Reimbursement For Treatment Research Articles

We Are on the Verge of Breakthrough Cures for Type 1 Diabetes, but Who Are the 2 Million Americans Who Have It?

Background: Two million Americans have type 1 diabetes (T1DM). Innovative treatments have standardized insulin delivery and improved outcomes for patients, but patients' access to such technologies depends on social determinants of health, including insurance coverage, proper diagnosis, and appropriate patient supports. Prior estimates of US prevalence, incidence, and patient characteristics have relied on data from select regions and younger ages and miss important determinants. Objectives: This study sought to use large, nationally representative healthcare claims data sets to holistically estimate the size of the current US population with T1DM and investigate geographic nuances in prevalence and incidence, patient demographics, insurance coverage, and device use. This work also aimed to project T1DM population growth over the next 10 years. Methods: We used administrative claims from 4 sources to identify prevalent and incident T1DM patients in the US, as well as various demographic and insurance characteristics of the patient population. We combined this data with information from national population growth projections and literature to construct an actuarial model to project growth of the T1DM population based on current trends and scenarios for 2024, 2029, and 2033. Results: We estimated 2.07 million T1DM patients nationally across all insurance coverages in our 2024 baseline model year: 1.79 million adults (≥20 years) and 0.28 million children. This represents a US T1DM prevalence rate of 617 per 100 000 and an incidence rate of 0.016%. By 2033, we project the US population with T1DM will grow by about 10%, reaching approximately 2.29 million patients. Discussion: Our results showed important differences in T1DM prevalence and incidence across regions, payers, and ethnic groups. This suggests studies based on more geographically concentrated data may miss important variation in prevalence and incidence across regions. It also indicates T1DM prevalence tends to vary by income, consistent with several international studies. Conclusions: Accurate projections of T1DM population growth are critical to ensure appropriate healthcare coverage and reimbursement for treatments. Our work supports future policy and research efforts with 2024, 2029, and 2033 projections of demographics and insurance coverage for people with T1DM.

We Are on the Verge of Breakthrough Cures for Type 1 Diabetes, but Who Are the 2 Million Americans Who Have It?

Background: Two million Americans have type 1 diabetes (T1DM). Innovative treatments have standardized insulin delivery and improved outcomes for patients, but patients’ access to such technologies depends on social determinants of health, including insurance coverage, proper diagnosis, and appropriate patient supports. Prior estimates of US prevalence, incidence, and patient characteristics have relied on data from select regions and younger ages and miss important determinants. Objectives: This study sought to use large, nationally representative healthcare claims data sets to holistically estimate the size of the current US population with T1DM and investigate geographic nuances in prevalence and incidence, patient demographics, insurance coverage, and device use. This work also aimed to project T1DM population growth over the next 10 years. Methods: We used administrative claims from 4 sources to identify prevalent and incident T1DM patients in the US, as well as various demographic and insurance characteristics of the patient population. We combined this data with information from national population growth projections and literature to construct an actuarial model to project growth of the T1DM population based on current trends and scenarios for 2024, 2029, and 2033. Results: We estimated 2.07 million T1DM patients nationally across all insurance coverages in our 2024 baseline model year: 1.79 million adults (≥20 years) and 0.28 million children. This represents a US T1DM prevalence rate of 617 per 100 000 and an incidence rate of 0.016%. By 2033, we project the US population with T1DM will grow by about 10%, reaching approximately 2.29 million patients. Discussion: Our results showed important differences in T1DM prevalence and incidence across regions, payers, and ethnic groups. This suggests studies based on more geographically concentrated data may miss important variation in prevalence and incidence across regions. It also indicates T1DM prevalence tends to vary by income, consistent with several international studies. Conclusions: Accurate projections of T1DM population growth are critical to ensure appropriate healthcare coverage and reimbursement for treatments. Our work supports future policy and research efforts with 2024, 2029, and 2033 projections of demographics and insurance coverage for people with T1DM.

