Repurposing of Medicines in the EU: Launch of a Pilot Framework

Repurposing of authorised medicines has been under discussion for a long time. Drug repurposing is the process of identifying a new use for an existing medicine in an indication outside the scope of the original approved indication. Indeed, the COVID-19 health crisis has brought the concept to the frontline by proving the usefulness of this practise in favour of patients for an early access to treatment. Under the umbrella of the Pharmaceutical Committee and as a result of the discussions at the European Commission Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) a virtual Repurposing Observatory Group (RepOG) was set up in 2019 to define and test the practical aspects of a pilot project thought to provide support to “not-for-profit” stakeholders generating or gathering data for a new therapeutic use for an authorised medicine. The group's initial plan was impacted by the outbreak of the SARS-CoV-2 pandemic and the launch of the pilot needed to be postponed. This article describes the progress and the activities conducted by the group during this past and yet extraordinary 2020–2021 to keep the project alive and explores on the background of this topic together with the obvious opportunities this health crisis has brought up in terms of repurposing of medicines.

A Systematic Review on Immunotherapy for Guillain- Barre Syndrome

Guillain-Barré syndrome (GBS) is an acute immunemediated polyradiculoneuropathy with a highly variable clinical course and outcome. Intravenous immunoglobulin (IVIG) and plasma exchange (PE) are proven effective treatments, but about half of the patients may not respond to these therapies; moreover, these are not established yet to treat patients of every stage of GBS or its variants. Results from the International Guillain-Barré Syndrome Outcome Study suggest that geographical variations exist in Guillain-Barré syndrome, including insufficient access to immunotherapy in low-income countries. There is a need to provide improved access to treatment for all patients with Guillain-Barré syndrome, and to develop effective disease-modifying therapies that can limit the extent of nerve injury. In this review, the current literature about immunotherapeutic options is highlighted in the context of stages of the disease and its variants and additionally, upcoming modalities are discussed briefly. J Bangladesh Coll Phys Surg 2022; 40: 57-64

Caregivers’ experiences and perspectives of factors associated with relapse in Iranian people living with schizophrenia: A qualitative study

Background: Relapse in People Living with Schizophrenia (PLS) has several reasons and recognizing these can increase the effectiveness of treatment interventions. Formal and informal caregivers are an informed source to reduce relapse in PLS. Aim: This study explores the caregivers’ perspective in Iran on the factors affecting relapse in PLS. Method: A total of 28 caregivers (16 formal caregivers and 12 informal caregivers) of PLS were enrolled in our qualitative study. A content analysis was conducted using individual and group, semi-structured in-depth interviews with informal and formal caregivers of PLS. This study was conducted in a hospital, three universities, and a non-governmental organization in Tehran, Iran. Results: The majority (69%) of the participants were females. About half of the informal caregivers were over 60 years old and about 40% of the formal caregivers were in the age range of 30 to 40 years. The average number of years of work for informal caregivers was 17.6 years and the average of work experience among the formal caregivers was 14.1 years. Seven key dual themes were identified from data: ‘awareness-stigma’, ‘social support-social exclusion’, ‘treatment adherence-treatment discontinuation’, ‘holistic approach – one-dimensional approach’, ‘supported employment-social dysfunction’, ‘emotional management in family – family with high emotional expression’, and ‘access to treatment-treatment gap’. Conclusion: The results of this research can help practitioners and policymakers to enable evidence-based practices to reduce relapse in PLS by emphasizing and acting on factors identified in our analyses.

The First Interchangeable Biosimilar Insulin: Insulin Glargine-yfgn

On March 23, 2020, all insulin products were reclassified as biologics instead of drugs under the Biological Price Competition and Innovation (BPCI) Act of 2009. This allows biosimilar insulin products to be manufactured when the patent expires for the reference biologic, sometimes called the originator or brand name product. A biosimilar product may not be substituted for the reference biologic at the pharmacy counter unless the biosimilar undergoes further switch trials to earn the designation as an interchangeable biosimilar. Insulin glargine-yfgn 100 units/mL is the first biosimilar insulin to attain interchangeable status with the reference insulin glargine. In the INSTRIDE 1 and INSTRIDE 2 trials, insulin glargine-yfgn has proven noninferiority regarding blood glucose reduction and adverse effect profile versus reference insulin glargine; even in the INSTRIDE 3 trial in which treatment of diabetes was switched between insulin glargine-yfgn and reference insulin glargine throughout the trial without statistically significant changes to glucose levels or adverse effects. Insulin glargine-yfgn may be substituted at the pharmacy counter without consultation with the prescriber, in accordance with state laws. In suit with other biosimilars, insulin glargine-yfgn’s list price is significantly lower than other insulin glargine products. This increases market competition leading to decreases in costs of other insulin glargine products. Many patients who could not previously afford insulin therapy may now have significantly improved access to treatment. Providers will need education to increase awareness of these new biosimilars and interchangeable biosimilar insulin products, cost benefits, and substitution allowances.

HIV surveillance and research for migrant populations: a protocol integrating respondent-driven sampling, case finding, and medicolegal services for Venezuelans living in Colombia (Preprint)

BACKGROUND Epidemiologic research among migrant populations is limited by logistical, methodological, and ethical challenges, but necessary for informing public health and humanitarian programming. OBJECTIVE We describe a methodology to estimate HIV prevalence among Venezuelan migrants in Colombia. METHODS Respondent-driven sampling (RDS), a non-probability sampling method, was selected for attributes of reaching highly networked populations without sampling frames and analytic methods that permit estimation of population parameters. RDS was modified to permit electronic referral of peers via SMS and Whatsapp. Participants complete socio-behavioral surveys and rapid HIV and syphilis screening tests with confirmatory testing. HIV treatment is not available for migrants who have entered Colombia through irregular pathways; thus, medicolegal services integrated into post-test counseling provide staff lawyers and legal assistance to participants diagnosed with HIV or syphilis for sustained access to treatment through the national health system. Case finding is integrated into RDS to allow partner referral. The study is implemented by a local community-based organization providing HIV support services and related legal services for Venezuelans in Colombia. RESULTS Data collection launched in four cities in July and August 2021. As of November 2021, 3,105 of the target 6,100 participants were enrolled. CONCLUSIONS Tailored methods that combine community-led efforts with innovations in sampling and linkage to care can aid in advancing health research for migrant and displaced populations. Worldwide trends in displacement and migration underscore the value of improved methods for translation to humanitarian and public health programming.

