

Study Groups

Esketamine 56 mg (n= 117)

Esketamine 84 mg (n= 116)

Placebo (n= 113)

Inclusion Criteria

Age 18 to 64 years with recurrent major depressive disorder (MDD) or single-episode MDD without psychotic features confirmed by Mini-International Neuropsychiatric Interview, moderate-to-severe depression unresponsive to treatment confirmed at a screening phase

Exclusion Criteria

Suicidal ideation with intent to act within the prior 6 months or suicidal behavior within the prior year; diagnosis of psychotic disorder, bipolar, or related disorders; moderate or severe substance use disorder within the prior 6 months; positive test result for drugs of abuse

Methods

Patients were randomized (1:1:1) based on a computer-generated randomization schedule to double-blind nasal spray treatment with either 1 or 2 fixed doses of esketamine, 56 or 84 mg, or matching placebo administered twice-weekly intranasally for 4 weeks under direct supervision at the study site. An open-label antidepressant was added for all patients from four options: duloxetine, escitalopram, sertraline, or venlafaxine extended-release (ER) and must be one that the patient had not previously received. Oral doses were administered daily for 4 weeks and titrated as mandated by a predefined protocol. Patients could switch to another oral antidepressant.

Montgomery-Asberg Depression Rating Scale (MADRS) was measured via telephone by independent investigators blinded to the study protocol and patient clinical status. MADRS were performed two days before nasal spray dosing.

Duration

4 weeks

Outcome Measures

Primary: change in MADRS from day 1 to day 28

Baseline Characterisitics

 

Esketamine 56 mg (n= 117)

Esketamine 84 mg (n= 116)

Placebo (n= 113)

Age, years

46.4 45.7 46.8

Female

70.4% 69.3% 71.7%

Race

Asian

Black/African American

White

Other

Multiple

Not reported

 

1.7%

6.1%

79.1%

7.0%

0

6.1%

 

0.9%

6.1%

74.6%

10.5%

0

7.9%

 

1.8%

4.4%

76.1%

8.8%

0.9%

8.0%

Duration of current antidepressant episode, weeks

202.8

212.7

193.1

Oral antidepressant

Duloxetine

Escitalopram

Sertraline

Venlafaxine ER

 

42.6%

22.6%

20.9%

13.9%

 

37.7%

20.2%

21.1%

21.1%

 

38.9%

21.2%

22.1%

17.7%

Results

Endpoint

Esketamine 56 mg (n= 117)

Esketamine 84 mg (n= 116)

Placebo (n= 113)

Change in MADRS from day 1 to day 28

-19.0 ± 13.86

-18.8 ± 14.12

-14.8 ± 15.07

Least-square difference vs placebo (95% confidence interval [CI])

p-Value

-4.1 (-7.67 to 0.49)

0.027

-3.2 (-6.88 to 0.45)

0.088

--

Adverse Events

The most common (>20%) adverse events reported for esketamine plus oral antidepressant were nausea, dissociation, dizziness, vertigo, and headache

Study Author Conclusions

Statistical significance was not achieved for the primary endpoint; nevertheless, the treatment effect (Montgomery-Asberg Depression Rating Scale) for both esketamine/antidepressant groups exceeded what has been considered clinically meaningful for approved antidepressants vs placebo. Safety was similar between esketamine/antidepressant groups and no new dose-related safety concerns were identified.

InpharmD Researcher Critique

Patients with significant psychiatric conditions were excluded, limiting the generalizability of the study results. Non-significance was found for the higher esketamine 84 mg dose which questions the acceptability of the statistical significance of the lower 56 mg dose.