An Update on Access to Novel Treatment for Metastatic Melanoma in Europe — A 2024 Survey of the European Melanoma Registry and the European Association of Dermato-Oncology

Advances in cancer treatments have significantly improved their effectiveness, yet access to first-line therapies remains limited. A 2017 survey revealed that over 25% of metastatic melanoma patients in Europe lacked access to recommended therapies. To address this, the European Association of Dermato-Oncology and the European Melanoma Registry conducted a follow-up study on the registration and reimbursement of first-line treatments.A web-based survey using LimeSurvey was distributed to melanoma experts across 27 European countries from February to April 2022 and updated from February to April 2024. The questionnaire covered the percentage of patients receiving recommended treatments, as well as treatment authorization and reimbursement dates for systemic and adjuvant therapies.There has been significant improvement in the registration and reimbursement of BRAFi/MEKi, anti-PD1, and anti-PD1/anti-CTLA4 therapies, increasing from 48%, 63%, and 37% in 2017 to 96%, 96%, and 78% in 2024, respectively. Despite these gains, restrictions persist. Anti-PD1/anti-CTLA4 combination immunotherapy is still not available without restrictions in 48% of the surveyed countries. The nivolumab/relatlimab combination is licensed only for PDL-1-negative melanoma and reimbursed in seven countries of Europe. Tebentafusp is reimbursed in 12 countries and talimogene laherpervec in 6. In 2024, adjuvant treatments for stage III melanoma are reimbursed in 22 countries for dabrafenib/trametinib and 24 of 27 for anti-PD1 antibodies. Pembrolizumab and nivolumab are reimbursed in 16 and 8 countries, respectively, for stage IIB/IIC disease.While there have been improvements in the reimbursement of metastatic melanoma treatments in Europe, challenges and discrepancies remain. Further efforts at European and global levels are needed to harmonize and enhance access to cancer medicines.

Alignment, Anticipation, Adaptation, or Lagging Behind? Age-Based Regulations in Assisted Reproduction and Late Fertility.

This paper focuses on age restrictions on access to infertility treatments and eligibility for their public reimbursement, exploring their relevancy in contexts of rising late birth rates (40+). I explore how age-based reimbursement policies for in vitro fertilization treatments have responded to these fertility trends in 27 high-income countries and in which regulatory frameworks for medically assisted reproduction (MAR) very late births (45+) have particularly increased. First, I show that while age limits for treatment reimbursement are well aligned with the prevalence of late fertility in some national contexts, in most countries, strict age restrictions are lagging behind the rise in late births. In others, pronatalist policies have prompted permissive age criteria or law revisions, anticipating or adapting to rising trends in late births. Second, the rise in very late births has been limited in some contexts with strict age-based rules. However, the analysis suggests that the impact of MAR on very late births may also be influenced by contextual factors other than regulations.

11th revision of the International Classification of Diseases chronic primary pain diagnoses in children and adolescents: representation of pediatric patients in the new classification system.

Chronic pain is common among children and adolescents; however, the diagnoses in the newly developed 11th revision of the International Classification of Diseases (ICD-11) chronic pain chapter are based on adult criteria, overlooking pediatric neurodevelopmental differences. The chronic pain diagnoses have demonstrated good clinical applicability in adults, but to date, no field study has examined these diagnoses to the most specific diagnostic level in a pediatric sample. The current study aimed to explore pediatric representation within the ICD-11, with focus on chronic primary pain. Healthcare professionals (HCPs) at a specialized pediatric pain center documented the symptoms of and assigned both ICD-10 and ICD-11 diagnoses to N = 402 patients. Using criteria-based computer algorithms, specific ICD-11 pain diagnoses were allocated for each documented pain location, with residual diagnoses (ie, "unspecified") assigned if criteria were not (fully) met. Within the ICD-11, the algorithms assigned specific pain diagnoses to most patients (73.6%). In ICD-10, HCPs could not specify a diagnosis for 5.2% of patients; the ICD-11 algorithm allocated a residual chronic primary pain diagnosis in 51.2%. Residual categories were especially prevalent among younger children, boys, patients with headaches, and those with lower pain severity. Overall, clinical utility of the ICD-11 was high, although less effective for chronic back pain and headache diagnoses. The latter also exhibited the lowest agreement between HCPs and algorithm. The current study underscores the need for evidence-based improvements to the ICD-11 diagnostic criteria in pediatrics. Developing pediatric coding notes could improve the visibility of patients internationally and improve the likelihood of receiving reimbursement for necessary treatments through accurate coding.

