Challenges for gene therapy in the financial sustainability of health systems: a scoping review.

AIM: To review the available evidence about the strategies implemented or proposed for coverage or reimbursement for currently approved gene therapies. METHODS: A scoping review was conducted to analyze the evidence published during the years 2016 to 2023. The main search criteria were coverage or reimbursement of gene therapy by healthcare systems. The eligible articles were those that described or proposed a financing model used to provide coverage in the various systems around the world. RESULTS: The study identified 279 publications, and after removing duplicates and screening for eligibility, 10 were included in the study. The results show that various financing models have been proposed, including subscription-based payment models, outcome-based payment models, and amortization strategies. However, several barriers to implementing these models were identified, such as deficiencies in informatics systems for data collection, changes in laws or regulations, the lack of accessible clinical endpoints and administrative costs. CONCLUSION: This scoping review provides an overview of financing strategies for gene therapies. Gene therapies can cure rare or previously intractable diseases, but their high cost can make access difficult. Publishing experiences with these models can help evaluate their use and gather more evidence for their effectiveness.

Insights into the Gut Microbiome of the South American Leaf-Toed Gecko (Phylodactylus gerropygus) Inhabiting the Core of the Atacama Desert.

Living in arid environments presents unique challenges to organisms, including limited food and water, extreme temperatures, and UV exposure. Reptiles, such as the South American leaf-toed gecko (Phyllodactylus gerrhopygus), have evolved remarkable adaptations to thrive in such harsh conditions. The gut microbiome plays a critical role in host adaptation and health, yet its composition remains poorly characterized in desert reptiles. This study aimed to characterize the composition and abundance of the gut microbiome in P. gerrhopygus inhabiting the hyperarid Atacama Desert, taking into account potential sex differences. Fecal samples from adult female and male geckos were analyzed by 16S rRNA gene amplicon sequencing. No significant differences in bacterial alpha diversity were observed between the sexes. However, the phylum Bacteroidota was more abundant in females, while males had a higher Firmicutes/Bacteroidota ratio. The core microbiome was dominated by the phyla Bacteroidota, Firmicutes, and Proteobacteria in both sexes. Analysis of bacterial composition revealed 481 amplicon sequence variants (ASVs) shared by female and male geckos. In addition, 108 unique ASVs were exclusive to females, while 244 ASVs were unique to males. Although the overall bacterial composition did not differ significantly between the sexes, certain taxa exhibited higher relative abundances in each sex group. This study provides insight into the taxonomic structure of the gut microbiome in a desert-adapted reptile and highlights potential sex-specific differences. Understanding these microbial communities is critical for elucidating the mechanisms underlying host resilience in Earth's most arid environments, and for informing conservation efforts in the face of ongoing climate change.

Rol de enfermería en el proceso de adaptación y autopercepción en pacientes ostomizados / Nursing role in the process of adaptation and self-perception in ostomized patients

Objetivo: Demostrar la importancia del rol de enfermería en los pacientes con ostomía. Metodología: Estudio cualitativo de un caso a través de una entrevista y artículos relacionados. Se ilustra el caso de una paciente ostomizada. Resultados: La ostomía supone un proceso de adaptación y aceptación, la cual afecta el autoestima y la percepción sobre sí mismo, siendo esto más frecuente en mujeres. Conclusiones: El paciente ostomizado sufre un fuerte impacto de su imagen corporal, lo que conlleva trastornos a nivel físico, psicológico y social. Es por esto que la importancia del profesional de enfermería quien brindará cuidados de manera integral a cada persona en el proceso de adaptación[AU]

Objective: To demonstrate the importance of the nursing role in ostomy patients. Methodology: Qualitative case study through an interview and related articles. The case of an ostomized patient is illustrated. Results: Ostomy involves a process of adaptation and acceptance, which affects self-esteem and self-perception, this being more frequent in women. Conclusions: The ostomized patient suffers a strong impact on his body image, which leads to physical, psychological and social disorders. This is why the importance of the nursing professional who will provide comprehensive care to each person in the adaptation process stands out[AU]

Objetivo: Demonstrar a importância do papel da enfermagem em pacientes com ostomia. Metodologia: Estudo de caso qualitativo através de uma entrevista e artigos relacionados. O caso de um paciente ostomizado é ilustrado. Resultados: A ostomia envolve um processo de adaptação e aceitação, que afeta a auto-estima e a autopercepção, sendo esta mais freqüente nas mulheres. Conclusões: O paciente ostomizado sofre um forte impacto sobre sua imagem corporal, o que leva a distúrbios físicos, psicológicos e sociais. É por isso que se destaca a importância do profissional de enfermagem, que prestará cuidados abrangentes a cada pessoa no processo de adaptação[AU]

Different Alternatives to Assess the Burden of Disease Using Attributable Fraction on a Disability Variable: The Case of Pain and Chronic Musculoskeletal Disorders in Chile.

