Background Patients with alpha-1 antitrypsin deficiency (AATD), commonly categorized as a rare disease, have been affected by the changes in healthcare management brought about by COVID-19. This study’s aim was to identify the changes that have taken place in AATD patient care as a result of the COVID-19 pandemic in Spain and to propose experts’ recommendations aimed at ensuring humanized and quality care for people with AATD in the post-pandemic situation. Methods A qualitative descriptive case study with a holistic single-case design was conducted, using focus groups with experts in AATD clinical management, including 15 health professionals with ties to the Spanish health system (12 pneumologists and 2 hospital pharmacists from 11 different hospitals in Spain) and 1 patient representative. Results COVID-19 has had a major impact on numerous aspects of AATD clinical patient management in Spain, including diagnostic, treatment, and follow-up phases. The experts concluded that there is a need to strengthen coordination between Primary Care and Hospital Care and improve the coordination processes across all the organizations and actors involved in the healthcare system. Regarding telemedicine and telecare, experts have concluded that it is necessary to promote this methodology and to develop protocols and training programs. Experts have recommended developing personalized and precision medicine, and patient participation in decision-making, promoting self-care and patient autonomy to optimize their healthcare and improve their quality of life. The possibility of monitoring and treating AATD patients from home has also been proposed by experts. Another result of the study was the recommendation of the need to ensure that plasma donations are made on a regular basis by a sufficient number of healthy individuals. Conclusion The study advances knowledge by highlighting the challenges faced by health professionals and changes in AATD patient management in the context of the COVID-19 pandemic. It also proposes experts’ recommendations aimed at ensuring humanized and quality care for people with AATD in the post-pandemic situation. This work could serve as a reference study for physicians on their daily clinical practice with AATD patients and may also provide guidance on the changes to be put in place for the post-pandemic situation.
We conducted research to assess hospital pharmacists’ familiarity with/interpretation of data requirements for the different regulatory approval frameworks and the impact of this on their approach to substitution in the formulary. The online questionnaire included a small molecule (acetylsalicylic acid—follow-ons approved via the generic pathway), two biologic drugs (insulin glargine and etanercept—follow-ons approved via the biosimilar pathway), a non-biologic complex drug (NBCD; glatiramer acetate—follow-ons approved via the hybrid pathway) and a nanomedicine, ferric carboxymaltose (no follow-ons approved as yet). The study was conducted in two phases: an initial qualitative pilot study with 30 participants, followed by a quantitative stage involving 201 pharmacists from five European countries. Most expected negligible safety/efficacy differences between reference and follow-on products. Head-to-head clinical data showing therapeutic equivalence as a prerequisite for reference product/follow-on substitution was perceived to be needed most for biologics (47%), followed by NBCDs (44%)/nanomedicines (39%) and small molecules (23%). Overall, 28% did not know the data requirements for follow-on approval via the hybrid pathway; 16% were familiar with this pathway, compared with 50% and 55% for the generic and biosimilar pathways, respectively. Overall, 19% of respondents thought the European Medicines Agency (EMA) was responsible for defining the substitutability of follow-ons. Education is required to increase hospital pharmacist’s knowledge of regulatory approval frameworks and their relevance to substitution practices.
Background:The availability of oral janus kinase inhibitors, as tofacitinib, has extended the treatment pathways for management of patients with rheumatoid arthritis (RA)Objectives:To assess the cost-effectiveness of tofacitinib as second-line treatment compared to treatment sequences containing standard biological-therapies in patients with moderate to severe RA after failure to disease-modifying antirheumatic drugs (DMARDs) from the Spanish National Health System perspective.Methods:A patient-level microsimulation model was used to compare the lifetime cost and quality-adjusted life-years (QALY) for treatment sequences initiating with tofacitinib (5mg BID) followed by biological therapies versus sequences of biological treatments excluding tofacitinib. The sequences were selected by a panel of experts based on clinical practice in Spain. Concomitant treatment with methotrexate (MTX) was considered along all the therapies in the treatment sequences.Model parameters included age, weight, initial Health Assessment Questionnaire (HAQ) score and clinical response to short and long treatment. Efficacy was measured by means of HAQ score changes using mixed-treatment-comparisons (for the first 6 months) and data from long-term extension trials (for later periods).Serious adverse event (SAE) information derived from literature.The estimation of total cost for sequences included: drugs acquisition (public ex-factory prices with mandatory deduction or reference prices) and parenteral administration, disease progression and SAE management. Local unitary costs (€, 2018) were applied. Additional comparisons were explored testing other potential sequences.Results:The base case results showed that sequences initiating with tofacitinib provided greater outcomes than the correspondent sequences excluding tofacitinib. In scenario 1 tofacitinib+MTX→Rituximab+MTX→sc Tocilizumab+MTX→Etanercept+MTX→Certolizumab+MTX provided 13.99 QALYs versus 13.92 QALYs for adalimumab+MTX→Rituximab+MTX→sc Tocilizumab+MTX→Etanercept+MTX→Certolizumab+MTX. In scenario 2 tofacitinib+MTX→Adalimumab+MTX→Rituximab+MTX→sc Tocilizumab+MTX→Etanercept+MTX provided 13.75 QALYs versus 13.62 QALYs for Baricitinib+MTX→Adalimumab+MTX→Rituximab+MTX→sc Tocilizumab+MTX→Etanercept+MTX.Tofacitinib-containing sequences provided lower total costs than the alternative sequences (-€5,783 and -€13,975 for the pairwise comparisons previously described), resulting, therefore, dominant options versus sequences excluding tofacitinib.On the probabilistic sensitivity analyses, sequences initiating with tofacitinib resulted cost-effective in 64.0% (scenario 1) and 56.9% (scenario 2) of the 1,000 iterations performed, because incremental cost-effectiveness ratio fell below a €25,000/QALY gained willingness to pay threshold.Conclusion:These results suggest that the inclusion of tofacitinib could be a dominant strategy for treatment of moderate to severe RA patients after DMARDs failure in Spain.Disclosure of Interests:Carmen Peral Shareholder of: Carmen Peral is employee of and shareh...
