Access to Care | Europe
Blood Directive (2002/98/EC)
The European plasma protein industry is a critical provider of life-saving therapies for patients affected by chronic, life-threatening and debilitating conditions. The production of these medicines is heavily influenced by the access to quality plasma as source material which is made possible by the constant contribution of qualified donors based in North America and Europe. The PPTA recognizes the importance to have regulations that sets standards for the collection, testing, processing, storage and distribution of plasma so that patients can access the safe treatments. The PPTA represented the interests of the plasma protein industry during the drafting and implementation of Directive 2002/98/EC setting the standards on quality and safety for the collection, testing, processing, storage and distribution of blood and blood components. The PPTA Health Policy Steering Committee continues to monitor the developments and implementation of this legislation and on related issues such as self-sufficiency, compensation and rationalization of product. This is done through direct advocacy with key European stakeholders such as Members of the European Parliament and representatives of the European Commission as well as with national competent authorities.
- Comments on the 2nd Report on Voluntary and Unpaid Donation of Blood and Blood Components (2011) to the European Commission
- Upcoming Report on the Promotion by Member States of Voluntary Unpaid Blood & Tissues and Cells Donations (2009) to the European Commission
- Requirements Introduced by Decree of August 19, 2005 (2006): to the Italian Medicine Agency (AIFA) and to the Italian Health Ministry
- Europe Needs to Collect More Plasma (2020)
- The PPTA Vision on the Plasma Protein Therapies Sector for the Next Decade in Europe (2014)
- PPTA Position on the Call for tender SANCO/2008/C6/012 on Testing Methods and Testing Laboratories (2008)
- The Intrinsic Differences Between Blood Products and Plasma Therapies (2007)
The European Institutions have been increasingly active in the field of rare diseases by either promulgating soft laws or by coordinating Member States activities. Although these actions are not legally binding for Member States, the European Union strongly encourages participation to the initiatives and as a result national policy is often influenced and follows EU policies. In 2008, for example the "Commission Communication on Rare Diseases: Europe's challenge" defines an EU-wide strategy to improve the visibility of rare diseases and coordinate Member States efforts with regards to activities in this area. In 2009, the Council of the European Union published its Recommendations on an action in the field of rare diseases including the creation and adoption of national plans for rare diseases. In 2011, the Council of the European Union adopted the directive on cross-border healthcare. This piece of legislation is unique for the rare disease community in the sense that it legally binds Member States to cooperate in the field of diagnosis and treatment capacity for rare diseases. As plasma protein therapies treat primarily rare diseases, the PPTA Health Policy Steering Committee's focus has been to advocate this fact to policy-makers and to stress the importance to maintain an adequate access to treatment for patients. In 2008, under the auspices of two Members of the European Parliament, two lunch-debates were organized. Further to this action of the two Parliamentarians supported the development by patients and physicians of a set of guidelines for the diagnosis and treatments of Primary Immunodeficiency.
- Improving Care for Rare Plasma Related Disorders (December 2008)
- Plasma products in the treatment of rare diseases (January 2008)
- European Commission's Public Consultation on Rare Diseases: Europe's Challenges (2008) to the European Commission
Health Technology Assessments
Health Technology Assessment (HTA) can be defined as a multi-disciplinary field of policy analysis intended to examine the medical, economic, social and ethical implications of the incremental value, diffusion and use of medical technology in healthcare. HTA is intended to bridge developments in medical research and the world of decision-making.
Economic evaluation of pharmaceuticals, medical devices or medical interventions consists of assessing the value of a novel technology compared to an existing one or an alternative treatment. This procedure is needed to support decisions on granting new medicinal products market access and reimbursement, and as such it is of great importance to various stakeholders such as patients, governmental agencies and industry.
Currently most European Member States have established agencies that evaluate the benefits of new technologies and these appraisals may have an impact on the granting of an official reimbursement price. The PPTA is working towards integrating the HTA dialogue by advocating that plasma protein therapies treat mostly rare, chronic and life threatening conditions. The PPTA is also following closely the developments of the EUnetHTA activities.