References:

Fedgchin M, Trivedi M, Daly EJ, Melkote R, Lane R, Lim P, Vitagliano D, Blier P, Fava M, Liebowitz M, Ravindran A, Gaillard R, Ameele HVD, Preskorn S, Manji H, Hough D, Drevets WC, Singh JB. Efficacy and Safety of Fixed-Dose Esketamine Nasal Spray Combined With a New Oral Antidepressant in Treatment-Resistant Depression: Results of a Randomized, Double-Blind, Active-Controlled Study (TRANSFORM-1). Int J Neuropsychopharmacol. 2019 Oct 1;22(10):616-630. doi: 10.1093/ijnp/pyz039.

 

Cost-Effectiveness of Esketamine Nasal Spray for Patients With Treatment-Resistant Depression in the United States

Design

Cost analysis 

N= N/A

Objective

To estimate the cost-effectiveness of esketamine, a novel intranasally-dosed antidepressant, for patients in the United States with treatment-resistant depression

Study Groups

Usual care

Esketamine

Inclusion Criteria

Adults with the major depressive disorder who have failed two prior antidepressants: 1) Esketamine: initial treatment with esketamine nasal spray plus oral antidepressants, followed by additional antidepressants and/or psychotherapy if depressive symptoms do not respond; 2) Usual care: initial treatment with oral antidepressants, followed by additional antidepressants and/or psychotherapy if depressive symptoms do not respond.

Exclusion Criteria

N/A

Methods

A decision-analytic model, parameterized with efficacy data from phase III randomized trials of esketamine (TRANSFORM-1, TRANSFORM-2, TRANSFORM-3, SUSTAIN-1 trial), was used to simulate the effects of treatment with esketamine versus oral antidepressants over a 5-year horizon in 2015 United States dollars (USD). The incremental cost-effectiveness ratio (ICER) of esketamine was calculated as the ratio of its incremental cost to its incremental quality-adjusted life-years (QALYs) relative to usual care. A lower ICER indicates a better health-economic value.

Based on results for distinct cost-effectiveness thresholds of $50,000/QALY, $100,000/QALY, and $150,000/QALY, the value-based price of esketamine, defined as the per-dose price at which its ICER is equal to the threshold (highest price at which esketamine would be deemed cost-effective), was calculated for each threshold. 

Key model parameters included the efficacy of esketamine versus oral antidepressants (relative risk 1.39 for remission, 1.32 for response) and the monthly cost of esketamine ($5,572 for month 1, $1,699–2,244 thereafter) based on pooled efficacy data (esketamine n= 421; placebo n= 289).

Duration

Between March 2019 and December 2019

Outcome Measures

Remission and response of depression, QALYs, costs, ICERs, and value-based prices

Baseline Characteristics

See Methods regarding model description and model input data

Results

Endpoint

Usual care

Esketamine

Difference

Fraction of time spent in health state, %

Initiation/non-response/relapse

Response

Remission

 

72.3%

2.4%

25.3%

 

66.9%

2.0%

31.1%

 

-5.4%

-0.4%

5.7%

QALYs

3.00

3.07 0.07

QALY costs, 2015 USD

Total cost, societal perspective

Total cost, healthcare sector perspective

Cost components

Esketamine treatment costs

Medication cost

Physician and medical assistant services

Patient time

Other healthcare costs

Lost productivity costs

 

121,073

79,786

 

 

-

-

-

79,786

41,287

 

137,690

96,781

 

 

16,352

2,062

2,074

78,367

38,836

 

16,617

16,995

 

 

16,352

2,062

2,074

−1,419

−2,451

Incremental cost-effectiveness ratios, $/QALY

Societal perspective

Healthcare sector perspective

 

-

-

 

-

-

 

237,111

242,496

In probabilistic sensitivity analysis in which the model was run using parameter values drawn at random from distributions reflecting their uncertainty, there was a > 95% likelihood that esketamine’s ICER would be above $150,000/QALY. At a cost-effectiveness threshold of $150,000/QALY, esketamine’s value-based price was approximately $140/dose (versus a current price of $240/dose).