Development of an multicriteria decision analysis framework for rare disease reimbursement prioritization in Malaysia.

Rare diseases (RD)-related policies have received significant attention due to the pressing medical requirements associated with these medical conditions and the substantial impact and treatments they may have on healthcare budgets. Nevertheless, policymakers frequently encounter difficulties in managing issues concerning resource allocation and prioritization within this population. Realizing the need to address such problems, this study was conducted to develop a framework based on the multicriteria decision analysis to improve RD reimbursement prioritization in Malaysia. Primarily, a scoping review was performed to identify the methods and criteria used for the reimbursement of RD treatment, followed by strategic stakeholder engagement and a deliberative process on determining the best approach for the framework, including criteria identification, elicitation of weights, and a pilot assessment using the framework. The findings reflected the priorities and perspectives of the stakeholders, which identified eight key criteria and their associated weights, namely effectiveness (19.6 percent), disease severity (15.6 percent), safety (14.2 percent), access to treatment (12.6 percent), economic consideration (12.2 percent), type of therapeutic treatment (11.5 percent), availability of alternatives (8.3 percent), and population group (6 percent). In summary, the developed framework was well-accepted by the Rare Disease Committee, which will be applied as part of the committee deliberation for transparent and equitable decision making on fund allocation and reimbursement of orphan and RD treatment in Malaysia.

A Review of Current Approaches to Pain Management in Knee Osteoarthritis with a Focus on Italian Clinical Landscape.

The global cases of knee osteoarthritis (KOA) are projected to increase by 74.9% by 2050. Currently, over half of patients remain dissatisfied with their pain relief. This review addresses unmet needs for moderate-to-severe KOA pain; it offers evidence and insights for improved management. Italian experts from the fields of rheumatology, physical medicine and rehabilitation, orthopedics, primary care, and pain therapy have identified several key issues. They emphasized the need for standardized care protocols to address inconsistencies in patient management across different specialties. Early diagnosis is crucial, as cartilage responds better to early protective and structural therapies. Faster access to physiatrist evaluation and reimbursement for physical, rehabilitative, and pharmacological treatments, including intra-articular (IA) therapy, could reduce access disparities. Concerns surround the adverse effects of oral pharmacological treatments, highlighting the need for safer alternatives. Patient satisfaction with corticosteroids and hyaluronic acid-based IA therapies reduces over time and there is no consensus on the optimal IA therapy protocol. Surgery should be reserved for severe symptoms and radiographic KOA evidence, as chronic pain post-surgery poses significant societal and economic burdens. The experts advocate for a multidisciplinary approach, promoting interaction and collaboration between specialists and general practitioners, to enhance KOA care and treatment consistency in Italy.

Quality of Life and Burden of Moderate-to-Severe Atopic Dermatitis in Adult Patients Within the Asia-Pacific Region: A Cross-sectional Survey.

