Highlights from IPPC 2016

Dr. Victor Grifols welcomed attendees to Barcelona and provided a personal overview of the evolution of Grifols. He recounted his family’s dedication and perseverance in times of adversity and opportunity.

Dr. Grifols was introduced by PPTA's Chairman of the Global Board of Directors, David Bell. Dr. Grifols commented that he strongly believed that Grifols needed to become a "member of PPTA to become an International company." He noted several important PPTA contributions to improving safety and quality of plasma collection and manufacturing; in particular: "Inventory Hold and Lookback, NDDR, IQPP and QSEAL." Dr. Grifols concluded, "PPTA doors are open for other companies to receive guidance to improve quality and safety of products."

Mr. David Bell, PPTA Chairman, introduced a new project by Grifols and its partners to help combat emerging pathogens around the world. The project was initiated by Victor Grifols in response to the Ebola outbreak in 2014 in West Africa. It is a self-contained, modular unit that can collect convalescent plasma, conduct viral inactivation, freeze and store plasma, and train local health care providers. The unit has arrived in Monrovia, Liberia and will begin operations soon to produce IVIG for the Liberian government and conduct a clinical trial to determine the efficacy of IVIG made from convalescent plasma.

Patrick Robert of the Marketing Research Bureau participated in the opening session and provided a comprehensive overview of the differences between the plasma therapeutics and pharma industries.

Session two: Ethical aspects delved into the complicated subjects of donor compensation and "ethical" labeling. This session featured presentations from leading ethicists and legal experts who examined common arguments against compensated plasma donation, recent initiatives in Europe to institute ethical labeling, and the precise definition of the word exploitation. Dr. James Taylor invoked the philosopher John Locke in exploring the definition of the word exploit. Dr. Stuart Youngner examined common arguments against compensating plasma donors including safety, crowding out, and donor exploitation. Dr. Youngner effectively countered these arguments, noting that in a well-regulated environment donor compensation is ethical and necessary to meet patient need. Finally, Cristiana Spontoni detailed recent initiatives in France and Italy to institute "ethical" labels on plasma therapies derived only from voluntary unpaid donations. She noted that these initiatives violate EU and WTO regulations. All concluded that compensated plasma donation was not only ethical but necessary to meet the needs of patients around the world. The presentations were followed by a lively discussion with members of the audience. There was agreement that the focus should be on the patients and ensuring access to safe, quality plasma protein therapies.

Professor Flora Peyvandi, of the University of Milan, presented the conclusions of the Study of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) Study, which is the first randomized clinical trial examining this issue. The study looked at inhibitor development in previously untreated patients under the age of six with plasma derived factor VIII and recombinant factor VIII. Prof. Peyvandi noted that patients in the study who used recombinant factor VIII were 87% more likely to develop inhibitors. This held true even when controlling for risk factors and different demographics. She noted that because inhibitor development is associated with increased morbidity and mortality, it is important to study and understand this relationship.

Professor Flora Peyvandi, of the University of Milan, was the 2016 Hilfenhaus Award winner. Prof. Peyvandi has a long history of research and engagement with rare hemostatic disorders. She received the 2016 Hilfenhaus Award in recognition of her many accomplishments in advancing understanding of rare bleeding disorders.

In Session four: Patient voice inclusion in the organization of care featured presentations from IPOPI/PLUS, ECPC, and AEDIP. Johan Prevot, Executive Director of IPOPI, spoke about the role PLUS has played in bringing together key stakeholders in the patient community. This has resulted in a stronger patient voice in the medical community, the European Commission, and national health commissioners. PLUS has also been a key participant in the development of a European Resource Network. Laura Miralles Paya from the Asociacion Espanola de Deficits Immunitarios Primarios AEDIP) discussed the challenges that PID patients face in Spain and the ways AEDIP is working to improve that including building awareness in the healthcare community and building government support for better patient treatment and support. Jana Pelouchova of the European Cancer Patient Coalition (ECPC), introduced RARECAREnet, a Europe-wide network to help patients facing rare cancers and the challenges associated with them including late or incorrect diagnosis and lack of clinical knowledge by healthcare providers. RARECAREnet provides an effective model for creating rare disease patient support networks.

Day two started with a sponsor session highlighting facility design services and batch traceability software. Sue Walker outlined the capability that MERCK offers in terms of designing small footprint plasma manufacturing facilities with hybrid technology. This capability could allow for local production of plasma therapies and help countries move toward self-sufficiency. Frans Nieuweboer of Goldfish ICT presented on their global batch traceability software, which integrates supply chain management from donation to end product.

The session on manufacturing (Session 6) featured a presentation by Daniel Fleta from Grifols Engineering who highlighted the role of Grifols Engineering and its pioneering equipment, which has advanced the quality and safety of plasma therapies across this industry. Of particular note is Grifols' Automatic Bottle Opener, Plasma Bag Opener, and Grifols Sterile Filling technology.

Professor Albert Farrugia provided a global perspective on contract fractionation. Prof. Farrugia covered the arguments for and against contract fractionation, noting that it can help address the issues of local market presence, local manufacturing capacity, and self-sufficiency. If implemented, he said, "contract fractionation demands a well-regulated system and sensitivity to political factors if it is to be effective."

Dr. Richard Schicho introduced Baxalta's new fractionation complex in Covington, Georgia. As a new facility, it incorporates the latest technologies, lessons learned, and design principles. The "complex" will conduct plasma fractionation, bulk manufacturing of albumin, and immunoglobulin manufacturing. It also has a technical operations building with a next generation pilot plant and laboratory and is equipped for scale-down operations.

Session 7: How to maintain and develop access to care focused on health technology assessments and registries in Europe. Irina Odnoletkova of PPTA started the session by examining health technology assessment (HTA) in hemophilia. She noted that HTAs play an important scientific role in the assessment of new medicine and treatments by policymakers but that they are executed inconsistently across Europe. 

The final session, Blood and plasma: Visions for a sustainable system, featured some of the foremost leaders in the industry. The panel was moderated by David Bell, Grifols, and featured Dr. Kari Aranko of the European Blood Alliance, Dr. Stephan Walsemann, Chairman of the European Plasma Alliance, Dr. Paul Strengers of the International Plasma Fractionation Association, and Jan M. Bult, President and CEO of PPTA.