Adverse Events

N/A

Study Author Conclusions

In this decision-analytic modeling analysis, we found that esketamine is unlikely to be cost-effective for management of treatment-resistant depression at its current price of $240 per dose. Esketamine could become cost-effective by US standards if its price were to fall to $140 or less per dose. Achieving these price reductions while ensuring continued access for the patients who stand to benefit from esketamine will require careful and concerted efforts from payers and policymakers.

InpharmD Researcher Critique

Data used to stimulate esketamine are only based on a few hundred clinical trial participants and limited long-term results. The 5-year time horizon may fail to capture relevant longer-term health-economic consequences. If data were considered inadequate pertaining to the patient, relevant data were derived from the overall population with major depressive disorder which may have underestimated the degree of occupational impairment and mortality in selected treatment-resistant individuals. Due to the absence of comparative outcomes data, the study was unable to directly assess esketamine’s cost-effectiveness versus other alternatives (e.g., electroconvulsive therapy, transcranial magnetic stimulation)



References:

Ross EL, Soeteman DI. Cost-Effectiveness of Esketamine Nasal Spray for Patients With Treatment-Resistant Depression in the United States. Psychiatr Serv. 2020;71(10):988-997. doi:10.1176/appi.ps.201900625

 

A Double-Blind, Randomized, Placebo-Controlled, Dose-Frequency Study of Intravenous Ketamine in Patients With Treatment-Resistant Depression

Design

Double-blind, randomized, placebo-controlled, parallel-group multicenter, open-label, phase II trial

N= 67

Objective

To evaluate the efficacy of two dosing regimens of ketamine at 0.5 mg/kg administered intravenously over 40 minutes at sustaining the antidepressant effects of ketamine in patients with treatment-resistant depression beyond the initial dose

Study Groups

Placebo two times/week (n= 16)

Ketamine two times/week (n= 18)

Placebo three times/week (n= 16)

Ketamine three times/week (n= 17)

Inclusion criteria

adults who met Diagnostic and Statistical Manual (DSM) IV criteria for recurrent major depressive disorder without psychotic features; inadequate response to at least two antidepressants; score ≥34 on the 30-item Inventory of Depressive Symptomatology–Clinician Rated at screening and preinfusion assessment on day 1

Exclusion criteria

primary DSM-IV diagnosis of obsessive-compulsive disorder, posttraumatic stress disorder, anorexia nervosa, or bulimia nervosa or a prior history or current diagnosis of a psychotic disorder, bipolar disorder, mental retardation, borderline personality disorder, mood disorder with postpartum onset, or somatoform disorders; history of previous nonresponse of depressive symptoms to ketamine

Methods

Patients randomized in a 1:1:1:1 ratio to one of the four treatment groups (ketamine or placebo either two or three times per week). Patients fasted overnight (≥8 hours) before study drug administration until two hours after the start of infusion. 

There were four phases: an up-to-four-week screening phase; a four-week double-blind treatment phase (day 1 to day 29); an optional two-week open-label treatment phase; and an up-to-three-week ketamine-free follow-up phase. An optional two-week open-label treatment of IV ketamine 0.5 mg/kg was offered to those who completed at least until day 15 but discontinued due to lack of efficacy before day 29.

Patients continued any antidepressant medications they were receiving at screening, at the same stable dosages throughout the study.

Duration

Each patient completed 13 weeks from July 2012 to September 2013

Outcome Measures

Primary outcome: change from baseline to day 15 in total Montgomery-Asberg Depression Rating Scale (MADRS) score

Secondary outcomes: common and serious adverse reactions

Baseline Characteristics

 

Twice-weekly dosing

Thrice weekly dosing

Placebo (n= 16) Ketamine (n= 18) Placebo (n= 16) Placebo (n= 17)