The burden of atopic dermatitis (AD) is significant, with a substantial impact on quality of life (QoL). This cross-sectional study aimed to ascertain the burden of AD, its impact on QoL, and associated costs. Patients with moderate-to-severe AD were enrolled from eight territories, namely Hong Kong, India, Japan, Mainland China, Singapore, South Korea, Taiwan, and Thailand. After screening was performed and informed consent was obtained, eligible participants were asked to provide responses on their AD symptoms, severity, treatment, and out-of-pocket costs via an online survey. QoL was assessed using EQ-5D-5L and Dermatology Life Quality Index (DLQI), while productivity loss was quantified using the Work Productivity and Activity Impairment (WPAI) questionnaire. Data from completed submissions were analyzed using descriptive statistics. The study was reviewed by the institutional review board in each territory. Median age of enrolled patients (N = 1103) was 41.0years (interquartile range, IQR 16.0). The majority of patients reported that their head/neck, trunk, upper limbs, and lower limbs were affected during a flare. Topical (74.2%) and oral steroids (58.7%) were frequently prescribed to manage AD. Common atopic comorbidities were allergic urticaria (64.2%), allergic rhinitis (61.8%), and allergic conjunctivitis (51.5%). Median DLQI score was 13.0 (IQR11.0), while median EQ-5D-5L (based on China value set) score was 0.8 (IQR0.4); 87.2% and 77.2% of patients reported pain/discomfort and anxiety/depression on the EQ-5D-5L domains, respectively. Median total annual costs associated with AD were USD10,128.52 (IQR12,963.26) per patient, with indirect costs being the largest component. Findings from WPAI indicated that presenteeism is a major contributor to productivity loss. This multinational survey study showed that AD is associated with substantial QoL impairment and economic burden among Asian adult patients with moderate-to-severe AD. To alleviate burden of AD, clinicians should be more proactive in managing other concomitant conditions including psychological issues, and advocate for increased reimbursement for AD treatments.

How to Implement Pressurized Intraperitoneal Aerosol Chemotherapy into a National Health System Scenario: A Single-Center Retrospective Analysis of Costs and Economic Sustainability at a High-Volume Italian Hospital.

PIPAC is a new surgical procedure and a viable treatment option for PSM patients, due to promising therapeutic outcomes, minimal invasiveness, limited surgical morbidity, and systemic toxicity side effects. However, its implementation throughout hospitals is hard to obtain due to its fragile economical sustainability. A retrospective health economic analysis was conducted in order to evaluate the cost of hospitalization for patients undergoing PIPAC treatment at Fondazione Policlinico Universitario Agostino Gemelli, IRCCS, in Rome. The average cost of a PIPAC procedure was defined based on the cost of surgery (cost of surgical material, operating room, intraperitoneal chemotherapy), hospital stay, diagnostic examinations, and drugs used during the stay. A total of 493 PIPAC procedures were performed on 222 patients with peritoneal metastases or primary peritoneal cancer from 2017 to 2023. Since the mean remuneration for each PIPAC hospitalization is €5916 and the mean expenditure per hospitalization is €6538, this results in an operating profit per PIPAC hospitalization of -€622. The reimbursement of PIPAC treatment by the Italian National Health System currently only partially covers the hospital's costs. Development of specific codes and adequate reimbursement for PIPAC by recognizing this procedure as a proper treatment for peritoneal carcinomatosis is essential.

Onco@home: comparing the costs and reimbursement of cancer treatment at home with the standard of care

BackgroundOncological home hospitalization (HH) was implemented in a Belgian context to evaluate the feasibility of oncological HH. In a first HH model (HH1), implemented by three Belgian hospitals, two home nursing organizations and a grouping of independent nurses, the blood draw and monitoring prior to intravenous therapy was performed by a trained home nurse at the patient’s home the day before the visit to the day hospital. In a second HH model (HH2), implemented in one hospital, the administration of two subcutaneous treatments (Azacitidine and Bortezomib) for myelodysplastic syndrome and multiple myeloma were provided at home instead of in the hospital. A previous study on this pilot showed that oncological HH is feasible and safe and improves the Quality of Life. The aim of this study is to investigate the cost and reimbursement of cancer treatment in these two HH models compared to the Standard of Care (SOC).MethodsA bottom-up micro-costing study was conducted to compare the costs and revenues for the providers (hospitals and home care organizations) of the SOC and the HH models.ResultsCosts associated to HH were higher than the SOC in the hospital. Comparing revenues with costs, the research revealed that the reimbursement from the National Health Insurance of HH for oncological patients is insufficient. In HH1, costs were higher than in the SOC (+ €50.4). There was a reduction in costs in the hospital by moving the blood draw to the home setting (-€23.9), but the costs in home care were higher (+ €74.3). The extra revenues in home care (+ €33.6) were insufficient to cover the costs. The cost difference between the SOC and HH2 (+ €9.5 for Azacetidine) was smaller than in HH1. But, there was almost no funding for subcutaneous administration in home care. If the product is administered in a day hospital, the hospital receives a revenue of €124 per administration, while in home care the funding is €5 per visit.ConclusionCosts of HH are higher and the reimbursement from Belgian NHI is insufficient to organize HH. As a result, HH for oncology patient is still limited in Belgium.

