A crisis in US drug pricing: Consequences for patients with neuromuscular diseases, physicians and society, part 1.

Drug prices in the United States have reached astounding heights, negatively impacting patients and society. The vast majority of prescription drug spending is on brand name drugs, which are protected from typical market pressures by FDA exclusivity and intellectual property patents. Drugs to treat "orphan" diseases, of particular relevance to neuromuscular clinicians, are some of the most expensive in all of medicine. The Orphan Drug Act's original intent was to incentivize the creation of drugs that would otherwise provide little economic payoff. While it has facilitated incredible, life-changing drugs for our patients, it has also become a source of abuse. Many expensive drugs approved under the Orphan Drug Act were previously available for compassionate use or for another indication at much lower prices. As patients increasingly face high drug prices, it is important for clinicians to understand a drug's risk for inducing financial toxicity, as the financial and emotional consequences of an overpriced low-value drug may outweigh its intended benefit.

A crisis in US drug pricing: Consequences for patients with neuromuscular diseases, physicians, and society, part 2.

Escalating drug costs place patients at risk for financial toxicity and demand that physicians understand and act on the ethical and economic principles related to drug pricing. This manuscript reviews these principles and provides clinicians with a framework to think about the value of the drugs prescribed for patients with neuromuscular diseases. A key component of addressing the drug pricing crisis will be establishing a value based (benefit/cost) drug pricing framework. Determining the value of a drug is difficult and requires estimating the benefit and costs to patients and society while integrating indirect and contextual variables. Other considerations in drug pricing include "externality," the value to society derived from innovation. The Institute for Clinical and Economic Review (ICER) is a leading independent research organization providing clinicians with value-based price "benchmarks." All physicians must educate themselves in drug pricing principles and be prepared to have conversations regarding individual and societal value with the patients they serve.

Health and Economic Outcomes of Posterior Spinal Fusion for Children With Neuromuscular Scoliosis.

OBJECTIVES: Neuromuscular scoliosis (NMS) can result in severe disability. Nonoperative management minimally slows scoliosis progression, but operative management with posterior spinal fusion (PSF) carries high risks of morbidity and mortality. In this study, we compare health and economic outcomes of PSF to nonoperative management for children with NMS to identify opportunities to improve care. METHODS: We performed a cost-effectiveness analysis. Our decision analytic model included patients aged 5 to 20 years with NMS and a Cobb angle ≥50°, with a base case of 15-year-old patients. We estimated costs, life expectancy, quality-adjusted life-years (QALYs), and incremental cost-effectiveness from published literature and conducted sensitivity analyses on all model inputs. RESULTS: We estimated that PSF resulted in modestly decreased discounted life expectancy (10.8 years) but longer quality-adjusted life expectancy (4.84 QALYs) than nonoperative management (11.2 years; 3.21 QALYs). PSF costs $75 400 per patient. Under base-case assumptions, PSF costs $50 100 per QALY gained. Our findings were sensitive to quality of life (QoL) and life expectancy, with PSF favored if it significantly increased QoL. CONCLUSIONS: In patients with NMS, whether PSF is cost-effective depends strongly on the degree to which QoL improved, with larger improvements when NMS is the primary cause of debility, but limited data on QoL and life expectancy preclude a definitive assessment. Improved patient-centered outcome assessments are essential to understanding the effectiveness of NMS treatment alternatives. Because the degree to which PSF influences QoL substantially impacts health outcomes and varies by patient, clinicians should consider shared decision-making during PSF-related consultations.

[Health policy indicators of neuromusculosceletal rehabilitation in Hungary]. / A neuromusculoskeletalis rehabilitáció szakmapolitikai indikátorai Magyarországon.

