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Background There is limited evidence on within-country discrepancies in biosimilar uptake. This study analyzes differences in timing and diffusion of biosimilar uptake across Portuguese NHS hospitals and explores possible determinants. Research design and methods We analyzed publicly accessible consumption data of originator biologic and biosimilar drugs for adalimumab, etanercept, infliximab, rituximab, and trastuzumab, by hospital and month for the years 2015–2021 (N = 9,467). We modeled the time to biosimilar adoption using survival regression models and the share of biosimilar consumption using generalized estimated equations with random hospital effects. Results Academic hospitals were characterized by a quicker uptake of adalimumab and infliximab biosimilars but lower shares for other drugs. A higher total consumption of biologics was related to a lower share of biosimilar uptake. A stronger participation in randomized controlled trials was linked to higher biosimilar shares and quicker uptake, except for rituximab. If all NHS hospitals had biosimilar shares equal to the highest ones, potential annual savings could reach 13.9 million euros. Conclusion The findings suggest a need for capacity-building on biosimilar prescribing, including for doctors of academic hospitals and those working in settings where high biosimilar use would be expected.

Background: The role of demographic and socio-economic determinants of COVID-19 transmission is still unclear and is expected to vary in different contexts and epidemic periods. Exploring such determinants may generate a hypothesis about transmission and aid the definition of prevention strategies. Objectives: To identify municipality-level demographic and socio-economic determinants of COVID-19 in Portugal. Methods: We assessed determinants of COVID-19 daily cases at 4 moments of the first COVID-19 epidemic wave in Portugal, related with lockdown and post-lockdown measures. We selected 60 potential determinants from 5 dimensions: population and settlement, disease, economy, social context, and mobility. We conducted a multiple linear regression (MLR) stepwise analysis (p < 0.05) and an artificial neural network (ANN) analysis with the variables to identify predictors of the number of daily cases. Results: For MLR, some of the identified variables were: resident population and population density, exports, overnight stays in touristic facilities, the location quotient of employment in accommodation, catering and similar activities, education, restaurants and lodging, some industries and building construction, the share of the population working outside the municipality, the net migration rate, income, and renting. In ANN, some of the identified variables were: population density and resident population, urbanization, students in higher education, income, exports, social housing buildings, production services employment, and the share of the population working outside the municipality of residence. Conclusions: Several factors were identified as possible determinants of COVID-19 transmission at the municipality level. Despite limitations to the study, we believe that this information should be considered to promote communication and prevention approaches. Further research should be conducted.

We aimed to determine how Portuguese physicians handle major bleeding. We also aim to establish global diagnostic and therapeutic recommendations to be followed in clinical practice by using a step-wise approach of evidence generation. This study followed a three-step process: a steering committee desk review, a Delphi technique, an expert panel meeting. A modified 3-round Delphi including 31 statements was performed. Questions were answered in a five-point Likert-type scale. Consensus threshold was established as a percentage of agreement among participants ≥90% in the first round, and ≥85% in the second and third rounds. The level of consensus achieved by panelists was discussed with the scientific committee (January-2020). Fifty-one physicians participated in the study (compliance rate >90%). Analyzing the three rounds, consensus was reached on 20 items (64.5%) in the first, 4/11 items (36.4%) in the second and 6/7 items (85.7%) in the third. One statement about administration of clotting factor concentrates for bleeding control did not reach consensus. A high level of consensus was reached toward the need for implementing Patient Blood Management strategies in Portuguese hospitals, reduce exposure to allogeneic blood components, to use goal directed therapies for acquired bleeding management, and the need for evaluating blood transfusion indirect costs. A final version with 12 recommendations was built, according to the Grading of Recommendations Assessment, Development and Evaluation (GRADE). Our results provide critically appraised and updated evidence on bleeding coagulopathies management in Portugal. Additional studies, mainly about indirect costs of blood transfusion, are needed.

Background We aimed to identify the perception of physicians on the limitations and delays for diagnosing, staging and treatment of lung cancer in Portugal. Methods Portuguese physicians were invited to participate an electronic survey (Feb-Apr-2020). Descriptive statistical analyses were performed, with categorical variables reported as absolute and relative frequencies, and continuous variables with non-normal distribution as median and interquartile range (IQR). The association between categorical variables was assessed through Pearson’s chi-square test. Mann-Whitney test was used to compare categorical and continuous variables (Stata v.15.0). Results Sixty-one physicians participated in the study (45 pulmonologists, 16 oncologists), with n = 26 exclusively assisting lung cancer patients. Most experts work in public hospitals (90.16%) in Lisbon (36.07%). During the last semester of 2019, responders performed a median of 85 (IQR 55–140) diagnoses of lung cancer. Factors preventing faster referral to the specialty included poor articulation between services (60.0%) and patients low economic/cultural level (44.26%). Obtaining National Drugs Authority authorization was one of the main reasons (75.41%) for delaying the begin of treatment. The cumulative lag-time from patients’ admission until treatment ranged from 42–61 days. Experts believe that the time to diagnosis could be optimized in around 11.05 days [IQR 9.61–12.50]. Most physicians (88.52%) started treatment before biomarkers results motivated by performance status deterioration (65.57%) or high tumor burden (52.46%). Clinicians exclusively assisting lung cancer cases reported fewer delays for obtaining authorization for biomarkers analysis (p = 0.023). Higher waiting times for surgery (p = 0.001), radiotherapy (p = 0.004), immunotherapy (p = 0.003) were reported by professionals from public hospitals. Conclusions Physicians believe that is possible to reduce delays in all stages of lung cancer diagnosis with further efforts from multidisciplinary teams and hospital administration.