Withdrawing versus Withholding Treatments in Medical Reimbursement Decisions: A Study on Public Attitudes.

The use of policies in medical treatment reimbursement decisions, in which only future patients are affected, prompts a moral dilemma: is there an ethical difference between withdrawing and withholding treatment? Through a preregistered behavioral experiment involving 1,067 participants, we tested variations in public attitudes concerning withdrawing and withholding treatments at both the bedside and policy levels. In line with our first hypothesis, participants were more supportive of rationing decisions presented as withholding treatments compared with withdrawing treatments. Contrary to our second prestated hypothesis, participants were more supportive of decisions to withdraw treatment made at the bedside level compared with similar decisions made at the policy level. Our findings provide behavioral insights that help explain the common use of policies affecting only future patients in medical reimbursement decisions, despite normative concerns of such policies. In addition, our results may have implications for communication strategies when making decisions regarding treatment reimbursement. We explore public' attitudes toward withdrawing and withholding treatments and how the decision level (bedside or policy level) matters.People were more supportive of withholding medical treatment than of withdrawing equivalent treatment.People were more supportive of treatment withdrawal made at the bedside than at the policy level.Our findings help clarify why common-use policies, which impact only future patients in medical reimbursement decision, are implemented despite the normative concerns associted with thesepolicies.

Patients’ Perceptions of Nusinersen Effects According to Their Responder Status

Background and Objective: Patients with spinal muscular atrophy (SMA) treated with a disease-modifying therapy (DMT) are often classified as responders or non-responders based on the attainment of a specific improvement threshold on validated functional scales. This categorization may significantly impact treatment reimbursement in some countries. The aim of this research is to evaluate the perception of treatments and their benefit by patients considered as responders or non-responders. Methods: In this non-commercial multicenter study, 99 post-symptomatically treated SMA type I–III patients with a median age of 11.2 (0.39–57.4) years at treatment initiation were stratified into three groups based on their treatment outcomes, i.e., those exhibiting clinically significant improvement (N = 41), those with non-clinically significant improvement (N = 18), or those showing no improvement (N = 40). Fifteen months after treatment, the initiation patients or patients’ caregivers were assessed using a patient-rated scoring system based on the Patient Global Impression of Change (PGIC) scale, comprising 22 questions targeting important aspects and tasks in the daily life of patients with SMA. Results: We found no statistical difference in the patient perception of treatment benefits in 17 out of 22 domains across patient groups. Conclusions: Our results suggest that functional motor scales do not recapitulate patients’ and patients’ caregivers’ experience of the effect of nusinersen treatment in SMA.

APA theme for annual meeting and current journal: Addiction

Last week's annual meeting of the American Psychiatric Association (APA) featured much more than the usual addiction‐focused programming, which has been growing year by year. It was, under the direction of outgoing president Petros Levounis, M.D., himself an addiction psychiatrist, based on addiction. So is the current issue of the APA's flagship journal, the American Journal of Psychiatry, which featured three major articles on addiction. Two were presented last week; one, a commentary by Keith Humphreys, Ph.D., and colleagues, focused on the barriers to Medicaid reimbursement for treatment, mainly erected by a few states (ADAW will cover that in depth next week).

