Maarten Van Baelen

Since its first use to treat primary immunodeficiency (PID) in 1952 human Normal Polyvalent Immunoglobulins (IgGs) have vastly expanded therapeutic options in the previously untreatable and/or fatal diseases and conditions that either require compensation of significant immune
deficiency or (immuno) modulation of an aberrant immunological homeostasis. The value of these treatments must not be underestimated, as they can prevent premature death, halt disease progression, minimise… Show more

Since its first use to treat primary immunodeficiency (PID) in 1952 human Normal Polyvalent Immunoglobulins (IgGs) have vastly expanded therapeutic options in the previously untreatable and/or fatal diseases and conditions that either require compensation of significant immune
deficiency or (immuno) modulation of an aberrant immunological homeostasis. The value of these treatments must not be underestimated, as they can prevent premature death, halt disease progression, minimise disabilities, and promote patients’ quality of life, thereby having high clinical, societal, and economic impact. A good example of this high impact is the survival rateof patients with common variable immune deficiency (CVID), which was only 22 years in 1979, whilst now the IgGs have increased it significantly and improved overall quality of life. From a socio-economic perspective, in PID, affecting 44,000 patients across Europe, this translates into ~1bnEur/year health gains and almost 1bnEur/ year avoidable healthcare costs.

However, whilst the therapeutic value of IgGs is evident, the mechanisms involved are not entirely understood, specifically in the case of immunomodulation in autoimmune diseases. This, as well as regulatory and policy issues, has led to differentiation of the indications into those approved by EMA (on-label) and those used off-label. This categorisation varies greatly across European countries, leading to different care standards and treatment protocols. Additionally, the COVID-19 pandemic
has led to disruption in plasma collection and consequently caused focal challenges in access to plasma derived medicinal products (incl. IgGs). In most European countries, limited availability of plasma and plasma derived medicinal products (actual or anticipated) have triggered further restrictions on IgG use via prioritisation of specific patient subpopulations. Show less

This paper aims to analyse and demonstrate the unique nature and value of PDMPs (Plasma-derived Medicinal Products) across clinical, economic, and societal dimensions, and focuses on improving Patient Access. Patient Access is viewed from two angles: formal access based on reimbursement coverage, and therapeutic access based on the availability of an optimal treatment paradigm. It also analyses key challenges that affect the full realisation of the value of PDMPs. Finally, it offers a… Show more

This paper aims to analyse and demonstrate the unique nature and value of PDMPs (Plasma-derived Medicinal Products) across clinical, economic, and societal dimensions, and focuses on improving Patient Access. Patient Access is viewed from two angles: formal access based on reimbursement coverage, and therapeutic access based on the availability of an optimal treatment paradigm. It also analyses key challenges that affect the full realisation of the value of PDMPs. Finally, it offers a comprehensive view of possible solutions to the identified challenges.

PDMPs are unique biological therapies derived from human plasma and are used to treat patients with rare, often genetic conditions with a high disease burden. Despite decades of effective therapeutic use in Europe, and demonstrable clinical and societal value, these treatments still face numerous Patient Access challenges pertaining to the plasma donation landscape, regulatory and reimbursement frameworks, and treatment paradigms. There is a growing clinical need of European patients for PDMPs, and considerably more plasma must be collected in Europe. As new indications arise more patients are diagnosed with diseases requiring PDMP treatment. Even when diagnosed and if therapy is available, patients often are denied adequate PDMP treatment because of therapeutic and formal Patient Access challenges. To overcome these challenges, it is necessary to form close and trustbased partnerships between industry and all healthcare stakeholders. Show less

This consensus information on biosimilar medicinal products was drafted by and for patients
together with representatives of the European Medicines Agency, the European Commission and
their stakeholders [the European Patients Forum (EPF), the European Federation of Crohn's &
Ulcerative Colitis Associations (EFCCA), the Standing Committee of European Doctors, European
Federation of Pharmaceutical Industries and Associations (EFPIA), European Association for… Show more

This consensus information on biosimilar medicinal products was drafted by and for patients
together with representatives of the European Medicines Agency, the European Commission and
their stakeholders [the European Patients Forum (EPF), the European Federation of Crohn's &
Ulcerative Colitis Associations (EFCCA), the Standing Committee of European Doctors, European
Federation of Pharmaceutical Industries and Associations (EFPIA), European Association for Bioindustries
(EuropaBio) and Medicines for Europe]. Show less

Generic medicines have proven to be pivotal in increasing patient access and preventing medicine shortages through increased choice and availability of treatments. However, generic medicines policies have recently focused exclusively on reducing prices thereby increasing the risk of medicine shortages.