Differences in Baseline Characteristics and Access to Treatment of Newly Diagnosed Patients With IPF in the EMPIRE Countries

Idiopathic pulmonary fibrosis (IPF) is a rare lung disease with poor prognosis. The diagnosis and treatment possibilities are dependent on the health systems of countries. Hence, comparison among countries is difficult due to data heterogeneity. Our aim was to analyse patients with IPF in Central and Eastern Europe using the uniform data from the European Multipartner IPF registry (EMPIRE), which at the time of analysis involved 10 countries. Newly diagnosed IPF patients (N = 2,492, between March 6, 2012 and May 12, 2020) from Czech Republic (N = 971, 39.0%), Turkey (N = 505, 20.3%), Poland (N = 285, 11.4%), Hungary (N = 216, 8.7%), Slovakia (N = 149, 6.0%), Israel (N = 120, 4.8%), Serbia (N = 95, 3.8%), Croatia (N = 87, 3.5%), Austria (N = 55, 2.2%), and Bulgaria (N = 9, 0.4%) were included, and Macedonia, while a member of the registry, was excluded from this analysis due to low number of cases (N = 5) at this timepoint. Baseline characteristics, smoking habit, comorbidities, lung function values, CO diffusion capacity, high-resolution CT (HRCT) pattern, and treatment data were analysed. Patients were significantly older in Austria than in the Czech Republic, Turkey, Hungary, Slovakia, Israel, and Serbia. Ever smokers were most common in Croatia (84.1%) and least frequent in Serbia (39.2%) and Slovakia (42.6%). The baseline forced vital capacity (FVC) was >80% in 44.6% of the patients, between 50 and 80% in 49.3%, and <50% in 6.1%. Most IPF patients with FVC >80% were registered in Poland (63%), while the least in Israel (25%). A typical usual interstitial pneumonia (UIP) pattern was present in 67.6% of all patients, ranging from 43.5% (Austria) to 77.2% (Poland). The majority of patients received antifibrotic therapy (64.5%); 37.4% used pirfenidone (range 7.4–39.8% between countries); and 34.9% nintedanib (range 12.6–56.0% between countries) treatment. In 6.8% of the cases, a therapy switch was initiated between the 2 antifibrotic agents. Significant differences in IPF patient characteristics and access to antifibrotic therapies exist in EMPIRE countries, which needs further investigation and strategies to improve and harmonize patient care and therapy availability in this region.

Minimum manufacturing costs, national prices and estimated global availability of new repurposed therapies for COVID-19

Background Currently, only dexamethasone, tocilizumab and sarilumab have conclusively been shown to reduce mortality of COVID-19. Safe and effective treatments will need to be both affordable and widely available globally to be used alongside vaccination programmes. This analysis will estimate and compare potential generic minimum costs of a selection of approved COVID-19 drug candidates with available international list prices. Methods We searched for repurposed drugs that have been approved by at least one of the WHO, FDA or NICE, or at least given emergency use authorisation or recommended for off-label prescription. Drug prices were searched for, for dexamethasone, budesonide, baricitinib, tocilizumab, casirivimab and imdevimab, and sarilumab using active pharmaceutical ingredients (API) data extracted from global shipping records. This was compared with national pricing data from a range of low, medium, and high-income countries. Annual API export volumes from India were used to estimate the current availability of each drug. Results Repurposed therapies can be generically manufactured for some treatments at very low per-course costs, ranging from $2.58 for IV dexamethasone (or $0.19 orally) and $4.34 for inhaled budesonide. No export price data was available for baricitinib, tocilizumab, casirivimab and imdevimab or sarilumab, but courses of these treatments are priced highly, ranging from $6.67 for baricitinib to $875.5 for sarilumab. When comparing international list prices, we found wide variations between countries. Conclusions Successful management of COVID-19 will require equitable access to treatment for all populations, not just those able to pay high prices. Dexamethasone and budesonide are widely available and affordable, whilst monoclonal antibodies and IV treatment courses are more expensive.

Experiences and ways PLWHA deal with their lives

The issue of HIV and AIDS and people living with HIV and AIDS is very sensitive which needs great attention. The population of infected individuals seems not to seek help and health services due to their illness. The barriers which prevent this population experience the meaning of social construction of their illness. The main objective of this study is to understand the day-to-day lives and the ways PLWHA deals with their lives. Qualitative method and participatory action research were used to gather information. The study found out that PLWHA have much to fight for in the complex and frightening areas of HIV research and treatment. Though there have been lacking scientific skills, all PLWHA can bring unique experiences and perspectives to an open discussion. The study concluded that facing the challenges of working with HIV and AIDS–individuals; consider the participatory research approach because it can reach out to individuals, groups and organizations.It was recommended that the ultimate goals of AIDS treatment and research activism are to accelerate scientific research knowledge that contributes towards cure, and to win an early widespread access to treatment for everyone infected with HIV. Developing a well-designed research can help PLWHA learn how to ask themselves different questions and ask if the medical and research establishments will address their concerns. Keywords: Health Services;, seeking behavior; persons living with HIV.