Cost-Effectiveness of Treatment Alternatives for Treatment-Refractory Pediatric Obsessive-Compulsive Disorder

Highlights

• 9 strategies for treatment of refractory obsessive-compulsive disorder are assessed.

• Intensive Outpatient Patient (IOP) treatment outperformed trial-based strategies.

• IOP improved CY-BOCS by 16.42 units compared to 15.56 for the nearest strategy.

• Improvements delivered for an Incremental Cost Effectiveness Ratio (ICER) of $48,834.

• Lack of access to intensive treatment approaches is a barrier for patients and families.

Abstract

Purpose

Current guidelines for first-line treatment of childhood OCD are cognitive-behavioral therapy (CBT) utilizing exposure and response prevention (ERP), and/or antidepressant (ADM) pharmacotherapy, specifically serotonin reuptake inhibitors (SRI). Given that first-line are relatively similar in terms of clinical effectiveness, the role of costs to provide such services may help influence treatment decisions. In the case of treatment refractory pediatric OCD, this cost-effectiveness analysis (CEA) aims to further evaluate two additional, higher intensity combination therapies, namely OCD-specific Intensive Outpatient (IOP) and Partial Hospitalization Programs (PHP), to determine the additional benefits, in terms of effectiveness, that may result, and the corresponding increase in costs for these higher-intensity courses of therapy.

Results

IOP was the most cost-effective strategy in terms of change in CY-BOCS, pre/post treatment, equal to 16.42 units, followed by PHP and CBT monotherapy augmented with ADM CBT-monotherapy augmented with additional CBT and ADM-only augmented with CBT followed closely with 15.56 and 14.75 unit improvements in CY-BOCS. IOP accomplished its superior cost-effectiveness with an Incremental Cost-Effectiveness Ratio (ICER), of $48,834, lower than either of the established willingness to Pay thresholds.

Conclusions

Lack of access to high fidelity, high dose CBT paired with pharmacotherapy is an issue for OCD patients and families. Among youth who were treatment non-responsive, these results indicate the superiority of a high dosage CBT strategy, indicating the need to increase access to these treatments.

Introduction

Obsessive-compulsive disorder (OCD) affects 1-2% of children and adolescents (Zohar, 1999), confers significant functional (Storch, Larson et al., 2010) and familial (Lebowitz, Panza, & Bloch, 2016; Wu et al., 2016) impairment, and negatively impinges upon quality of life (Lack et al., 2009). Without treatment, children are at risk of experiencing chronic symptomology (Bloch et al., 2013; Stewart et al., 2004). Current guidelines for first-line treatment of childhood OCD are cognitive-behavioral therapy (CBT) utilizing exposure and response prevention (ERP), and/or antidepressant (ADM) pharmacotherapy, specifically serotonin reuptake inhibitors (SRI) (Geller & March, 2012; Lewin, Park et al., 2014; Lewin, Wu, McGuire, & Storch, 2014). As many as 85% of children respond to CBT monotherapy, while 50-60% respond to pharmacological monotherapy. It is unclear if combined treatment (CBT + ADM) confers additional benefit beyond CBT alone with some studies finding the affirmative (Pediatric OCD Treatment Study (POTS) Team (2004)), and others finding no advantage for children with OCD of moderate or worse severity (Storch et al., 2013a, 2013b). For those who fail to respond to first-line therapies, there is little evidence available to support clinicians, patients, and their parents/family in terms of what course of treatment to pursue next.

CBT is effective in reducing symptoms in treatment naïve children and adolescents, and may be more effective that pharmacotherapy alone (Ivarsson et al., 2015; McGuire et al., 2015; Pediatric OCD Treatment Study (POTS) Team (2004)). Pharmacotherapy consists of SRIs, approved for use in children and adolescents (Geller et al., 2003; Geller & March, 2012; Varigonda, Jakubovski, & Bloch, 2016). Comparison trials have demonstrated combined treatment and CBT monotherapy was more effective than ADM monotherapy; however, it remains unclear if there is a significant difference between combination therapy and CBT monotherapy for pediatric patients with moderate severity (Ivarsson et al., 2015; McGuire et al., 2015; Romanelli, Wu, Gamba, Mojtabai, & Segal, 2014). For pediatric patients with high severity (Franklin et al., 2011; Geller & March, 2012; Simpson et al., 2008), CBT combined with pharmacotherapy has been demonstrated to be effective (Franklin et al., 2011; Ivarsson et al., 2015).

The cost-effectiveness of treatments for refractory pediatric OCD has yet to be examined. For the purposes of this analysis, “refractory” is defined as individuals that have received an adequate dose of cognitive behavioral therapy (∼12 weeks) and initiated ADM without significant improvement in symptomology, as measured by C-YBOCS. Patients initiated up to two ADM molecules without significant symptom improvement or discontinued due to lack of tolerability. This is definition of refractory is consistent inclusion criteria across the trials and admission criteria for the practice-based participants (Bloch & Storch, 2015). The contribution and advantage of cost-effectiveness analysis (CEA) is the ability to compare the effectiveness of treatments and their respective costs, ranking treatment alternatives by the incremental cost effectiveness ratio (ICER), a ratio of costs to effectiveness, therefore revealing the treatment strategies that yield the largest marginal effectiveness gain per unit of cost. Given that first-line therapies - combined ADM + CBT, and ADM and CBT monotherapy - are relatively similar in terms of clinical effectiveness, the role of costs to provide such services may help influence treatment decisions.