Disease-related malnutrition is a common pathology in the developed world. It affects 30 million people in Europe and it has an associated cost of one hundred and seventy billion euros per year. In Spain, around 1.7 million adults, 4.4% of total population, are negatively affected. Malnutrition affects one out of four patients on hospital admissions. It is estimated that hospitalized patients who are malnourished throughout their stay range from 23.7% to 37%; sharpening these figures for multi-patients over 70 years old, and almost 10% are disensuaded during their hospital stay. All this implies an increase in hospital stays and associated costs; in particular, the direct costs of hospital malnutrition were estimated at 1.143 million annually in 2009, representing 1.8% of health expenses within the national health system. Advances in technology and infrastructure have facilitated the transfer of complex hospital-to-home services. Nowadays, the international trend points to the increasing development of the domicile as a care center. A meta-analysis of 61 randomized and controlled trials, published in 2012, showed that patients treated at home had a six-month mortality rate between 19% and 38% lower than those who were hospitalized. Home artificial nutrition (HAN) is the nutrients home-administration, among other therapeutic agents, administered through the digestive tract -home enteral nutrition (HEN)- or intravenously -home parenteral nutrition (NPD-), with the objective of improving or maintaining, at home, the patient’s nutritional status. That reports into a benefit for the patient as being able to continue the family, social and work commitments. The environment, in which 21st century health professionals work, is changing significantly. There are multiple innovations in process management, given by the growing potential offered by new technologies in remote care. It supports the patient’s home stay, either in the community or in a more familiar environment, that is, in a more humanized environment. Finally, HAN is shown to be an effective-cost alternative in relation to classical hospital care, resulting in the adoption of the efficiency criterion as an element of prioritization in healthcare.
e19596 Background: ZA therapy has been associated with the development of ONJ. In Nov 8th, 2005, osteonecrosis of the jaw was elevated to a safety warning by Spanish Drug Agency (SDA). The aim of this study was to investigate if that warning has had any effect in the pattern of ZA administration in a general hospital. Methods: We retrospectively analysed database using pharmacy, medical and nurse claims data from the Oncobass integrated claims database for all pts diagnosed with cancer who had received treatment with ZA between Jan 1, 2002, and Dec 31, 2011. Delivered doses of ZA were stratified in 2 groups: used before the SDA warning and after the SDA warning. Main variable: time between two consecutive doses (TTCD). Secondary variables: age, sex and diagnosis. Descriptive statistical analysis were applied for both groups. Comparisons of means of TTCD before and after the warning were performed with Student’s test, or the Mann-Whitney test, as necessary. Statistic significance: p value < 0.05. Results: We identified 761 patients (374 (49.2%) male; 387 (50.8%) female), median age 64 y (25 – 92) who were delivered 7301 doses [mean: 9.6 doses/patient (d/p)]: 2,353 (32.2%) before the SDA warning and 4948 (67.8%) after. Pathologies: breast cancer (BC) 257 patients (pts) (33,77%), 2,918 doses (40%), mean 11.4 d/p; multiple myeloma (MM) 167 pts (21.94%), 2001 doses (27%), 12 d/p; prostate adenocarcinoma (PA) 129 pts (16.95%), 1,510 doses (20.7%), 11.7 d/p; lung cancer (LC), 112 pts (14.72%), mean 4.2 d/p, 465 doses (6.4%); other 96 pts (12.61%), 407 doses (5,5%), 4.2 d/p. 3,126 (43%) doses were delivered to male and 4,149 (57%) to female. TTCD was significantly increased after ths SDA warning [32.12 vs 33.87 days (d); p< 0.001; CI 95% (-0.79)- (-0.90)]. By pathologies, differences were significant only for MM [35.8 vs 44.4 d; p<0.001; CI 95% (-10.19)-(-6.99)] and for PA [28.67 vs 32.03; p < 0.001; CI 95% (-5.08)-(-1.63)], but not for the other pathologies. Median d/p was unchanged before and after warnings for all pathologies except for MM (13 d/p vs 8.3 d/p). Conclusions: In our environment, ONJ warnings have not changed substantially the ZA schedule pattern, except for MM.
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