- On the Modalities of Stakeholder Consultation in the Voluntary Health Technology Assessment Network (2012)
Chronic Conditions policy
Typically chronic conditions are viewed as those conditions caused by lifestyle choices and therefore preventable. Policies for chronic conditions tend to focus on the prevention of the disease through healthier lifestyle. However, plasma protein therapies treat conditions that can be both acute and chronic. Patients living with a chronic plasma protein disorder such as, for example, as primary immunodeficiency or hemophilia, face similar issues as faced by patients with preventable chronic conditions like the need of continued access to therapies. These conditions have also an impact on the ability for the patient to conduct a "normal life". The PPTA has been bringing this particular point to the attention of policy-makers and regulators.
PPTA works on patient access issues at both the European Union and Member State levels. A Health Policy Steering Committee engages with the European Parliament, the European Commission and Member States Permanent Representations based in Brussels on legislative and policy issues. PPTA Europe also works actively in the Member States below and in other countries as needed.
PPTA collaborates on matters of common interest with Pharmig, the Austrian Trade Association including Good Manufacturing Practices Annex 14 and the inclusion of doping warning into package leaflets for plasma protein therapies.
- PPTA Letter on Clinical Rationale for Antithrombin Concentrate in Acquired Antithrombin Deficiencies
The PPTA Belgian working group has been very active on matters on market access. Notably, the group liaised with government officials to be granted equal tax exemptions as the not-for profit sector. Furthermore, the PPTA Belgian working group provided considerable input during the preparation by the KCE, the Belgian Healthcare Knowledge Centre, of a report on "How to ensure self-sufficiency of stable plasma-derivatives in Belgium". The Belgian working group is working towards improving level-playing field on the Belgian market with regards to the national fractionator.
- KCE Report (PPTA contributed and is acknowledged as source of information in the document) (2009)
- Final Letter to Onkelinx Price Decrease March 19 (2009)
- Letter to Minister Rudy Demotte, Minister van Sociale zaken en Volksgezondheid and Xavier de Cuyper, Federaal Agentschap voor Geneesmiddelen en Gezondheidsproducten. (2007)
- Letter to Monsieur Rudy Demotte, Ministre de la Santé Publique et des Affaires Sociales. (2007)
PPTA Deutschland was established as a non-profit special interest group in late 2008 to address the revision of albumin guidelines. Since then, they have taken a leadership role in advocating for patient access among stakeholders including policymakers, government agencies, regulatory authorities, patient organizations and medical associations. PPTA Deutschland monitors legislative and policy developments related to plasma donation, public health, rare diseases, pricing and reimbursement, innovation and technology. In addition, PPTA Deutschland is an active member of the FIND-ID network, a physician-based network that seeks to raise awareness, increase the diagnosis rate and improve the quality of life of patients with primary immune deficiency (PI) in Germany.
- Position Paper on Review of legal Manufacturer Rebates (2011)
- Cross Sectional Guidelines - Revision Chapter 5 Human Albumin (2011)
- Letter sent to BfArM (2011)
PPTA members have been working in France since 2004; the main objective of their work was to improve market access, more specifically by focusing on article 5121-11 of the French health code which provides only a two-year marketing authorization for plasma-derived products made from compensated plasma. Since then PPTA France has registered as an official association under the French law of 1901. Although, the focus of the group has remained on market access, it has also expanded to focus on building an official stakeholder network targeting patient groups, physicians, government officials, Members of the French Parliament and Senate. In this context, the group organized several roundtables to discuss issues of interest to both patients, physicians and the industry such as patients' product choice, the French Rare Disease National Plan and patient access to therapies.
The PPTA Dutch Working group was established to interface with government officials with an objective to level the playing field in the Netherlands, a market primarily dominated by the national fractionator. The group has also addressed issues related to official reimbursement. Since 2009, the Dutch government has been investigating the national blood and plasma collection system and their use in the supply chain for the manufacturing of plasma-derived treatments. These reports confirmed concerns with the national fractionator who operates as both a public and private entity. PPTA worked withconsultants and government officials on this issue.
- Ministerial Plan for Blood Supplies, 2012-2014 (2012)
- Reply Minister of Health regarding ConQuaestor Report to the Parliament (2012)
- PPTA Press Release on Conquaestor Report (2011)
- PPTA Letter to the Minister Following the Plexus Report (2010)
In 2005, a UK Group was established with the primary goal to ensure patient access to IVIG. The UK group had been particularly active in liaising with the authorities during the preparation and implementation of the UK Demand Management Plan and Database for the supply of immunoglobulin.