PEDIATRICS Vol. 123 No. 1 January 2009, pp. 407-412 (doi:10.1542/peds.2007-2875)
SPECIAL ARTICLE |
Models of Comprehensive Multidisciplinary Care for Individuals in the United States With Genetic Disorders
a National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, Georgia
b Department of Pediatrics, Emory University, Atlanta, Georgia
c Maternal and Child Health Bureau, Health Resources and Services Administration, Rockville, Maryland
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ABSTRACT |
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 TOP ABSTRACT MODELS OF COMPREHENSIVE CAREDISORDER-SPECIFIC TREATMENT...CONCLUSIONSREFERENCES |
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Approaches to providing comprehensive coordinated care for individuals with complex diseases include the medical home approach, the chronic care model in primary care, and disease-specific, multidisciplinary specialty clinics. There is uneven availability and utilization of multidisciplinary specialty clinics for different genetic diseases. For 2 disorders (ie, hemophilia and cystic fibrosis), effective national networks of specialty clinics exist and reach large proportions of the target populations. For other disorders, notably, sickle cell disease, fewer such centers are available, centers are less likely to be networked, and centers are used less widely. Models of comanagement are essential for promoting ongoing communication and coordination between primary care and subspecialty services, particularly during the transition from pediatric care to adult care. Evaluation of the effectiveness of different models in improving outcomes for individuals with genetic diseases is essential.
Key Words: genetic services • care coordination • health care disparities • health care utilization
Abbreviations: CDC—Centers for Disease Control and Prevention • CF—cystic fibrosis • HRSA—Health Resources and Services Administration • HTC—hemophilia diagnostic and treatment center • SCD—sickle cell disease
The modern health care delivery system evolved in response to the need to deliver acute episodic care for infectious diseases, and it was not designed to provide ongoing care for complex chronic illnesses.1,2 Recent attempts to provide comprehensive coordinated care more effectively for individuals with complex diseases have introduced the medical home concept,3 the chronic care model in primary care,4 and the disease-specific, multidisciplinary, specialty clinic.5 Models of comanagement are essential to promote ongoing communication and coordination between primary care and subspecialty services. It would be inefficient for subspecialists to provide primary care and ineffective for primary care providers to attempt to stay abreast of the latest therapies for rare diseases.
Both access to specialty care and effective comanagement by primary care and subspecialty providers are limited for genetic and other chronic diseases. For example, a study of children enrolled in Medicaid programs in 4 states in 1989–1992 found that only 36% of those with sickle cell disease (SCD) and 44% of those with hemophilia had 
1 visit with a subspecialist in a given year.6 A similar analysis of the National Health Interview Survey found that <30% of children with special health care needs saw a specialist or subspecialist in a year, regardless of whether they had public or private insurance.7
This article reviews different models of multidisciplinary care provided to individuals diagnosed as having 3 specific genetic disorders, namely, hemophilia, cystic fibrosis (CF), and SCD. The first 2 disorders have extensive networks of comprehensive specialty care centers, whereas no comparable nationwide network of specialized comprehensive treatment centers exists for SCD.
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MODELS OF COMPREHENSIVE CARE |
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TOPABSTRACTMODELS OF COMPREHENSIVE CAREDISORDER-SPECIFIC TREATMENT...CONCLUSIONSREFERENCES |
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The existence of fragmented health care for children with special health care needs led to the development of the medical home concept among pediatricians, beginning in the 1970s.3 In 1992, the American Academy of Pediatrics adopted the recommendation that all children should have a medical home, defined as care that is "accessible, continuous, comprehensive, family centered, coordinated, and compassionate."3 The American Academy of Pediatrics, with support from the Health Resources and Services Administration (HRSA) and other organizations, notably the American Academy of Family Physicians and the American College of Physicians, has continued to develop and to popularize the medical home concept, which is no longer restricted to pediatrics.3,8 As the usual place for both sick and well care, the medical home should be family-centered, culturally effective, accessible, and actively engaged in the coordination and provision of primary and subspecialty health care services within the health care system and across other community-based agencies and services (eg, other clinicians, educational programs, and community-based counseling and support services). A recent survey found that, when adults have health insurance coverage and a medical home (defined as a health care setting that provides patients with timely, well-organized care and enhanced access to providers), racial and ethnic disparities in health care access and quality are reduced or even eliminated.9
The chronic care model, developed with support from the Robert Wood Johnson Foundation, has emerged as an evidence-based mechanism to improve the management of chronic illnesses, such as type 2 diabetes mellitus, within the primary care setting.2,4,10 The model was developed for adults with chronic diseases. Pediatric care providers have expanded the chronic care model to include child health, integrating the medical home concept into that model. Both the chronic care model and the medical home concept involve a multidisciplinary practice team that provides coordination of care based on planned visits and follow-up telephone and electronic contacts that focus on monitoring disease markers, delivering preventive services, and coaching patients and family members in disease self-management. The model has been adapted by the Institute for Healthcare Improvement and the National Initiative for Children's Healthcare Quality, with support from the HRSA, as a model for provision of health services in the medical home. Both the medical home concept and the chronic care model emphasize the need for accessible, ongoing, comprehensive primary care, coordinated and comanaged with specialty care.
