Mitigating the Nocebo Effect in Biosimilar Use and Switching: A Systematic Review.

Background: In the context of biosimilar use and switching, a patient's negative perception of a biosimilar might trigger a nocebo effect, where negative expectations unrelated to its pharmacologic action may lead to worsened symptoms, resulting in less perceived therapeutic benefit or worsened adverse events. Considering the crucial role biosimilars have in reducing health care costs and expanding access to safe biologics globally, it is essential to develop and implement effective strategies to mitigate the possible occurrence of such nocebo effects.

Objective: The aim of this systematic literature review was two-fold: (i) to review strategies that have been applied and/or tested for minimising the nocebo effect in clinical practice, within and outside the context of biosimilar switching, and (ii) to propose recommendations on useful mitigation strategies to minimise the occurrence of the nocebo effect in the context of biosimilar switching.

Can Endangered Biosimilar Markets be Rescued? The Need to Bridge Competing Interests for Long-Term Gain.

Market signals such as: (1) the limited number of biosimilars in the development pipeline, (2) the focus of biosimilar development on high-profit therapeutic areas only, and (3) the increase in the number of biosimilar discontinuations and withdrawals, are indicative of sustainability threats facing biosimilar markets in Europe. Two prominent factors that undermine sustainability are: competing interests between the various stakeholders and a preferential focus on short-term gains, disregarding future sustainability threats, hence the need for effective policies that create sustainable competition in biologic markets. Thus far, measures implemented to foster biosimilar adoption have not been necessarily complied with and have had mixed success.

Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease.

Pompe disease is a lysosomal storage disorder caused by deficiency of acid alpha-glucosidase (GAA), resulting in glycogen accumulation with profound pathology in skeletal muscle. We recently developed an optimized form of lentiviral gene therapy for Pompe disease in which a codon-optimized version of the GAA transgene (LV-GAAco) was fused to an insulin-like growth factor 2 (IGF2) peptide (LV-IGF2.GAAco), to promote cellular uptake via the cation-independent mannose-6-phosphate/IGF2 receptor.

Leveraging the holistic benefits of biosimilars in Europe - part 2: how payers can safeguard the future of a healthy biosimilar market environment.

Introduction: Biosimilars have improved access to biologic medicines; however, historical thinking may jeopardize the viability of future markets.

Areas Covered: An expert panel of eight diverse European stakeholders provided insights about rethinking biosimilars and cost-savings, reducing patient access inequalities, increasing inter-market equity, and improving education. The insights reported here (Part 2) follow a study that provides perspectives on leveraging the holistic benefits of biosimilars for market sustainability based on independent survey results and telephone interviews of stakeholders from diverse biosimilar markets (Part 1).

Capturing the holistic value of biosimilars in Europe - part 1: a historical perspective.

Introduction: Approved biosimilars exhibit comparable efficacy, safety, and immunogenicity to reference products. This report provides perspectives on the societal value of biosimilars within Europe and potential factors that have influenced market dynamics.

Methods: An independent, self-administered survey or one-on-one in-depth interview was used to collect viewpoints about the impact of biosimilar medicines within European markets.

The impact of policy interventions to promote the uptake of biosimilar medicines in Belgium: a nationwide interrupted time series analysis.

Background: The Belgian government has taken several measures to increase the uptake of biosimilars in past years. However, no formal evaluation of the impact of these measures has been made yet. This study aimed to investigate the impact of the implemented measures on biosimilar uptake.

Points to consider for cost-effective use of biological and targeted synthetic DMARDs in inflammatory rheumatic diseases: results from an umbrella review and international Delphi study.

Objectives: To develop evidence-based points to consider for cost-effective use of biological and targeted synthetic disease-modifying antirheumatic drugs (b/tsDMARDs) in the treatment of inflammatory rheumatic diseases, specifically rheumatoid arthritis, psoriatic arthritis and axial spondyloarthritis.

Methods: Following EULAR procedures, an international task force was formed, consisting of 13 experts in rheumatology, epidemiology and pharmacology from seven European countries. Twelve strategies for cost-effective use of b/tsDMARDs were identified through individual and group discussion.

An exploration of biosimilar TNF-alpha inhibitors uptake determinants in hospital environments in Italy, Portugal, and Spain.

Background: The availability of biosimilar medicines in Southern European markets has allowed purchasing biologics at a lower cost for healthcare systems. However, the capacity to seize this cost-reduction opportunity in the long run depends on fostering a sustainable competitive environment for all the market players involved. Diverse policies and information campaigns have been launched in Italy, Portugal and Spain to support uptake of "best-value" biologics (BVB).