The sustainability of healthcare budgets in Europe has been intensely pressured by multiple factors, such as a growing and ageing population, an increased disease burden, the introduction… Show more

Generic medicines have proven to be pivotal in increasing patient access and preventing medicine shortages through increased choice and availability of treatments. However, generic medicines policies have recently focused exclusively on reducing prices thereby increasing the risk of medicine shortages.

The sustainability of healthcare budgets in Europe has been intensely pressured by multiple factors, such as a growing and ageing population, an increased disease burden, the introduction and increased cost of new innovative medicines. To overcome these challenges, several national authorities have adopted austerity measures and applied short-term cost-containment measures to pharmaceuticals, including to generic medicines despite their low cost (around 4% of total healthcare expenditure in Europe) and their relevance for care (62% of medicines dispensed today in Europe are generic medicines). Short-term cost-cutting measures, such as ad-hoc price cuts, external reference pricing, payback, tendering, etc. have driven the prices of some off-patent medicines to unsustainably low levels. This causes manufacturers of generic medicines to withdraw from the market, resulting in the increased risk of medicine shortages.

The evidence now shows that many national markets across Europe are relying on too few suppliers for essential life-saving medicines in both the hospital and ambulatory sectors. Tackling medicines shortages in a multi-source context, requires an approach that addresses both the root causes of medicines shortages and mitigates medicine shortages once they occur. Addressing the economic causes of shortages to ensure market predictability and competition, improving regulatory efficiency, improving transparency in supply chain and a proper and controlled communication are important measures to tackle medicine shortages.
Show less

Based on the paramount objective of protecting human health, Directive 2011/62/EU (amending Directive 2001/83/EC) introduces provisions which aim to prevent the entry of falsified medicinal products into the legal supply chain.
Directive 2011/62/EU states that the number of medicinal products that have been detected as being falsified concerning their identity, history, and source has had an alarming increase in the European Union (“EU”). Also, according to Directive 2011/62/EU, falsified… Show more

Based on the paramount objective of protecting human health, Directive 2011/62/EU (amending Directive 2001/83/EC) introduces provisions which aim to prevent the entry of falsified medicinal products into the legal supply chain.
Directive 2011/62/EU states that the number of medicinal products that have been detected as being falsified concerning their identity, history, and source has had an alarming increase in the European Union (“EU”). Also, according to Directive 2011/62/EU, falsified medicinal products reach patients not only through illegal means but also through the legal supply chain (which could cause additionally the creation of a lack of trust among patients in the legal supply chain).
By way of preliminary remark, it should be noted that Directive 2011/62/EU refrains from stating that the legal supply chain in the EU is concerned by an alarming increase of falsified medicinal products. Indeed, the vast majority of falsified medicinal products are currently still marketed outside the legal supply chain (something which Directive 2011/62/EU does not address).
This legal analysis is limited to an assessment of the different policy options considered by the Commission in relation to the system of safety features laid down in Directive 2011/62/EU (consisting of the so-called “unique identifier” and the “anti-tampering device”), the specifics in relation to which are to be determined by the Commission on the basis of a (number of) delegated act(s). Show less

According to the European Commission, the threat to public health and safety from falsified medicinal products is on the rise. The impact assessment (2008) of the European Commission claims that by 2020, 0.05% of all prescription medicinal products dispensed through the legal supply
chain will have been counterfeit products. In order to control and combat the possible threat, the EC has introduced new legislation to put in place preventive measures to improve the protection of public… Show more

According to the European Commission, the threat to public health and safety from falsified medicinal products is on the rise. The impact assessment (2008) of the European Commission claims that by 2020, 0.05% of all prescription medicinal products dispensed through the legal supply
chain will have been counterfeit products. In order to control and combat the possible threat, the EC has introduced new legislation to put in place preventive measures to improve the protection of public health. The basis for this new legislation was defined in the Directive 2011/62/EU. KPMG has conducted an independent study requested by the European Generic medicines Association
‘EGA’ and has concluded that with the implementation of safety features a possible risk of failure and a risk of exceeding the budgeted time and costs can arise. Show less