Introduction and objectives We aimed to build a national consensus to optimize the use of oral corticosteroids (OCS) in severe asthma in Portugal. Material and methods A modified 3-round Delphi including 65 statements (topics on chronic systemic corticotherapy, therapeutic schemes, asthma safety and monitoring) was performed via online platform (October-November 2019). A five-point Likert-type scale was used (1-‘strongly disagree’; 5-‘strongly agree’). Consensus threshold was established as a percentage of agreement among participants ≥90% in the 1st round and ≥85% in the 2nd and 3rd rounds. The level of consensus achieved by the panel was discussed with the participants (face-to-face meeting). Results Forty-eight expert physicians in severe asthma (specialists in allergology and pulmonology) participated in the study. Almost half of the statements (28/65; 43.1%) obtained positive consensus by the end of round one. By the end of the exercise, 12 (18.5%) statements did not achieve consensus. Overall, 87% of physicians agree that further actions for OCS cumulative risk assessment in acute asthma exacerbations are needed. The vast majority (91.7%) demonstrated a favorable perception for using biological agents whenever patients are eligible. Most participants (95.8%) are more willing to accept some degree of lung function deterioration compared to other outcomes (worsening of symptoms, quality of life) when reducing OCS dose. Monitoring patients’ comorbidities was rated as imperative by all experts. Conclusions : These results can guide an update on asthma management in Portugal and should be supplemented by studies on therapy access, patients’ adherence, and costs. This work was supported by AstraZeneca. The funding source had no role in study design, data collection, analysis and interpretation, and in the writing or submitting decision of the article.

Background: Transthyretin-associated familial amyloid polyneuropathy (TTR-FAP) is a rare, hereditary, progressive and neurodegenerative disease. We aimed to study ­TTR-FAP epidemiology in Portugal. Methods: National, observational, prospective and retrospective, case identification of adults with TTR-FAP. Countrywide patient multiple identification sources included reference centers registries and centralized medical electronic prescription database. Crude rates were reported per 100,000 adult inhabitants. Results: Over 2010–2016 period, mean incidence rates was 0.87/100,000 (95% CI 0.68–1.10) corresponding to 71 new patients yearly, that has decreased 31% in the last 7 years. The proportion of late-onset cases (age ≥50 years) among incident cases was 28.7%. Estimated crude 2016 prevalence was 22.93/100,000 adult inhabitants (95% CI 21.90–23.99) corresponding to 1,865 TTR-FAP individuals in Portugal (45.8% male; mean age: 52.3 ± 15.4 years). In 2016, the Portuguese region with the highest TTR-FAP prevalence shows a 16% prevalence increase over the last 25 years. Conclusions: In Portugal, TTR-FAP affects both genders and mainly young adults. TTR-FAP incidence appears to be decreasing while prevalence is increasing. In comparison to previous studies, there is an increased representativeness of late-onset patients. This epidemiological setting poses future and complex challenges for the social and healthcare system, strengthening the relevance of regular epidemiologic surveillance.

The assessment of olfactory dysfunction (partial or total anosmia) is important in the early diagnosis of neurodegenerative diseases and may be a good indicator in clinical diagnosis. Currently, there is no diagnostic test for partial or total anosmia validated for the Portuguese population. The aim of this study was the development and rheological and sensory characterization of hospital compounded formulations containing 23 different fragrances adapted to Portuguese population. During the studies, several PEG-based formulations were developed. Viscoelasticity assays were performed in the Rheometer Kinexus using the oscillatory method. Differential scanning calorimeter and microscopy were assessed as well as the volatile components analysis by GC-MS. Thus, a new compounding for the diagnosis of olfactive dysfunction with a structured and rheological characteristic adapted to the Portuguese population were developed.