Age, years

40.3 ± 11.8 45.7 ± 9.6 46.1 ± 10.5 43.3 ± 12.0

Weight, kg

80.5 ± 19.7 80.6 ± 18.6 87.0 ± 23.7 75.9 ± 18.6

Body mass index, kg/m2

27.7 ± 6.4 29.3 ± 6.5 28.8 ± 5.8 26.6 ± 6.2

Baseline MADRS score

35.6 ± 3.8 33.3 ± 4.9 36.8 ± 5.8  35.4 ± 5.3

Women

12 (75%) 12 (66.7%) 9 (56.3%) 12 (70.6%)

White

15 (93.8%) 12 (66.7%) 15 (93.8%) 64.7 (53%)

Number of antidepressants in current episode

1

2

3

4

≥5

 

6 (37.5%)

9 (56.3%)

1 (6.3%)

0 (0%)

0 (0%)

 

9 (50%)

6 (33.3%)

1 (5.6%)

2 (11.1%)

0 (0%)

 

10 (62.5%)

2 (12.5%)

2 (12.5%)

0 (0%)

2 (12.5%)

 

11 (64.7%)

4 (23.5%)

1 (5.9%)

0 (0%)

1 (5.9%)

Results

 

Twice weekly dosing

Thrice weekly dosing

Placebo (n= 16) Ketamine (n= 18) Placebo (n= 16) Ketamine (n= 17)

Mean change in MADRS score from baseline to day 15

-5.7 ± 10.2

-18.4 ± 12*

-3.1 ± 5.7

-17.7 ± 7.3*

Common adverse events

Headaches

Anxiety

Dissociation

Nausea

Dizziness

 

5 (31.3%)

0 (0%)

0 (0%)

1 (6.3%)

1 (6.3%)

 

4 (22.2%)

5 (27.8%)

5 (27.8%)

3 (16.7%)

4 (22.2%)

 

1 (6.3%)

0 (0%)

0 (0%)

2 (12.5%)

0 (0%)

 

7 (41.2%)

1 (5.9%)

1 (5.9%)

4 (23.5%)

2 (11.8%)

Serious adverse events

Anxiety 

Suicide attempt 

 

0 (0%)

0 (0%)

 

1 (5.6%)

1 (5.6%)

 

0 (0%)

0 (0%)

 

0 (0%)

0 (0%)

Discontinued study due to Adverse Event 

1 (6.3%)

4 (22%)

0 (0%)

4 (23%)

*p< 0.001; MADRS= Montgomery-Asberg Depression Rating Scale

Study Author Conclusions

Twice-weekly and thrice-weekly administration of ketamine at 0.5 mg/kg similarly maintained antidepressant efficacy over 15 days. 

InpharmD Researcher Critique

Intravenous ketamine when given twice or three times weekly can be used for treatment-resistant depression with common adverse events such as headaches and anxiety. This study was a double-blind, parallel-group, and randomized with all groups having similar baseline characteristics. One limitation is the short duration of 15 days follow-up. More time could better assess if the clinical benefits can be maintained. 

References:

Singh J, Fedgchin M, Daly E et al. A Double-Blind, Randomized, Placebo-Controlled, Dose-Frequency Study of Intravenous Ketamine in Patients With Treatment-Resistant Depression. American Journal of Psychiatry. 2016;173(8):816-826.

 

Double-blind, placebo-controlled, dose-ranging trial of intravenous ketamine as adjunctive therapy in treatment-resistant depression (TRD)

Design

Double-blind, placebo-controlled study

N= 99

Objective

To compare to active placebo the rapid acting antidepressant properties of a broad range of subanesthetic doses of IV ketamine among outpatients with treatment-resistant depression (TRD) 

Study Groups

Ketamine 0.1 mg/kg (n= 18)

Ketamine 0.2 mg/kg (n= 20)

Ketamine 0.5 mg/kg (n= 22)

Ketamine 1 mg/kg (n= 20)

Midazolam 0.045 mg/kg (n= 19)