OBJECTIVES: To estimate the burden of disease through 4 complementary procedures to years lived with disability (YLDs) using the concept of attributable fraction and including analysis of subdomains of disability. METHODS: We explored the burden on disability for 7 common musculoskeletal disorders (CMD) using the 2009 to 2010 Chilean National Health Survey, which included the Community Oriented Programme for the Control of Rheumatic Disease Core Questionnaire to identify cases with CMD, and an 8-domain questionnaire for health state descriptions. We calculated the proportion of disability attributable to pain in the general population and people with CMD. We also estimated the burden of CMD expressed as YLD and as the proportion of the disability in the general population attributable to people with CMD, with a particular focus in the pain domain of disability. Second order of uncertainty around point estimations was also characterized. RESULTS: Pain domain of disability accounted for 23.4% of the total disability in the general population, and between 20% (fibromyalgia) to 27.1% (osteoarthritis of the hip) in people with some of the selected CMD. People with chronic musculoskeletal pain accounted for 21.2% of total disability from general population, which generated 1.2 million of YLD (6679 YLD/100 000 inhabitants). Chronic low back pain and osteoarthritis of the knee were in the top position of specific CMDs, explaining the highest national burden. CONCLUSION: Pain is an essential component of disability in people with CMD and also in the general population. The approach used can be easily applied to other health conditions and other domains of disability.

Costs and consequences of chronic pain due to musculoskeletal disorders from a health system perspective in Chile.

BACKGROUND: Chronic pain is a prevalent and distressing condition caused by an unceasing pain lasting more than 3 months or a pain that persists beyond the normal healing time. There is evidence of inadequate management partly explained by the unawareness regarding the magnitude of the problem. OBJECTIVES: To estimate the annual expected costs and consequences of chronic pain caused by musculoskeletal diseases from the health system perspective in Chile. METHODS: A Markov cohort model was built to represent chronic pain and estimate expected costs and consequences over 1-year time horizon. Transition probabilities were obtained through expert elicitation. Consequences examined were: years lost to disability (YLD), depression, anxiety, and productivity losses. Direct health care costs were estimated using local sources. Probabilistic sensitivity analysis was performed to characterize second-order uncertainty. RESULTS: The annual expected cost due to musculoskeletal chronic pain was estimated in USD $1387.2 million, equivalent to 0.417% of the national GDP. Lower back pain and osteoarthritis of the knee explained the larger proportion of the total cost, 31.8% and 27.1%, respectively. Depression attributed to chronic pain is another important consequence accounting for USD $94 million (Bayesian credibility interval 95% $49.1-$156.26). Productivity losses were also important cost, although early retirement and presenteeism were not measured. Chronic pain causes 137,037 YLDs. CONCLUSION: Chronic pain is not only an important cause of disability but also responsible for high social and financial burden in Chile. Public health programs focused on managing chronic pain may decrease burden of disease and possibly reduce costs.

Cost Effectiveness of Daclatasvir/Asunaprevir Versus Peginterferon/Ribavirin and Protease Inhibitors for the Treatment of Hepatitis c Genotype 1b Naïve Patients in Chile.

INTRODUCTION: Daclatasvir and Asunaprevir (DCV/ASV) have recently been approved for the treatment of chronic hepatitis C virus infection. In association, they are more effective and safer than previous available treatments, but more expensive. It is unclear if paying for the additional costs is an efficient strategy considering limited resources. METHODS: A Markov model was built to estimate the expected costs in Chilean pesos (CL$) and converted to US dollars (US$) and benefits in quality adjusted life years (QALYs) in a hypothetic cohort of naive patients receiving DCV/ASV compared to protease inhibitors (PIs) and Peginterferon plus Ribavirin (PR). Efficacy was obtained from a mixed-treatment comparison study and costs were estimated from local sources. Utilities were obtained applying the EQ-5D survey to local patients and then valued with the Chilean tariff. A time horizon of 46 years and a discount rate of 3% for costs and outcomes was considered. The ICERs were estimated for a range of DCV/ASV prices. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: PIs were extendedly dominated by DCV/ASV. The ICER of DCV/ASV compared to PR was US$ 16,635/QALY at a total treatment price of US$ 77,419; US$11,581 /QALY at a price of US$ 58,065; US$ 6,375/QALY at a price of US$ 38,710; and US$ 1,364 /QALY at a price of US$ 19,355. The probability of cost-effectiveness at a price of US$ 38,710 was 91.6% while there is a 21.43% probability that DCV/ASV dominates PR if the total treatment price was US$ 19,355. Although the results are sensitive to certain parameters, the ICER did not increase above the suggested threshold of 1 GDP per capita. CONCLUSIONS: DCV/ASV can be considered cost-effective at any price of the range studied. These results provide decision makers useful information about the value of incorporating these drugs into the public Chilean healthcare system.