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy.

Pompe disease is caused by deficiency of acid α-glucosidase (GAA), resulting in glycogen accumulation in various tissues, including cardiac and skeletal muscles and the central nervous system (CNS). Enzyme replacement therapy (ERT) improves cardiac, motor, and respiratory functions but is limited by poor cellular uptake and its inability to cross the blood-brain barrier. Previously, we showed that hematopoietic stem cell (HSPC)-mediated lentiviral gene therapy (LVGT) with codon-optimized (LV-) caused glycogen reduction in heart, skeletal muscles, and partially in the brain at high vector copy number (VCN).

Determinants of prescribing decisions for off-patent biological medicines in Belgium: a qualitative study.

Background: A competitive market for off-patent biologicals leads to more affordable and high-quality healthcare. In recent years, Belgium has been characterized by its low use of biosimilars and by its shifts from off-patent biologicals toward new alternative therapies. Yet, the prescribing decisions involved in these observations are poorly understood.

How to select a best-value biological medicine? A practical model to support hospital pharmacists.

Purpose: With the growing availability of biosimilars on the global market, clinicians and pharmacists have multiple off-patent biological products to choose from. Besides the competitiveness of the product's price, other criteria should be considered when selecting a best-value biological. This article aims to provide a model to facilitate transparent best-value biological selection in the off-patent biological medicines segment.

Applications of Behavioral Economics to Pharmaceutical Policymaking: A Scoping Review with Implications for Best-Value Biological Medicines.

Background And Objective: Pharmaceutical policies are generally based on the assumption that involved stakeholders make rational decisions. However, behavioral economics has taught us that this is not always the case as people deviate from rational behavior in rather predictable patterns. This scoping review examined to what extent behavioral concepts have already been applied in the pharmaceutical domain and what evidence exists about their effectiveness, with the aim of formulating future applications and research hypotheses on policymaking for best-value biologicals.

Biosimilar Use and Switching in Belgium: Avenues for Integrated Policymaking.

By improving the affordability and accessibility of biologicals, biosimilar competition provides important benefits to healthcare systems and patients. In Belgium, biosimilar uptake and competition is limited compared to other European markets. Whereas other countries have initiated structured biosimilar introduction or switching plans, no such framework or guiding principles are yet available in Belgium.

Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.

Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a lysosomal storage disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). ERT has shown to be lifesaving in patients with classic infantile Pompe disease. However, a major drawback is the development of neutralizing antibodies against ERT.

Regulatory Information and Guidance on Biosimilars and Their Use Across Europe: A Call for Strengthened One Voice Messaging.

Background: Beyond evaluation and approval, European and national regulators have a key role in providing reliable information on biosimilars and the science underpinning their development, approval, and use.

Objectives: This study aims to (i) review biosimilar information and guidance provided by EMA and national medicines agencies and (ii) explore stakeholder perspectives on the role of regulators in enabling acceptance and use of biosimilars.

Methods: This study consists of (i) a comparative review of regulatory information and position statements across medicine agencies ( = 32) and (ii) qualitative interviews with stakeholders in Europe ( = 14).

Qualitative Analysis of the Design and Implementation of Benefit-Sharing Programs for Biologics Across Europe.

Background: To encourage the rational prescribing of biologics, payers across Europe have experimented with the implementation of benefit-sharing programs. Benefit-sharing programs are incentive programs that promote the use of 'best-value' off-patent biologics and biosimilars by driving changes in prescribing practices. The aim of these programs is to generate savings that can be shared among stakeholders involved (e.

Perceptions About Biosimilar Medicines Among Belgian Patients in the Ambulatory Care.

Biosimilar medicines have been on the European market for 15 years. Despite the extensive and positive experience with biosimilars across Europe, their uptake remains limited in Belgium. One of the possible factors limiting uptake in clinical practice is the inadequate understanding and lack of trust in biosimilars among patients.

Availability of age-appropriate paediatric formulations in the Netherlands: the need in daily clinical practice remains.

Objectives: To quantify the availability of authorised, age-appropriate paediatric medicines in clinical practice in the Netherlands and to identify gaps by assessing dispensing practice in a paediatric hospital.

Methods: The availability of age-appropriate formulations was assessed by conducting a survey on the use of pharmacy compounded medicines among the paediatric hospitals in the Netherlands, and by analysing dispensing data of oral medication from the inpatient pharmacy of the largest paediatric hospital in the Netherlands. The age-appropriateness of the dispensed formulations was assessed on two aspects: dose-capability and acceptability.