INTRODUCTION: With the increasing number of the incidence of neuromusculoskeletal and brain circulation disorders, there is a higher demand for neuromusculoskeletal rehabilitation care. AIMS: The aim of our study is to analyse the performance indicators of neuromusculoskeletal rehabilitation care in Hungary financed by the statutory public health insurance system. METHODS: Data were derived from the financial database of the National Health Insurance Fund of Hungary. We analysed the period between 2014 and 2017. We investigated the distribution of neuromusculoskeletal rehabilitation hospital beds, the patient turnover and patients' pathways. We analysed the regional inequalities in the access to (hospital beds) and utilization (number of patients) of rehabilitation care. RESULTS: In 2017, there were 6798 publicly financed neuromusculoskeletal rehabilitation hospital beds in Hungary (6.94 beds/10 000 population). We observed the lowest number of hospital bed in Komárom-Esztergom (1.5 beds/10 000 population), Somogy (2.0) and Pest (2.7) counties. We found the highest number of hospital beds in Zala (12.6), Gyor-Moson-Sopron (12.2) and Baranya (11.5) counties. The more than 2-fold difference in the utilization (Komárom-Esztergom: 52.3 patients/10 000 population; Gyor-Moson-Sopron: 136 patients/10 000 population) confirms regional inequalities. Between 2014 and 2017, the annual number of patients showed an increasing tendency, while the average length of stay varied between 21.8 and 22.4 days/patient. The correlation coefficient between hospitals beds and the number of patients was very high (0.798). CONCLUSION: We found significant regional inequalities in the access to and utilization of neuromusculoskeletal rehabilitation. Orv Hetil. 2019; 160(Suppl 1): 13-21.

Developing multidisciplinary clinics for neuromuscular care and research.

Multidisciplinary care is considered the standard of care for both adult and pediatric neuromuscular disorders and has been associated with improved quality of life, resource utilization, and health outcomes. Multidisciplinary care is delivered in multidisciplinary clinics that coordinate care across multiple specialties by reducing travel burden and streamlining care. In addition, the multidisciplinary care setting facilitates the integration of clinical research, patient advocacy, and care innovation (e.g., telehealth). Yet, multidisciplinary care requires substantial commitment of staff time and resources. We calculated personnel costs in our ALS clinic in 2015 and found an average cost per patient visit of $580, of which only 45% was covered by insurance reimbursement. In this review, we will describe classic and emerging concepts in multidisciplinary care models for adult and pediatric neuromuscular disease. We will then explore the financial impact of multidisciplinary care with emphasis on sustainability and metrics to demonstrate quality and value. Muscle Nerve 56: 848-858, 2017.

The value transformation of health care: Impact on neuromuscular and electrodiagnostic medicine.

Beginning in 2017, most physicians who participate in Medicare are subject to the Medicare Access and CHIP Reauthorization Act (MACRA), the milestone legislation that signals the US health care system's transition from volume-based to value-based care. Here we review emerging trends in development of value-based healthcare systems in the US. MACRA and the resulting Quality Payment Program create 2 participation pathways, the Merit-based Incentive Payment System (MIPS) and the Advanced Alternative Payment Model (AAPM) pathway. Although there are several program incentives for AAPM participation, to date there have been few AAPM options for specialists. MIPS and its widening bonus and penalty window will likely be the primary participation pathway in the early years of the program. Value-based payment has the potential to reshape health care delivery in the United States, with implications for neuromuscular and electrodiagnostic (EDX) specialists. Meaningful quality measures are required for neuromuscular and EDX specialists. Muscle Nerve 56: 679-683, 2017.

Social and Economic Impacts of Managing Sleep Hypoventilation Syndromes.

Hypoventilation during sleep is often an early indicator of the development of respiratory failure. Alterations in ventilation are more pronounced during sleep and often present before the onset of daytime symptoms. This article discusses the most common sleep-related hypoventilatory disorders and recommended treatment approaches for obesity hypoventilation, chronic obstructive pulmonary disease, and neuromuscular disorders. Accurate diagnosis and appropriate treatment is of paramount importance because of the impact on individual health outcomes and overall cost of health care delivery. Appropriate treatment is successful at reducing hospitalizations and health care costs as well as improving quality of life and individual economic burden.

Trends in Health Care Spending for Children in Medicaid With High Resource Use.