One of the central elements of the medical home concept and the chronic care model is coordination of care. Coordination implies that the various aspects of comprehensive health care and related services are integrated, to promote their delivery as a unified whole.11 Individuals with complex chronic conditions can receive care through primary care practices in their community or through a clinic providing subspecialty services; in either case, effective coordination among providers is essential. Who provides the coordination of care for individuals with complex diseases requiring multiple subspecialties is an open question.12 On one hand, the primary care professional, with expertise in the provision of preventive care, as well as management of common chronic conditions, might be better able to integrate the comprehensive needs of the patient and the family. On the other hand, the primary care professional is less likely to see individuals with rare conditions on a regular basis and might lack training and expertise in the management of these disorders.
Within medical genetics, there is widespread support for specialized clinics that treat individuals with specific diseases or groups of diseases (eg, metabolic diseases). The goal of such clinics is to provide comprehensive, patient-centered care that brings together multiple medical specialties, as well as disciplines such as nursing, nutrition, and social work.5,13 Furthermore, genetic counseling, reproductive counseling, and prenatal diagnosis are thought to benefit both family members and affected individuals. In addition to disease-specific centers, multidisciplinary clinics could, in principle, treat individuals with a variety of complex disorders.14
Models of comprehensive care for genetic diseases often include specialized, disease-specific centers or clinics that provide multidisciplinary, family-centered care. Individuals with disorders that affect multiple organ systems have varied needs that require teams from multiple medical specialties and other disciplines, including nursing, nutrition, and social work. Coordination of care from different providers, case management services to monitor preventive practices, integration or coordination with early intervention and special education services, and social work or legal assistance with issues relating to health insurance, disability benefits, and social services also may be important components of comprehensive care.
Specialty treatment centers may take different forms. Certain centers provide dedicated facilities with core staff members available on a daily basis. Others offer periodic clinics that bring together a team of different staff members on a regular intermittent schedule (eg, weekly or monthly). Comprehensive multidisciplinary care should not be equated with one particular modality of service delivery. Regardless of the model for accessing and providing subspecialty services, it is important to remember that specialty centers or clinics focus on the provision of regular (eg, quarterly or annual), disease-focused, surveillance visits, specialized care, and coordination of care and psychosocial needs specific to the single-gene condition. These centers generally do not take the place of a primary care provider serving as a medical home for routine preventive services, such as immunizations and screenings, and any provision of acute care services is focused on expected complications of the disorder. In addition, the centers can provide assistance via telephone for medical emergencies specific to the condition.