Inclusion criteria

adults aged 18-70 years with a diagnosis of major depressive disorder (MDD) in a current depressive episode of at least 8 weeks; had treatment-resistant depression (TRD); were on a stable (for at least four weeks) and adequate dose of ongoing antidepressant therapy, with a total treatment duration of at least 8 weeks; BMI between 18-35 kg/m2; a Montgomery-Asberg Depression Rating Scale (MADRS) score of ≥20 at both screening and baseline visits

Exclusion criteria

MADRS total score of <20; current substance use disorder; history of ketamine or phencyclidine drug use; a history of bipolar disorder, schizophrenia, or schizoaffective disorders; failure to achieve satisfactory response to >7 treatment courses of a therapeutic dose of an antidepressant of at least eight weeks in the current major depressive episode

Methods

Following a washout period for patients on prohibited psychotropic agents, eligible subjects were randomly assigned to one of five 40 minute infusion arms in a 1:1:1:1:1 fashion. Prior to randomization, patients were grouped by body mass index (BMI) (group I: BMI≤ 30; group II: BMI>30) and were block randomized into each arm of the study, with the mg/kg ratio being maintained across all BMIs.

Duration

Follow-up: 30 days

Outcome Measures

Primary outcome: change in 6-item Hamilton Depression Rating Scale (HAM-D-6) score  within 72 hours of ketamine, when added to stable antidepressant therapy

Secondary outcomes: MADRS, self-rated Symptoms of Depression Questionnaire (SDQ), the self-rated Positive Affect Scale (PAS), and global severity and improvement scales of the Clinical Global Impressions (CGI-S) 

Baseline Characteristics

 

Ketamine 0.1 mg/kg (n= 18)

Ketamine 0.2 mg/kg (n= 20)

Ketamine 0.5 mg/kg (n= 22)

Keatmine 1 mg/kg (n= 20)

Midazolam 0.045 mg/kg (n = 19)

Age, years

43.1 ± 11.9 45.5 ± 14.6 48.6 ± 12.9 47.4 ± 10.1 45.6 ± 13.8

BMI, kg/m2

25.2 ± 3.1 24.9 ± 3.7 25.3 ± 5.7 26.1 ± 3.8 26.3 ± 4.1

Female

55.6% 45% 50% 40% 57.9%

White race

66.7%

100%

90.9%

90%

94.7%

Concomitant medications

Benzodiazepines

SSRIs

SNRIs

TCAs

Other antidpressant(s)

 

50%

44.4%

27.8%

5.6%

44.4%

 

50%

60%

35%

0

55%

 

45.5%

54.6%

31.8%

4.6%

59.1%

 

45%

50%

20%

5%

40%

 

31.6%

52.6%

26.3%

0

57.9%

Clinical severity at baseline

Failed antidepressants for current episode

HAM-D-6 score

MADRS score

 

3.3 ± 1.3

12.6 ± 1.8

33.8 ± 5.9

 

3.7 ± 1.6

12.8 ± 2.5

34.5 ± 8.5

 

2.7 ± 1.2

12.6 ± 1.5

31.6 ± 3.9

 

2.9 ± 1.2

12.6 ± 2.1

32.7 ± 5.9

 

2.9 ± 1.4

13.1 ± 2.3

33.6 ± 7.1

BMI, body mass index; SSRIs, selective serotonin reuptake inhibitors; SNRIs, serotonin-norepinephrine reuptake inhibitors; TCAs, tricyclic antidepressants; HAM-D-6, Hamilton Rating Scale for Depression, 6-item version; MADRS, Montgomery-Asberg Depression Rating Scale 

Results

  Ketamine 0.1 mg/kg (n= 14) Ketamine 0.2 mg/kg (n= 16) Ketamine 0.5 mg/kg (n= 21) Ketamine 1.0 mg/kg (n= 17)  

Change in HAM-D-6 score

Day 1

Day 3

 

-3.18

-2.04

 

-1.13

-0.36

 