Cost-Effectiveness of Sleeve Gastrectomy and Gastric Bypass as Revisional Surgery on Antidiabetic Reimbursement: A Nationwide Cohort Study.

This study compared the effectiveness of 4 main revisional bariatric surgery (RBS) sequences after sleeve gastrectomy (SG) and adjustable gastric banding (AGB), on the reimbursement of antidiabetic treatments in France. Few large-scale prospective cohort studies have assessed the changes in antidiabetic treatments after RBS. This nationwide observational population-based cohort study analyzed data from the French National Health Insurance Database. All patients who underwent primary SG and AGB in France between January 2012 and December 2014 were included and followed up until December 31, 2020. The changes in categories and costs of reimbursed antidiabetic treatments across different RBS sequences were assessed (presented as follows: bariatric surgery (BS)-RBS). Among the 107,088 patients who underwent BS, 6396 underwent RBS, 2400 SG-GBP (SG converted to gastric bypass [GBP] during follow-up), 2277 AGB-SG, 1173 AGB-GBP, and 546 SG-SG. Pre-RBS insulin was used in 10 (2.9%), 4 (0.9%), 8 (2.4%), and 10 (2.6%) patients, respectively. Two years after RBS, the treatment discontinuation or decrease (the change of treatment to a lighter one category rates [eg, insulin to bi/tritherapy]) was 47%, 47%, 49%, and 34%, respectively. Four years after RBS, the median annual cost per patient compared with baseline was lower (P < 0.01) for all sequences, except SG-SG (P = 0.24). The most notable effect concerned AGB-GBP (median of more than 220 euros to 0). This study demonstrated the positive impact of RBS over a 4-year follow-up period on antidiabetic treatments reimbursement, through the reduction or discontinuation of treatments and a significant decrease in costs per patient.

An analysis of benign prostatic hyperplasia surgical treatment reimbursement trends across Canada Examining provincial changes over the recent decade with comparison to cost of living changes.

A variety of procedures for the endoscopic surgical treatment of symptomatic benign prostatic hyperplasia (BPH) refractory to medical therapy have existed for decades. The present study examined trends in surgeon compensation for these treatments within Canada. The physician fee schedule for BPH surgery across 10 Canadian provinces for the years 2010 and 2023 were obtained. A descriptive study examined first the provincial reimbursement for transurethral resection of prostate (TURP) and laser ablative/enucleation surgery; second, the difference in TURP reimbursement between 2010 and 2023; and third, the annual change in TURP reimbursement juxtaposed with the annual change in the provincial Consumer Price Index (CPI) and annual salary for the working population aged 35-44. Seven of 10 Canadian provinces reimburse laser BPH surgery equally to TURP. The average provincial TURP reimbursement is $545, ranging from $451 in Ontario to $688 in Saskatchewan. Since 2010, TURP reimbursement has varied by province from a 0% net change in Ontario to an increase of 21% in Nova Scotia. Reimbursement for TURP has increased at a slower pace than the local CPI, and for half of the provinces at a slower pace than the annual salary for people aged 35-44. The compensation model for endoscopic BPH surgery does not have a unified structure in Canada that is consistent across provinces, nor does it keep up with inflation, possibly impacting future recruitment, increasing geographic disparities, and most importantly, limiting the adoption of new BPH therapies.

Medical Care For People In Precarious Living Situations In Germany: Comparative Analysis Of Five Clearing Centers

In addition to unemployment, poor living and housing conditions and increased morbidity, precarious living situations are often characterized by a lack of or inadequate health insurance. In the Federal Republic of Germany, there is a difficult-to-quantify number of people who, for various reasons, have only limited health insurance coverage or none at all. Those affected include German citizens, EU citizens, and non-EU-citizens who are frequently homeless and/or undocumented. Health care for those groups is predominantly provided outside the German health care system by voluntary non-governmental and welfare organizations. Several German states and municipalities together with non-governmental organizations have addressed this problem by establishing clearing centers often combined with cost reimbursement for medical treatment. In this article, five of these projects are analysed and compared with each other with regard to their structure and mode of operation. The results show that many people can be helped in this way, but in order to meet the high and increasing demand, effort must be put towards more comprehensive solutions as well as on the expansion and sustainability of these projects. At the same time, there is an urgent need to improve statistical and epidemiological data.