Experts have attributed the low availability or use of comprehensive specialty care for genetic and other chronic conditions to a variety of factors, including restrictions by health plans on full reimbursement for care by providers outside the plan's network, lack of specific billing codes for care coordination and services provided by nonmedical staff members, capitated reimbursements, and, perhaps most critically, limitations on the number of services that can be billed for a single visit or day.15,16 In particular, although a child might be seen by multiple subspecialists during a clinic visit, insurers typically refuse to pay for >1 physician's time. It is unclear whether referral requirements constitute a barrier to access.7 Nonfinancial barriers include the distance to specialty clinics that individuals must travel.17
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DISORDER-SPECIFIC TREATMENT CENTERS |
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TOPABSTRACTMODELS OF COMPREHENSIVE CAREDISORDER-SPECIFIC TREATMENT...CONCLUSIONSREFERENCES |
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Hemophilia
Multidisciplinary outpatient centers for patients with hemophilia that provide medical and psychosocial support to patients and their families and develop individual management plans to minimize complications were first established in Britain in the middle 1950s.18 In 1970, the availability of a concentrated form of clotting factor (cryoprecipitate) allowed hemophilia centers to promote home infusion or self-therapy, which was reported to reduce hospitalization rates markedly among patients with hemophilia.19
During the 1970s, several states and the National Hemophilia Foundation worked with the HRSA to establish a US network of hospital-based hemophilia diagnostic and treatment centers (HTCs), to provide comprehensive services for patients and families within 1 treatment facility.18,20 In 1975, the US Congress appropriated funds for the HRSA to establish a network of 22 HTCs, each including a coagulation laboratory, a blood bank, and a multidisciplinary hemophilia treatment team; states funded an additional 21 HTCs.18,21 The promotion of home infusion therapy by these HTCs was reported to be associated with fewer hospital and emergency department visits.5
In the 1980s, most patients with hemophilia became infected with HIV and hepatitis through blood products. In 1985, the Centers for Disease Control and Prevention (CDC) began funding HTCs for AIDS risk reduction through the HRSA; in 1995, this was expanded through direct funding from the CDC to the HTCs to provide ongoing surveillance for blood product safety and a program to prevent complications in this population. An evaluation using population-based surveillance data showed that, in 1993–1995, HTCs were successful in reducing both mortality and hospitalization rates among patients with hemophilia who attended HTCs.22,23 The Universal Data Collection project initiated by the HTCs and the CDC in 1998 to measure and improve the health of people with bleeding disorders18 has demonstrated that obesity is a major risk factor for limitation of joint range of motion in individuals with hemophilia.24
Currently, >130 HTCs receive federal funding through the HRSA and the CDC to provide comprehensive care and preventive services to >15000 individuals in the United States with hemophilia and to >10000 patients with other bleeding disorders.5 HTC core team members include a medical director (often an adult or pediatric hematologist) with extensive training and experience in the care of people with bleeding disorders, a nurse coordinator, a psychosocial professional, and a physical therapist. Extended team members can include dentists, orthopedists, and genetic counselors. Field clinics and telemedicine services provided by HTC staff members can extend comprehensive services to individuals in remote areas. The National Hemophilia Foundation Medical and Scientific Advisory Council issued care recommendations for the HTCs in the United States.25 More recently, federally funded HTCs became eligible to participate in the 340B drug pricing program, which decreases the price of clotting factor for consumers and increases the financial sustainability of the HTCs. In addition to an ongoing HTC hemophilia data reporting system (focused on clinical services) supported by the HRSA, the Universal Data Collection project, which has shown the importance of monitoring complications in male patients 2 years of age,24 has been expanded to include special populations such as infants (0–2 years of age) and female patients (all ages) with bleeding disorders.26
Hemophilia management today focuses increasingly on minimizing hemorrhaging into joints, the gastrointestinal tract, and the brain, preventing degenerative joint arthropathy, and improving health-related quality of life.18 Researchers are evaluating the optimal protocols for on-demand or prophylactic clotting factor replacement therapy, in terms of resource costs, morbidity, and health-related quality of life.27 With fewer complications, patients with hemophilia require fewer HTC visits, and decreasing demand for services has led to the attrition of specialized physicians.13
Cystic Fibrosis
Recommendations in the United States and Europe state that children and adults with CF should receive regular care from specialty CF centers.28,29 In the United States, a network of >115 comprehensive, multidisciplinary, CF care centers accredited and funded by the CF Foundation provide care for a large majority of individuals with diagnosed CF, especially children. CF care center teams include physicians, nurses, nutritionists, respiratory therapists, social workers, genetic counselors, and other medical professionals. The CF Foundation, using expert panels of physicians and scientists, publishes consensus guidelines that call for quarterly visits, with at least annual testing to monitor disease severity and complications.28