OBJECTIVES: To assess characteristics associated with health care spending trends among child high resource users in Medicaid. METHODS: This retrospective analysis included 48 743 children ages 1 to 18 years continuously enrolled from 2009-2013 in 10 state Medicaid programs (Truven MarketScan Medicaid Database) also in the top 5% of all health care spending in 2010. Using multivariable regression, associations were assessed between baseline demographic, clinical, and health services characteristics (using 2009-2010 data) with subsequent health care spending (ie, transiently, intermittently, persistently high) from 2011-2013. RESULTS: High spending from 2011-2013 was transient for 54.2%, persistent for 32.9%, and intermittent for 12.9%. Regarding demographic characteristics, the highest likelihood of persistent versus transient spending occurred in children aged 13 to 18 years versus 1 to 2 years in 2010 (odds ratio [OR], 3.0 [95% confidence interval (CI), 2.7-3.4]). Regarding clinical characteristics, the highest likelihoods were in children with ≥6 chronic conditions (OR, 4.8 [95% CI, 3.5-6.6]), a respiratory complex chronic condition (OR, 2.5 [95% CI, 2.2-2.8]), or a neuromuscular complex chronic condition (OR, 2.3 [95% CI, 2.2-2.5]). Hospitalization and emergency department (ED) use in 2010 were associated with a decreased likelihood of persistent spending in 2011-2013 (hospitalization OR, 0.7 [95% CI, 0.7-0.7]); ED OR, 0.8 [95% CI, 0.8-0.8]). CONCLUSIONS: Most children with high spending in Medicaid are without persistently high spending in subsequent years. Adolescent age, multiple chronic conditions, and certain complex chronic conditions increased the likelihood of persistently high spending; hospital and ED use decreased it. These data may help inform the development of new models of care and financing to optimize health and save resources in children with high resource use.

Management of Ventilatory Insufficiency in Neuromuscular Patients Using Mechanical Ventilator Supported by the Korean Government.

Since 2001, financial support has been provided for all patients with neuromuscular disease (NMD) who require ventilatory support due to the paralysis of respiratory muscles in Korea. The purpose of this study was to identify ventilator usage status and appropriateness in these patients. We included 992 subjects with rare and incurable NMD registered for ventilator rental fee support. From 21 February 2011 to 17 January 2013, ventilator usage information, regular follow-up observation, and symptoms of chronic hypoventilation were surveyed by phone. Home visits were conducted for patients judged by an expert medical team to require medical examination. Abnormal ventilatory status was assessed by respiratory evaluation. Chronic respiratory insufficiency symptoms were reported by 169 of 992 subjects (17%), while 565 subjects (57%) did not receive regular respiratory evaluation. Ventilatory status was abnormal in 102 of 343 home-visit subjects (29.7%). Although 556 subjects (56%) reported 24-hour ventilator use, only 458 (46%) had an oxygen saturation monitoring device, and 305 (31%) performed an airstacking exercise. A management system that integrates ventilator usage monitoring, counselling and advice, and home visits for patients who receive ventilator support could improve the efficiency of the ventilator support project.

Cost-effectiveness of shared medical appointments for neuromuscular patients.

OBJECTIVE: To assess whether shared medical appointments (SMAs) for neuromuscular patients represent a way of using clinicians' time efficiently without compromising quality of care for patients. METHODS: Patients with a chronic neuromuscular disease (NMD) (n = 272) were randomly allocated to either an SMA or a regular individual annual appointment and followed up for a period of 6 months. Data on resource utilization and quality of life (EQ-5D) were collected prospectively, using a health care perspective. Incremental costs and changes in quality-adjusted life-years (QALYs) were computed using a probabilistic decision model. Factors critical to the incremental cost-effectiveness of SMAs were explored in sensitivity analyses. RESULTS: No substantial differences between SMAs and individual visits in terms of costs per QALY were found (incremental cost-effectiveness ratio €-960.00; 95% confidence interval €-34,600.00, €+36,800.00). Sensitivity analyses showed that the cost-effectiveness ratio was particularly sensitive to SMA group size and proportion of patients seeing their treating neurologist. CONCLUSIONS: Cost-effectiveness of SMAs did not show a significant difference vs that of individual appointments based on data from our randomized controlled trial. On the other hand, we were able to show that a minimum of 6 patients per SMA and 75% of patients attending their treating neurologist are specific conditions under which SMAs qualify as a cost-effective alternative. This implies that SMAs may be a means to increase productivity of the physician without compromising quality of care. CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that SMAs are not significantly more cost-effective than individual appointments for patients with NMDs. The study lacks the precision to exclude important differences in cost-effectiveness between SMAs and individual appointments.