Nutritional care is a human right: incorporating principles into clinical practice

The landmark Vienna Declaration (VD), initiated by the international human rights working group (IHRWG) and endorsed by more than 75 professional societies acknowledged that nutritional care is a human right alongside the right to food and the right to health. In a series of position papers the IHRWG have elaborated on the commitments required for translating human rights and ethical principles into clinical practice.1-3 The ultimate objective of the VD is to ensure all patients have a right to be screened and diagnosed for disease related malnutrition (DRM) then to receive evidence based artificially administered nutrition and hydration (AANH) administered by an interdisciplinary team of experts to positively impact clinical outcomes and survival. Unfortunately, nutritional care in hospitals and post discharge is frequently below standard and is not part of the patient's holistic care.2 Reasons include; inadequate education of healthcare professionals (HCPs), lack of malnutrition awareness, no reimbursement for nutrition treatments, or absence of public health policy.2,4

Use of Healthcare Claims Data to Generate Real-World Evidence on Patients With Drug-Resistant Epilepsy: Practical Considerations for Research.

Objectives: Regulatory bodies, health technology assessment agencies, payers, physicians, and other decision-makers increasingly recognize the importance of real-world evidence (RWE) to provide important and relevant insights on treatment patterns, burden/cost of illness, product safety, and long-term and comparative effectiveness. However, RWE generation requires a careful approach to ensure rigorous analysis and interpretation. There are limited examples of comprehensive methodology for the generation of RWE on patients who have undergone neuromodulation for drug-resistant epilepsy (DRE). This is likely due, at least in part, to the many challenges inherent in using real-world data to define DRE, neuromodulation (including type implanted), and related outcomes of interest. We sought to provide recommendations to enable generation of robust RWE that can increase knowledge of "real-world" patients with DRE and help inform the difficult decisions regarding treatment choices and reimbursement for this particularly vulnerable population. Methods: We drew upon our collective decades of experience in RWE generation and relevant disciplines (epidemiology, health economics, and biostatistics) to describe challenges inherent to this therapeutic area and to provide potential solutions thereto within healthcare claims databases. Several examples were provided from our experiences in DRE to further illustrate our recommendations for generation of robust RWE in this therapeutic area. Results: Our recommendations focus on considerations for the selection of an appropriate data source, development of a study timeline, exposure allotment (specifically, neuromodulation implantation for patients with DRE), and ascertainment of relevant outcomes. Conclusions: The need for RWE to inform healthcare decisions has never been greater and continues to grow in importance to regulators, payers, physicians, and other key stakeholders. However, as real-world data sources used to generate RWE are typically generated for reasons other than research, rigorous methodology is required to minimize bias and fully unlock their value.

Use of Healthcare Claims Data to Generate Real-World Evidence on Patients With Drug-Resistant Epilepsy: Practical Considerations for Research