The CF Foundation maintains a national registry of patients attending accredited CF care centers, based on data submitted by center personnel.30 An industry-sponsored patient registry also includes a small number of patients who do not attend accredited CF centers.31 Registry data can be used to compare health outcomes among patients with CF across centers and to correlate outcomes with the type of care provided. Analyses have reported substantial differences among centers in survival rates and other end points that seem to be associated with differences in the quality of care provided.32 Specifically, centers in the top quartile for patients' median pulmonary function monitored their patients with more-frequent visits, airway cultures, and pulmonary function tests.33 Furthermore, CF centers with better pulmonary function hospitalized their patients more and used more orally and intravenously administered antibiotics.33 A recent study that examined the care provided during infancy at centers whose 6- to 12-year-old children had superior lung function, compared with children with CF in the rest of the country, found that those centers had more-frequent monitoring visits, performed more airway cultures, and treated patients more aggressively with antibiotics during infancy than did comparison centers.34
Sickle Cell Disease
It is generally agreed that optimal treatment of individuals with SCD requires access to comprehensive multidisciplinary care.35–38 The comprehensive care team should include a physician familiar with the multiple complications and presentations of SCD, 1 "physician extender" (such as a physician assistant or nurse practitioner), a health educator, and a medical social worker and should have access to laboratory services, radiology services, and a 24-hour blood bank.36 A knowledgeable primary care provider can coordinate care within the medical home,35 but this requires expertise, professional support, and time. Others recommend that the primary care medical home collaborate with SCD centers or subspecialists.39
Although SCD is more common than CF or hemophilia in the United States, only a minority of affected individuals in this country are seen at specialized SCD centers.40 Although newborn screening and comprehensive care have made it possible for the majority of patients to survive into adulthood, many adult patients lack access to adult specialists and continue to have inadequately satisfied or unmet needs. US managed-care plans often require individuals to be seen by primary care providers, regardless of expertise, and are said to impose barriers to timely access to specialists for acute SCD care.15,16
Adequate care for individuals with SCD requires that they receive both specialized services and comprehensive primary care. The fact that only a minority of affected individuals receive specialist care6 points to the need not only to improve access to specialty services but also to improve coordination of comprehensive care provided in community primary care settings, including private practices, clinics, and community health centers. The HRSA Bureau of Primary Health Care has adopted the chronic care model as a framework for improving chronic care management in community health centers for a number of chronic conditions. This primary care, quality improvement model has the potential to improve coordinated care for individuals with SCD.
Since 1972, a network currently including 10 SCD comprehensive clinical and research centers has received funding from the National Institutes of Health. A primary focus of the National Institutes of Health funding is to support the infrastructure for enrolling patients with SCD in clinical research studies. It is unclear to what extent these centers provide family-centered care comparable to that provided by HTCs or CF centers. Legislation passed in 2004, that is, the Sickle Cell Disease Treatment Act, authorized the HRSA to fund up to 40 community-based networks of care to link primary and subspecialty care to provide coordinated comprehensive care for individuals with SCD.39,40 The law received appropriations in 2006 ($2000000), and the HRSA funded 4 local SCD networks and 1 national coordinating center in late 2006, to enhance SCD care through coordination of service delivery, genetic counseling and testing, bundling of technical services, and training of health care professionals. The primary goal of these networks is to ensure that all individuals served have access to a medical home, with coordination and continuity of education, treatment, and care.
Among the major challenges in SCD care is the management of acute pain crises, most commonly associated with vasoocclusive events and acute chest syndrome.38 Effective pain management in SCD incorporates pharmacologic, behavioral, and physical pain management strategies.41 New technologies and treatments, such as hydroxyurea treatment, chronic transfusions with oral chelation therapy to prevent iron overload, and bone marrow transplantation, offer promise for the prevention of painful crises, although evidence on long-term outcomes is still needed, as well as data on which groups are most likely to benefit.42 New practices typically require specialized clinical experience and technical expertise; therefore, they are rarely used except in the context of comprehensive care centers.43 An alternative to separate outpatient pain management facilities is the provision of multidisciplinary teams within emergency departments or hospitals.44,45
Although experts think that children with SCD experience lower morbidity and mortality rates if they are seen by providers who offer comprehensive coordinated care,46 the lack of active SCD surveillance of long-term health outcomes makes it difficult to assess quality of care or outcomes.40 Black patients with SCD who live in remote or rural areas are likely to access SCD centers for acute care but not comprehensive care.17,47 In Alabama, the Children and Youth Sickle Network extended services to rural areas beginning in 1995, which resulted in sharp reductions in the average age at the first visit to a sickle cell clinic and the rate of death resulting from sepsis.48 This network is part of the newly funded HRSA SCD networks. It is expected that the HRSA-funded networks will establish a surveillance system to monitor long-term health outcomes within the 4 networks. In addition, the establishment of the networks affords the possibility of evaluating treatment protocols.
