National Institute for Health and Clinical Excellence (NICE)
Many jurisdictions, inspired by early examples from Australia and Canada, have mandated the use of evaluations in reimbursement decisions, particularly concerning pharmaceuticals A key player in this initiative is the National Institute of Health and Clinical Excellence (NICE), established as a Special Health Authority within the UK National Health Service (NHS).
Since its inception as the “National Institute for Clinical Excellence” in April
Since 1999, the technology appraisals conducted by the National Institute for Health and Clinical Excellence (NICE) have gained significant international recognition Their prominence has allowed them to influence areas beyond their primary focus of guiding the National Health Service (NHS) in England and Wales Consequently, NICE is often viewed as a role model in the field.
To develop objective, unbiased, and methodologically sound
Guidance for the NHS in England and Wales
(on interventional procedures, also for the NHS in Scotland)
Guidance on public health interventions and programs
Technology Appraisals; interventional procedure guidance
Promotion of good health and prevention of ill health
Recommendations on the use of new and existing medicines and treatments
Recommendations on treatment and care of people with specific diseases and conditions
Centre for Health Technology Evaluation
National Institute for Health and Clinical Excellence
Fig 1.1 Roles and responsibilities of NICE and its parts
Established in 1999 as a Special Health Authority within the National Health Service (NHS), NICE aims to promote clinical excellence and optimize resource use in England and Wales It consists of three distinct "Centres of Excellence," each with specific yet interconnected roles This report focuses on NICE's Technology Appraisal function and its Centre for Health Technology Evaluation, emphasizing the importance of cost-effectiveness analysis (CEA) as a key component of health technology assessments (HTAs) to facilitate informed decision-making.
NICE utilizes a specific form of cost-effectiveness analysis known as cost-utility analysis (CUA), which employs quality-adjusted life years (QALYs) as a universal metric for assessing health outcomes In this context, CUA will be distinguished from traditional cost-effectiveness analysis (CEA), which may use various health outcome measures, including QALYs, depending on the decision-making scenario Additionally, in the United States, the term "cost-effectiveness analysis" serves as an overarching term that encompasses both CEA and CUA.
1.1 National Institute for Health and Clinical Excellence (NICE) 5 the rational allocation of health care resources in an environment of economic limitations.
Leading health economists have raised concerns about the underutilization of economic evaluation (Drummond, 2004; Neumann, 2004), yet NICE has been praised for its exemplary approach to priority-setting in healthcare, described as the closest realization of the economist's ideal (Williams, 2004) It is noted that NICE focuses on challenging decisions that involve trade-offs between enhanced benefits and increased costs, highlighting situations where economic analysis can provide significant value, particularly in quantifying decision-related uncertainties (Drummond, 2004).
Technology appraisals are essential for NICE to formulate guidance on the optimal use of health technologies, addressing uncertainties regarding their value and ensuring standardized access across the country.
The implementation of NICE guidance is mandatory for the NHS in England and Wales, though its actual application has sparked debate (Freemantle, 2004; Sheldon et al., 2004; Burke, 2002) This guidance is intended to shape clinical guidelines developed by National Collaborating Centres and Guideline Developers (NICE, 2006g,h, 2004a) Furthermore, NICE guidance should be incorporated unchanged into clinical guidelines and assigned the highest strength of evidence ranking (NICE, 2004a), reflecting the expectation of consistently achieving the highest quality standards.
A review team of the World Health Organization (WHO) commissioned by NICE to appraise the methods and processes of its technology appraisal program
The World Health Organization (2003) praised the rigorous methodology employed in technology assessment, highlighting several key achievements These include a transparent process, active stakeholder involvement, adaptability to changes, a dedication to utilizing the best available evidence, and the engagement of academic centers of excellence for independent evaluations.
The WHO review team recognized that "published technology appraisals are already being used as international benchmarks" and recommended enhancing NICE's operations to assist similar organizations worldwide in overcoming challenges (WHO, 2003) While the report focused on the methods and scientific robustness of technology appraisals, it was seen as affirming NICE's status as a leading international authority on using evidence for clinical and cost-effectiveness in health decision-making (Devlin et al., 2003) This recognition has prompted calls from health economists to expand the NICE approach globally, leading to discussions by the European High-Level Group on Innovation and Provision of Medicines about establishing a "Euro-NICE" (Quam and Smith, 2005; Neumann et al., 2005a; WHO, 2003; Maynard, 2001b).
In July 2006, the German government initiated healthcare reforms, expanding the role of the German Institute for Quality and Efficiency in Health Care (IQWiG) to include cost-benefit evaluations of pharmaceuticals, aligning with international standards such as those set by NICE This shift reflects a broader trend in North America, where discussions are increasingly influenced by the UK's experiences The challenges faced by the Medicare Part D program in the United States have sparked interest in alternative methods for making informed drug choices based on evidence of efficacy, safety, and economic value Proposals for an independent information infrastructure aim to enhance the dissemination of data regarding the cost-effectiveness of pharmaceuticals.