Objectives: Regulatory bodies, health technology assessment agencies, payers, physicians, and other decision-makers increasingly recognize the importance of real-world evidence (RWE) to provide important and relevant insights on treatment patterns, burden/cost of illness, product safety, and long-term and comparative effectiveness. However, RWE generation requires a careful approach to ensure rigorous analysis and interpretation. There are limited examples of comprehensive methodology for the generation of RWE on patients who have undergone neuromodulation for drug-resistant epilepsy (DRE). This is likely due, at least in part, to the many challenges inherent in using real-world data to define DRE, neuromodulation (including type implanted), and related outcomes of interest. We sought to provide recommendations to enable generation of robust RWE that can increase knowledge of “real-world” patients with DRE and help inform the difficult decisions regarding treatment choices and reimbursement for this particularly vulnerable population. Methods: We drew upon our collective decades of experience in RWE generation and relevant disciplines (epidemiology, health economics, and biostatistics) to describe challenges inherent to this therapeutic area and to provide potential solutions thereto within healthcare claims databases. Several examples were provided from our experiences in DRE to further illustrate our recommendations for generation of robust RWE in this therapeutic area. Results: Our recommendations focus on considerations for the selection of an appropriate data source, development of a study timeline, exposure allotment (specifically, neuromodulation implantation for patients with DRE), and ascertainment of relevant outcomes. Conclusions: The need for RWE to inform healthcare decisions has never been greater and continues to grow in importance to regulators, payers, physicians, and other key stakeholders. However, as real-world data sources used to generate RWE are typically generated for reasons other than research, rigorous methodology is required to minimize bias and fully unlock their value.

P434 Incidence of chronic end-stage renal disease in patients with inflammatory bowel disease: a population-based cohort study from the French National Health Insurance Database

Abstract Background Renal disorders are among the extra-intestinal manifestations of inflammatory bowel disease (IBD), and treatments may also lead to renal failure. However, the incidence of end-stage renal disease (ESRD), defined as the need for renal replacement therapy (dialysis and/or renal transplantation(RT)), is poorly known. The primary objective of our study was to assess the incidence of ESRD in IBD patients, and to compare it with the non-IBD population. Methods We conducted a prognostic cohort study based on data from the French National Health Insurance Database(SNDS). Prevalent cases of IBD affiliated to the general health insurance scheme during the period 2011-2020 were identified on the basis of declarations long-term diseases ALD 24 or the presence of 2 hospital stays for IBD with ICD-10 codes or a single stay with reimbursement of specific treatments. Incident cases of ESRD were identified on the basis of a stay with a RT or a period with dialysis sessions for at least 45 days. Crude incidence rates of ESRD were calculated over the period for the IBD and the non-IBD population, with their 95% confidence intervals. Results A total of 280,283 IBD patients were included in the study over the period 2011-2020, 134,567 with ulcerative colitis(UC) and 145,716 with Crohn’s disease(CD). The mean age at study inclusion was 39.8 (± 17.1) years. The M/F sex ratio was 0.9. The mean age at incidence of ESRD in the non-IBD population was 65.0 (±17.0) years, the M/F sex ratio cases was 1.7. Among IBD patients, 534 patients began replacement therapy by dialysis or RT, they were younger than non-IBD patients (60.3(±16.7)years, P&amp;lt;0.001).The mean incidence rate of ESRD over the period was 26.4 per 100,000 person-years (IC95% 23.1-29.6), 480 (89.9%) patients entered ESRD by dialysis, and 54 (10.1%) by preemptive RT. In the non-IBD population the mean rate of ESRD was 15.9 per 100,000 person-years (IC95% 14.8- 17.1; P&amp;lt;0.0001 compared with IBD): 79,606 (94.5%) patients entered ESRD by dialysis, and 4,634 (5.5%) by RT (as compared with IBD,P&amp;lt;0.001). In subgroup analysis, the mean incidence rate of ESRD was 22.8 per 100,000 person-years (IC95% 18.9-26.7) in UC patients and 29.5 (IC95% 25.3-33.6) in CD patients. The rates of preemptive RT were 12.4% in UC patients (87.6% dialysis) and 8.6% in CD patients (91.4% dialysis). Over a median follow-up of 6.8 years, the survival rate after ESRD was 40.8% in ESRD-IBD patients VS 34.2% in ESRD non-IBD patients (P&amp;lt;0.001). Conclusion This study shows that the incidence of ESRD is significantly higher in IBD than in the non-IBD population, confirming the need for regular monitoring of renal function in our patients. The high incidence of RT confirms that access to transplantation is not restricted in this population.