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CONCLUSIONS |
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TOPABSTRACTMODELS OF COMPREHENSIVE CAREDISORDER-SPECIFIC TREATMENT...CONCLUSIONSREFERENCES |
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Two different approaches to the provision of care for multisystem genetic diseases have been implemented in the United States. One approach makes disease-specific, center-based, comprehensive, multidisciplinary care responsible for providing all or most disease-specific care and coordinating such care. This approach requires the availability and accessibility of specialized treatment centers throughout the United States for individuals of all ages, which is true for both CF and hemophilia. Both CF centers and HTCs have established patient registries to monitor outcomes and to evaluate the effectiveness of treatment protocols, which have been invaluable for demonstrating improvements with high-quality comprehensive care provided by specialized centers. The other approach relies on primary care providers as the dominant source of care, with subspecialists available for referral. This approach characterizes care for individuals with SCD. A small number of comprehensive SCD centers with a research orientation exist, as well as an even smaller network of SCD and community health centers funded by the HRSA for the delivery of comprehensive care. Most individuals with SCD, particularly adults, do not use subspecialty or multidisciplinary care. In the absence of either registries or population-based surveillance, little is known about the clinical outcomes of individuals with SCD or how outcomes differ depending on the type or model of care provided.
Comprehensive care can be difficult to pay for through individual health insurance, as discussed above. HTCs and CF centers have institutional sources of funding that are not available for SCD care. The HRSA Children with Special Health Care Needs program provides funding to states to provide, to reimburse, or to coordinate medical care for children with specified chronic health conditions, with upper age cutoff values set by states at 18 to 21 years. In addition, 17 states provide their own funds to help pay for medical care for adults with certain genetic conditions, including CF (12 states), hemophilia (8 states), and SCD (2 states).
Whether comprehensive care provided by multidisciplinary teams, either in disease-specific centers or through coordination of care by primary health care providers, improves measures of quality of care and health outcomes is an important issue that requires careful study. Although individuals with hemophilia who use HTCs are reported to have better outcomes,22,23 few studies have compared outcomes for groups defined on the basis of access to center-based care. One study found no difference in SCD mortality rates in Tennessee in relation to geographic access to SCD center care.49 It cannot be assumed that either specialty clinic staff members or primary care providers deliver evidence-based, patient-oriented care. It is important to measure the extent to which evidence-based interventions are prescribed in a standardized manner. Data from CF centers demonstrated that, if approaches used at the best-performing centers were adopted universally, then dramatic improvements in health outcomes could be achieved.31 In addition, it should not be assumed that specialty care is sufficient to optimize health and well-being for individuals with complex chronic conditions. Primary preventive care and acute care are both essential components of comprehensive health care.
To ensure that comprehensive care is provided to individuals throughout their lives, it is essential that the transition from pediatric care to adult care be planned with the involvement of adult care providers. Traditionally, care has been provided by pediatric subspecialists, because the greatest opportunities for lifelong prevention of secondary disabilities are among children with early diagnoses of these disorders. However, successful prevention efforts have resulted in most individuals with CF, hemophilia, and SCD now surviving to adulthood. As individuals transition into adulthood, it is essential to bridge pediatric or adolescent care and adult care. HTCs and CF centers routinely provide care to both children and adults, easing the transition of care. Providers of adult primary and specialty care for individuals with SCD need to be as informed and involved in the coordination of care as are pediatricians or primary subspecialists.
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ACKNOWLEDGMENTS |
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We appreciate helpful comments received from Ronald Bachman, Judith Hagopian, Muin Khoury, Esther Sumartojo, Marc Williams, and Paula Yoon.
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FOOTNOTES |
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Accepted Apr 24, 2008.
Address correspondence to Edwin Trevathan, MD, MPH, National Center on Birth Defects and Developmental Disabilities, CDC, Mail Stop E-87, 1600 Clifton Rd, NE, Atlanta, GA 30333. E-mail: net1{at}cdc.gov
The findings and conclusions in this report are those of the authors and do not necessarily represent the official positions of the Centers for Disease Control and Prevention, the Health Resources and Services Administration, or the US Department of Health and Human Services.
The authors have indicated they have no financial relationships relevant to this article to disclose.
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  D. G. Bundy, J. J. Strouse, J. F. Casella, and M. R. Miller Burden of Influenza-Related Hospitalizations Among Children With Sickle Cell Disease Pediatrics, February 1, 2010; 125(2): 234 - 243. [Abstract] [Full Text] [PDF]
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