Background The assessment of olfactory dysfunction, partial or total anosmia, is very important in the early diagnosis of neurodegenerative diseases. This diagnosis is difficult because it depends on the cultural habits of each population and in Portugal there is no test adapted to its population. Purpose The aim of this study was the development of a Portuguese kit (PT-smell test) to assess olfactory dysfunctions. Thus, several compounded formulations adapted to the Portuguese population were developed, characterised and clinically validated. Material and methods The PT-smell test was developed based on the results of the perception characterisation study, performed through a national cross-sectional survey, which allows the identification of Portuguese fragrances. Thus, different PEG-based formulations were developed and structure characterisation was performed using rheology, differential scanning calorimetry, microscopy, fragrance identification and stability tests. The olfactory performance of 27 patients presenting with olfactory disorders and 25 healthy controls were evaluated with the PTsmell test and Barcelona smell test, as the reference diagnostic test. Results A kit of 23 formulations containing polyethylene glycol 1500 and 400 (50:50) and 23 odours presented semisolid behaviour, a non-crystal structure and the fragrance’s volatile ingredients remained stable for 6 months when packed in amber glass flasks. Concerning the clinical study, the results obtained show no gender difference (p>0.05) between the two groups, although the mean age of the control group (39.03±12.91 years) is statistically different (p=0.0035) from the patient group (50.62±18.76 years). The PT-smell test is a reliable method for assessing human olfaction with good correlation to the Barcelona smell test (r2=0.9214). A limit for hyposmia has been determined, a score of 71.14% for forced choice identification. Conclusion The PT-smell test could be used as a reliable screening method and also used as an olfactory threshold test and as an olfactory training kit. Additionally, this study was characterised by its multidisciplinary aspect. Doctors and pharmacists have worked towards a common goal, improving the well-being of the patient. Interinstitutional collaboration between public hospitals and colleges should be improved and encouraged by the Ministry of Health and Governments in order to rationalise human and financial resources. References and/or acknowledgements No conflict of interest.

© Springer International Publishing 2016, The definitive version is available at http://link.springer.com/, Background In view of the current financial and demographic situation in Portugal, accessibility to health care may be affected, including the ability to adhere to medication. Objective To evaluate the perceived effects of the crisis on elderly patient's access to medicines and medical care, and its implications on medicine-taking behaviour. Setting Community pharmacy. Method A cross-sectional study was undertaken during April 2013, where elderly patients answered a self-administered questionnaire based on their health-related experiences in the current and previous year. Binary logistic regression was used to ascertain the effects of potential predictors on the likelihood of adherence. Main outcome measures self-reported adherence. Results A total of 1231 questionnaires were collected. 27.3% of patients had stopped using treatments or health services in the previous year for financial motives; mostly private medical appointments, followed by dentist appointments. Almost 30% of patients stopped purchasing prescribed medicines. Over 20% of patients reduced their use of public services. Out-of-pocket expenses with medicines were considered higher in the current year by 40.1% of patients. The most common strategy developed to cope with increasing costs of medicines was generic substitution, but around 15% of patients also stopped taking their medication or started saving by increasing the interdose interval. Conclusion Reports of decreasing costs with medicines was associated with a decreased likelihood of adherence (OR 0.42; 95% CI 0.27-0.65). Lower perceived health status and having 3 or more co-morbidities were associated with lower odds of adhering, whilst less frequent medical appointments was associated with a higher likelihood of exhibiting adherence.

Objective: the main objective of this study was to assess the pattern of use of oral antidiabetic drugs in a national sample of type 2 diabetes. Study design: descriptive and cross-over study. Place of collection of information: 110 community pharmacies distributed nationally, with a proportional distribution in each country’s health region. Results: The vast majority of patients reported the use of medical consultation in the Health Center to follow the usual diabetes. The most used drug was metformin, used by 74,56 per cent of patients, accounting for 44,62 per cent of oral antidiabetics drugs. In the group of patients treated exclusively with oral antidiabetic drugs, approximately 40 per cent of patients used only 1 oral antidiabetic drug and the remaining were treated with oral antidiabetic drugs in polytherapy. Conclusions: The pattern of use of oral antidiabetic drugs identified in this study is similar to that described in other european countries, in particular the pharmacotherapeutic sub-group more used, the biguanides, which is consistent with internationally accepted therapeutic guidelines.

Tese de doutoramento, Farmácia (Farmacoepidemiologia), Universidade de Lisboa, Faculdade de Farmácia, 2012 Submitted by amelia Janeiro (ajaneiro@reitoria.ul.pt) on 2012-04-03T14:10:49Z No. of bitstreams: 1 ulsd062274_td_Maria_Carmona.pdf: 4788632 bytes, checksum: daaa992dd724c3121695b19179fe8ae4 (MD5) Made available in DSpace on 2012-04-03T14:57:14Z (GMT). No. of bitstreams: 1 ulsd062274_td_Maria_Carmona.pdf: 4788632 bytes, checksum: daaa992dd724c3121695b19179fe8ae4 (MD5) Previous issue date: 2011

Background Transthyretin Familial Amyloid Polyneuropathy (TTRFAP) is a rare, progressive, debilitating and life-threatening neurodegenerative disease. TTR-FAP is a rare disease worldwide. In Europe a disease is defined as rare when it affects less than 1 in 2000 inhabitants. Portugal has the largest cluster worldwide nonetheless recent Portuguese epidemiologic data is lacking. The purpose of this study is to estimate TTR-FAP prevalence in Portugal.

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