-4.79*

-3.21

 

-3.76*

-1.84

 
Change in MADRS score after day 3 -5.15 -2.16

-9.85*

-7.72  
* p< 0.05

Adverse Events

Common Adverse Events: headache (11.3% vs 0%), nausea (10% vs 0%), vomiting (5% vs 0%), and depression (3.8% vs 0%)

High systolic blood pressure readings occurred in a total of 21 participants in the ketamine groups, and high diastolic blood pressure readings occurred in a total of 10 participants in the ketamine groups; at 40 minutes after infusion start, both 0.5 mg/kg and 1 mg/kg doses had significantly greater CADSS (Clinician-Administered Dissociative States Scale) scores than active placebo.

Serious Adverse Events: Spontaneously reported suicidal ideation was reported by two ketamine-treated patients, but none of the active-placebo-treated patients; one participant attempted suicide by overdosing on day 11 (received ketamine 0.2 mg/kg in study).

Study Author Conclusions

Results suggest evidence for the efficacy of the 0.5 mg/kg and 1.0 mg/kg subanesthetic doses of IV ketamine and no clear or consistent evidence for clinically meaningful efficacy of lower doses of IV ketamine.

InpharmD Researcher Critique

Treatment groups had small sample sizes, and results may have been confounded by the variability in the degree of responsiveness to ketamine across the treatment groups. This study does not answer the question of whether raising doses in poor responders to the standard dose of 0.5 mg/kg of ketamine is helpful and tolerated, or if lower doses are effective in patients who cannot tolerate the standard ketamine dose of 0.5 mg/kg.  Of this small group, two patients with suicidal ideation and one attempted suicide is noteworthy. 

Additionally, this study had a short duration. Although patients were followed for up to 30 days, their HAM-D and MADRS scores were not assessed at day 30. The midazolam group never had their HAM-D or MADRS scores reported, so there is no comparison to placebo.

References:

Fava M, Freeman MP, Flynn M, et al. Double-blind, placebo-controlled, dose-ranging trial of intravenous ketamine as adjunctive therapy in treatment-resistant depression (TRD). Mol Psychiatry. 2018.

 

Antidepressant efficacy of ketamine in treatment-resistant major depression: a two-site randomized controlled trial

Design

Two-site, parallel-arm, randomized, controlled trial

N= 73

Objective

To evaluate the rapid antidepressant efficacy of ketamine in a large group of patients with treatment-resistant major depression

Study Groups

Ketamine (n= 47)

Midazolam (n= 25)

Inclusion criteria

Patients 21 to 80 years old; major depressive disorder; inadequate response to at least three therapeutic trials of an antidepressant; score of 32 or greater on the Inventory of Depressive Symptomatology

Exclusion criteria

Patients who had a lifetime history of a psychotic illness or bipolar disorder, alcohol or substance abuse in the previous two years, unstable medical illness, serious and imminent suicidal or homicidal risk, a score less than 27 on the Mini-Mental State Examination, or if they were taking contraindicated medications

Methods

Randomly assigned in a 2:1 ratio, patients received a single intravenous infusion of ketamine hydrochloride (0.5 mg/kg) or midazolam (0.045 mg/kg) infused over 40 minutes. Patients were discharged from the research unit 24 hours after the infusion and received outpatient evaluation 48 hours, 72 hours, and seven days post-infusion.

Patients were instructed to abstain from psychotropic medications and to abstain from substances of abuse and alcohol while at home. Nonresponders were considered patients with less than 50% improvement from baseline in the score on the Montgomery-Asberg Depression Rating Scale (MADRS). Nonresponders were no longer followed seven days after the infusion. Responders were followed biweekly until relapse or for an additional four weeks, whichever came sooner.