Cost of illness for neuromuscular diseases in the United States.

INTRODUCTION: We conducted a comprehensive study of the costs associated with amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD). and myotonic dystrophy (DM) in the U.S. METHODS: We determined the total impact on the U.S. economy, including direct medical costs, nonmedical costs, and loss of income. Medical costs were calculated using a commercial insurance database and Medicare claims data. Nonmedical and indirect costs were determined through a survey of families registered with the Muscular Dystrophy Association. RESULTS: Medical costs were driven by outpatient care. Nonmedical costs were driven by the necessity to move or adapt housing for the patient and paid caregiving. Loss of income correlated significantly with the amount of care needed by the patient. CONCLUSIONS: We calculated the annual per-patient costs to be $63,693 for ALS, $50,952 for DMD, and $32,236 for DM. Population-wide national costs were $1,023 million (ALS), $787 million (DMD), and $448 million (DM).

Effects of shared medical appointments on quality of life and cost-effectiveness for patients with a chronic neuromuscular disease. Study protocol of a randomized controlled trial.

BACKGROUND: Shared medical appointments are a series of one-to-one doctor-patient contacts, in presence of a group of 6-10 fellow patients. This group visits substitute the annual control visits of patients with the neurologist. The same items attended to in a one-to-one appointment are addressed. The possible advantages of a shared medical appointment could be an added value to the present management of neuromuscular patients. The currently problem-focused one-to-one out-patient visits often leave little time for the patient's psychosocial needs, patient education, and patient empowerment. METHODS/DESIGN: A randomized, prospective controlled study (RCT) with a follow up of 6 months will be conducted to evaluate the clinical and cost-effectiveness of shared medical appointments compared to usual care for 300 neuromuscular patients and their partners at the Radboud University Nijmegen Medical Center. Every included patient will be randomly allocated to one of the two study arms. This study has been reviewed and approved by the medical ethics committee of the region Arnhem-Nijmegen, The Netherlands. The primary outcome measure is quality of life as measured by the EQ-5D, SF-36 and the Individualized neuromuscular Quality of Life Questionnaire. The primary analysis will be an intention-to-treat analysis on the area under the curve of the quality of life scores. A linear mixed model will be used with random factor group and fixed factors treatment, baseline score and type of neuromuscular disease. For the economic evaluation an incremental cost-effectiveness analysis will be conducted from a societal perspective, relating differences in costs to difference in health outcome. Results are expected in 2012. DISCUSSION: This study will be the first randomized controlled trial which evaluates the effect of shared medical appointments versus usual care for neuromuscular patients. This will enable to determine if there is additional value of shared medical appointments to the current therapeutical spectrum. When this study shows that group visits produce the alleged benefits, this may help to increase the acceptance of this innovative and creative way of using one of the most precious resources in health care more efficiently: time. TRIAL REGISTRATION: DutchTrial Register http://www.trialregister.nlNTR1412.

Electrodiagnostic consultation and identification of neuromuscular conditions in persons with diabetes.

INTRODUCTION: Although the American Association of Neuromuscular and Electrodiagnostic Medicine recommends that electrodiagnostic procedures should be performed by physicians with specialty training, these procedures are increasingly being performed by non-specialists. METHODS: We used a nationally representative sample of Medicare beneficiaries with diabetes who used electrodiagnostic services in 2006 to examine whether specialists and non-specialists were different in the rates of identifying common neuromuscular conditions. RESULTS: Specialists (neurologists and physiatrists) performed 62% of electrodiagnostic consultations; non-specialist physicians and non-physicians performed 31% and 5%, respectively. After adjusting for age, race/ethnicity, diabetes severity, and comorbidities, specialists were 1.26-9 times more likely than non-physicians to diagnose polyneuropathy, lumbosacral radiculopathy, cervical radiculopathy, carpal tunnel syndrome, and ulnar neuropathy. Almost 80% of electrodiagnostic studies performed by specialists included electromyography testing; fewer than 13% by non-specialists did. CONCLUSIONS: Inadequate use of electromyography and fewer specific diagnoses suggest that many non-specialists perform insufficiently comprehensive electrodiagnostic studies.