In the case of treatment refractory pediatric OCD, this CEA aims to further evaluate two additional, higher intensity combination therapies, namely OCD-specific Intensive Outpatient (IOP) and Partial Hospitalization Programs (PHP), to determine the additional benefits, in terms of effectiveness, that may result, and the corresponding increase in costs for these higher-intensity courses of therapy. Intensive treatment approaches were selected given evidence (Storch et al., 2007, 2010b) supporting its effectiveness in pediatric OCD, as well as strong supporting data among adults with OCD (Abramowitz, Foa, & Franklin, 2003; Foa et al., 2005).

In clinical practice, two other treatment strategies are being employed to treat refractory OCD in adults (Gregory et al., 2018), have shown superior cost-effectiveness in adults, and should be evaluated for pediatric OCD. For adults with treatment-refractory OCD, a similar strategy, PHP with a step-down to IOP was determined to be the most cost-effective (Gregory et al., 2018), compared to trial-based strategies, followed by PHP and IOP individually. This study inherits shared outcomes data, and approach with a similar study for adults (Gregory et al., 2018). No evidence for the PHP to IOP step-down strategy was available for pediatric OCD patients; therefore, it was not included in this analysis. However, PHP and IOP strategies were included as comparisons to the seven trial-based strategies.

Specifically, this study compares a total of nine treatment strategies, in terms of net health benefits, costs and incremental cost-effectiveness. We compared nine treatment strategies, beginning with seven first line therapies identified in the trial literature. There are three primary strategies, (1) ADM-only (DeVeaugh-Geiss et al., 1992; Geller et al., 2001, 2004; Greist et al., 1990; Liebowitz et al., 2002; March et al., 1998; Riddle et al., 2001, 1992), (2) CBT-only (Barrett, Healy-Farrell, & March, 2004; Bolton & Perrin, 2008; DeVeaugh-Geiss et al., 1992; Freeman et al., 2014a, 2014b; Freeman et al., 2008; Geller et al., 2001, 2004; Greist et al., 1990; Lewin, Park et al., 2014, 2014b; Liebowitz et al., 2002; March et al., 1998; Pediatric OCD Treatment Study (POTS) Team (2004); Piacentini et al., 2011; Riddle et al., 2001, 1992; Skarphedinsson et al., 2015a, 2015b; Storch et al., 2013a, 2013b), and (3) combined ADM + CBT (Pediatric OCD Treatment Study (POTS) Team (2004); Storch et al., 2013a, 2013b). These three strategies are then augmented into four additional strategies, (4) ADM-only augmented with an additional continued course of ADM (Franklin et al., 2011), (5) CBT-only augmented with an additional continued course of CBT (Skarphedinsson et al., 2015a, 2015b), and (6) ADM-only, augmented with CBT (Franklin et al., 2011), and (7) combined ADM + CBT, augmented with an additional course of ADM + CBT. These 7 trial-based strategies are all ambulatory-based pharmacology (ADM) and behavioral therapy (CBT).

In addition to evidence from trials we included evidence for two additional higher-intensity strategies. Two additional strategies included two variations in CBT intensity/dosage (Kay, Eken, Jacobi, Riemann, & Storch, 2016; Storch et al., 2007, 2010b), (8) IOP consisting of 12-15 hours per week of multimodal treatment 4-5 days/week for 12 weeks, and (9) PHP consisting of 30 hours of multimodal treatment 5 days/week, for 12 weeks. Multimodal therapy included CBT and Exposure-response therapy (ERP) within the behavioral therapy regime, each week (Gregory et al., 2018). Both of these practice-based strategies also include substantial medication management and optimization of pharmacology during the course of therapy, and for the balance of the 12-months inclusive of the treatment episode.

Cost-effectiveness parameters for these strategies sourced from an outcomes database maintained by a specialty center that delivers these treatment modalities to individuals with severe OCD. Our aim in including these strategies was to synthesize the both trial evidence, and specialty center evidence for treatment effectiveness, and denominate in terms of effectiveness, to assess the reasonable treatment alternatives available to patients and families. This is consistent with the approach of intergrading trial evidence and outcomes from practice, in a recent assessment of CEA for adult refractory OCD (Gregory et al., 2018).

We hypothesize that these high intensity, multimodal treatment strategies, PHP and/or IOP, will be more cost-effective the trial-based treatment approaches. The results of this study can serve as a guide to support informed decision-making for providers, and patients and their parents regarding optimum treatment of refractory OCD among children and adolescents.