Duration

Follow-up: at least 7 days post-infusion

Outcome Measures

Primary outcome: reduction in depression severity as assessed on the clinician-administered MADRS 24 hours following infusion

Secondary outcomes: MADRS response rate (defined as reduction in the baseline score by 50% or more), change in score on the Quick Inventory of Depressive Symptomatology, scores on the Clinical Global Improvement (CGI) severity and improvement measures, and durability of benefit for up to 7 days following infusion

Baseline Characteristics

 

Ketamine (n= 47)

Midazolam (n= 25)

Age, years

46.9 ± 12.8 42.7 ± 11.6

Female

26 (55%) 11 (44%)

White

37 (79%) 23 (92%)
Recurrent major depressive disorder 28 (60%) 16 (64%)
Chronic index episode (lasting ≥2 years) 33 (70%) 16 (64%)
Duration of index episode, months 146.6 ± 158.3 109.2 ± 139.0
Previous antidepressant failures 5.1 ± 2.0 5.0 ± 1.8
Montgomery-Asberg Depression Rating Scale score 32.6 ± 6.1 31.1 ± 5.6
16-item Quick Inventory of Depressive Symptomatology score 16.6 ± 4.1 16.3 ± 4.5

Results

 

Ketamine (n= 47)

Midazolam (n= 25)

MADRS score (95% CI)

After 24 hours

After 7 days

 

14.77 (11.73-17.80)

17.85 (14.29-21.42)

 

22.72 (18.85-26.59)

23.54 (18.29-28.79

Quick Inventory of Depressive Symptomatology-Self Report score (95% CI)

After 24 hours

After 7 days

 

8.38 (6.71-10.05)

8.58 (6.92-10.24)

 

11.78 (9.63-13.92)

11.42 (8.87-13.97)

≥50% decrease in MADRS score after 24 hours 30 (64%) 7 (28%)

Clinical Global Impression Scale

Improvement rating of 2 (much improved) or 1 (very much improved)

Severity rating of 2 (minimally ill) or 1 (not at all ill)

 

29 (62%)

25 (53%)

 

6 (24%)

2 (8%)

Linear modeling showed that patients had significantly lower MADRS scores at day 7 with ketamine compared to midazolam (p≤ 0.02).

Adverse Events

Common Adverse Events: 

  • Most common in ketamine group within four hours of infusion (ketamine vs midazolam): dizziness (45% vs 20%), blurred vision (43% vs 8%), headache (32% vs 20%), nausea or vomiting (34% vs 12%), dry mouth (26% vs 16%), poor coordination (26% vs 12%), poor concentration (26% vs 8%), restlessness (21% vs 20%)
  • Most common adverse events in the midazolam group within four hours of infusion (ketamine vs midazolam): general malaise (6% vs 28%), dizziness (45% vs 20%), headache (32% vs 20%), restlessness (21% vs 20%), nausea or vomiting (34% vs 12%), dry mouth (26% vs 16%), decreased energy (15% vs 12%), poor coordination (26% vs 12%)
  • Dissociative symptoms were observed immediately after ketamine infusion in 8 patients receiving ketamine (17%).

Serious Adverse Events: No severe psychotic symptoms (paranoia, hallucinations, delusions, or thought disorders) or other serious events were noted in any patient.

Percentage that Discontinued due to Adverse Events: Treatment was discontinued for two patients in the ketamine group because of hemodynamic changes (4.2%).

Study Author Conclusions

Treatment-resistant patients in a major depressive episode showed a rapid antidepressant response to a single infusion of ketamine.

InpharmD Researcher Critique

There was stringent enrollment criteria due to concerns about ketamine's psychoactive effects and abuse liability leading to small sample sizes. The follow-up was also relatively short term, with the study only being powered to detect a major difference after 24 hours. There was no report of patients who were followed longer than 7 days.
References:

Murrough JW, Iosifescu DV, Chang LC, et al. Antidepressant efficacy of ketamine in treatment-resistant major depression: a two-site randomized controlled trial. Am J Psychiatry. 2013;170(10):1134‐1142.