Botulinum toxin treatment in neurological practice: how much does it really cost? A prospective cost-effectiveness study.

Botulinum toxin (BTA) is a safe and effective therapeutic tool for many neurological conditions but few studies have investigated its real cost in neurological practice. We evaluated the daily cost of botulinum toxin type A (BTA) treatment through a cost effectiveness analysis alongside a prospective study of BTA injections at a French University Hospital over a 2-year follow-up period. The data of 3,108 BTA injections performed in 870 adult patients presenting with dystonia, facial hemispasm, or spasticity were entered in the database. Patients were questioned at each visit about the subjective effectiveness of the previous injection. The daily cost of BTA treatment was calculated as the ratio of each session's cost (including all additional costs) to the duration of subjective efficacy. The subjective rating of efficacy duration was 17.3 ± 7.7 weeks for facial hemispasm, 15.4 ± 7.7 for blepharospasm, 14.3 ± 6.7 for cervical dystonia, 14.5 ± 7.8 and 14.1 ± 7.4 weeks for upper and lower limb spasticity, respectively. The daily cost of BTA injections was 0.57 ± 0.20 for facial hemispasm, 0.95 ± 0.30 for blepharospasm, 2.85 ± 0.86 for cervical dystonia, 3.38 ± 1.49 and 3.62 ± 1.81 for upper and lower limb spasticity, respectively. When associated costs were considered, the daily cost of BTA injections was clearly increased (45-93%) in limb spasticity or rigidity but remained only modestly increased (15-37%) in other indications. These results obtained in a large cohort of patients show that BTA treatment has a low daily cost for a long-lasting effect, with a daily cost/benefit ratio that greatly depends on the indications.

Scoliosis surgery in children with neuromuscular disease: findings from the US National Inpatient Sample, 1997 to 2003.

BACKGROUND: Scoliosis is a frequent complication of pediatric neuromuscular disease (NMD). Scoliosis surgery in children with NMD is thought to carry greater morbidity and mortality. OBJECTIVES: To study demographics, comorbidities, outcomes, and hospitalization expenditures among children with NMD undergoing scoliosis surgery. DESIGN: Using the Kids Inpatient Database, a large all-payer US database of hospital discharges among children and adults younger than 20 years, we studied children undergoing scoliosis surgery between January 1, 1997, and December 31, 2003. Continuous variables were compared by t test, and categorical variables were compared by Pearson product moment correlation chi(2) test. SETTING: National database of pediatric hospital discharges. PATIENTS: Children with and without NMD. MAIN OUTCOME MEASURES: Demographics, hospital length of stay, and in-hospital mortality associated with scoliosis surgery. RESULTS: Of 17 780 reported hospitalizations owing to scoliosis surgery, 437 children (2.5%) had NMD. Compared with children undergoing scoliosis surgery for other indications, children with NMD were more likely to be younger (12.4 vs 14.2 years), male (73.5% vs 38.3%), and insured by Medicaid (35.6% vs 20.3%). Comorbidities that were more common among children with NMD included pulmonary complications (lung disease not classified, pulmonary collapse, pulmonary insufficiency, chronic respiratory failure, and ventilator requirement) and cardiovascular complications (cardiomyopathy, hypotension, and tachycardia). Scoliosis surgery in children with NMD was associated with increased hospital length of stay (10.3 vs 7.7 days) and hospitalization expenditures ($80 251 vs $62 154), and higher in-hospital mortality (1.6% vs 0.2%). CONCLUSION: Children with NMD have increased hospital length of stay and higher in-hospital mortality associated with scoliosis surgery, highlighting the need for further study of measures that could reduce complications and improve outcomes in this population.

The Teletón Centers Of Child Rehabilitation Mexico: 'El amor y la ciencia al servicio del hombre'.

This paper describes the foundation and distribution of the Teleton and Oritel networks of rehabilitation. An essential component of the Pan-American network, as reported by Dr. Jorge Hernandez Franco, Teleton follows a humanistic perspective in offering a comprehensive biopsychosocial model of therapy and care. Public engagement is central to the success of Teleton and highlights the importance of increasing public understanding of rehabilitation needs and service delivery.