In recent years, experts have proposed the establishment of new institutes to advise Medicare on the cost-effectiveness of new medical interventions (Neumann et al., 2005a; Reinhardt, 2004) Prominent representatives from NICE have expressed that the current conditions in the U.S are favorable for implementing a similar organization (Pearson and Rawlins, 2005).
The economic evaluation approach utilized by NICE has sparked considerable debate, as highlighted by various studies (Dolan et al., 2005; Maynard et al., 2004; Rawlins and Culyer, 2004) This controversy is not unexpected in the ideologically charged and heavily subsidized health care sector, where guidelines on technology use significantly impact financial interests Consequently, organizations like NICE often face criticism from stakeholders when their recommendations lead to restrictions on usage or denial of reimbursement (Ferner and McDowell, 2006; O’Brien, 2006).
The National Institute for Health and Care Excellence (NICE) is widely recognized for its exemplary processes and transparency in health technology assessment, demonstrating the potential of organizations focused on cost-effectiveness (Schlander, 2007a; WHO, 2003; Towse and Pritchard, 2002; Buxton, 2001) Recently, NICE has updated its methods guidance to include probabilistic sensitivity analyses, reinforcing its leadership in this critical field (Buxton, 2006; Neumann et al., 2005a).
2 Apparently there is still some confusion among German politicians with regard to terminol- ogy, such as the important differentiation between “cost-benefit” and “cost-effectiveness” / “cost- utility” evaluations.
For years, traditional methods for evaluating parameter uncertainty in cost-effectiveness analyses have relied on one-way or multi-way sensitivity analyses, as noted by Briggs (2000, 2001) and Petitti (2000) These analyses reveal the potential range of outcomes when key model parameters are altered In contrast, probabilistic sensitivity analyses provide insights into the likelihood of various results, often utilizing Monte Carlo simulations to enhance the robustness of the findings (Briggs and Gray).
1.1 National Institute for Health and Clinical Excellence (NICE) 7
The British Medical Journal editors, initially cautious and skeptical, have praised NICE as a significant achievement, labeling it a "triumph" and suggesting it could become one of Britain's most impactful cultural exports, alongside icons like Shakespeare, Newtonian physics, the Beatles, Harry Potter, and the Teletubbies (Claxton et al., 2005; NICE, 2004c; Smith, 2000, 2004).
However, little is known about the real-life robustness of the NICE approach.
Attention-Deficit/Hyperactivity Disorder (ADHD)
ADHD is defined by a consistent pattern of inattention and/or hyperactivity/impulsivity that is more intense than what is typically seen in individuals of similar developmental levels (American Psychiatric Association, 1994) While economic research on ADHD is still emerging and primarily focused in the United States, it is evident that this disorder imposes a significant economic burden (Matza et al., 2005a; Leibson and Long, 2003).
The appraisal of ADHD interventions by NICE is noteworthy due to its relevance and complexity, highlighting significant considerations in the evaluation process.
ADHD is widely recognized as the most prevalent psychiatric disorder among children and adolescents, with prevalence estimates based on DSM-IV criteria varying significantly Studies show that rates in 8- to 10-year-old children can range from 3.9% to 19.8%, influenced by factors such as the specific population examined and the diagnostic criteria applied (Faraone et al., 2003a).
ADHD is frequently linked to a high prevalence of comorbid conditions, with 50–60% of affected children exhibiting externalizing disorders such as oppositional defiant disorder and conduct disorder Additionally, 12–26% of children with ADHD experience internalizing issues, including anxiety and depression Various other psychiatric, neurological, and somatic disorders may also coexist with ADHD symptoms, highlighting the complexity of this condition.
In a systematic review by Green et al (1999), estimates of psychiatric comorbidities among individuals with ADHD indicate that 30% experience oppositional defiant and conduct disorders, 28% have conduct disorder, 26% suffer from anxiety, 18% are affected by depression, and 12% face learning disabilities Additionally, a study by Steinhausen et al (based on a European sample of 1,478 patients with ADHD or hyperkinetic disorder, Preuss et al., 2006) supports these findings.
(2006) reported the following rates of co-existing symptoms “of any degree:” oppositional-defiant
1.2 Attention-Deficit/Hyperactivity Disorder (ADHD) 9
Age e c n el a v er P male female
In 2003, the prevalence rates of ADHD among children and adolescents in Nordbaden, Germany, were analyzed using administrative data, revealing specific trends based on age and gender (Schlander and Schwarz, 2005a; Schlander et al., 2007) For detailed characteristics of the comprehensive Nordbaden claims database, refer to Box 1.
Table 1.1 Prevalence of co-existing conditions in children and adolescents with ADHD Co-existing disorders ADHD patients Control group Relative Risk (CI 1 )
Specific development disorders of scholastic skills
Disorders due to brain damage 1.8% 0.4% 4.95(3.54–6.92)
Disorders associated with sexual development
Disorders due to substance abuse 0.6% 0.2% 3.65(2.13–6.23)
A comprehensive analysis of administrative data from Nordbaden, Germany, reveals significant rates of psychiatric and developmental comorbidities among ADHD patients in a large European cohort, with all group differences showing statistical significance (p