Highlights from the 2018 IPPC
Mr. Jan M. Bult, President & CEO, PPTA, opened the well-attended IPPC 2018 by welcoming the more than 300 attendees to Budapest. PPTA thanks its sponsors who help make the IPPC possible every year.
Mr. David Bell, Chairman of the PPTA Global Board of Directors and Chief Innovation Officer, Grifols S.A., provided the Keynote address. Mr. Bell highlighted that over the past decades incredible developments have allowed people living with rare diseases to live more normal lives. Despite that, however, many patients still do not have access to the right treatment. PPTA’s mission is to promote the availability of and access to safe and effective plasma protein therapeutics for all patients in the world. He emphasized the need for plasma protein therapeutics worldwide continues to increase, but the geographical imbalance in plasma collection gives rise to potential disruptions of plasma supplies.
Session one featured a panel of speakers from Hungary, Klára Baróti-Tóth and Maria Judit Molnar, who gave respectively an overview of the blood transfusion service and a perspective of the current treatment practices and access situation for Immunoglobulins in Hungary.
The 2018 Hilfenhaus Award was presented to Alma Mater Professor Mauro Bernardi, MD of Bologna University, Italy. Professor Bernardi’s primary research focuses on Hepatology, with particular interests in cirrhosis, portal hypertension and related complications, hepatocellular carcinoma, chronic viral hepatitis, and liver transplantation.
Session two featured three panelists who examined various aspects of the global need for more plasma. Mr. Matthew Hotchko provided a comprehensive overview on global figures regarding changes and trends in the usage of the different plasma proteins worldwide. Dr. Graham Sher talked about plasma dynamics in Canada, highlighting Immunoglobulin supply and demand trends, stakeholder dynamics, and geopolitical landscape in the country. Mr. Jan M. Bult presented on global perspective and dynamics related to the need for more plasma around the world, addressing ways for potential regional and worldwide solutions to patient access.
Session three panel addressed the use of plasma proteins for the treatment of various pathological conditions. Prof. Richard Knight discussed uses of IVIG in neurological disease in the UK, highlighting the importance of correct diagnosis of neurological conditions and existing evidence supporting use of IVIG treatment. While IVIG is a recognized therapy for chronic inflammatory demyelinating polyradiculoneuropathy and multifocal motor neuropathy, the lack of therapeutic efficacy data exists for other neurological disorders. He voiced for wise use of IG therapies giving priority to patients who can benefit from it. Dr. Mark Brantly gave an overview of Alpha-1 Antitrypsin (AAT) Deficiency, describing the molecular, genetic, and clinical features of this disorder and presented data on beneficial effects of ATT therapy for patients improving their quality of life. Prof. Hans Peter Schwarz addressed the question “Plasma Proteins: Is the future behind us?” and elaborated on some new potential uses of plasma proteins in the future.
During the panel, Mr. Jan M. Bult presented the 2017 Otto Schwarz Award to Professor Hans Peter Schwarz, Independent Consultant; Former Member of the Board of BAXTER AG, Vienna Austria; Former VP R&D Baxter BioScience. Among his major achievements, he developed the first and only triple viral IGIV, which became the golden standard for the treatment of primary immunodeficiency.
Day one of the IPPC finished with a session focused on global awareness for plasma proteins. Mr. Alain Weill provided an overview on awareness of hemophilia in the world, including evolution of hemophilia care, different conditions of access to care worldwide, and WFH programs to increase awareness. Mr. Johan Prevot presented on the issue of under diagnosis of primary immunodeficiencies, discrepancies in access to treatment globally, and on IPOPI’s awareness programs. Mr. Jan M. Bult presented the new global initiative “How Is Your Day?” – Making the difference with plasma proteins, focusing on differentiation of these unique therapies from traditional pharmaceuticals and building awareness of the value they provide for people living with rare, life-threatening, chronic, and genetic diseases.
Day two of IPPC began with the Sponsors Session where Mr. Ruaidrí MacDomhnaill, REACHLaw, presented on REACH Authorization for chemical substances, providing some background information as well as describing the application process, exemptions, and timelines. Mr. Eric Youssef, Merck, talked about Triton® X-100, strategies for authorization and properties of the new Merck substitute agent for virus inactivation. Finally, Mr. Christian Fischer, Abbott Diagnostics provided an overview on Biotin interference in immunodiagnostic tests, highlighting the mechanism and relevant biotin concentrations, and the risk of interference on donor and patient management due to high consumptions of biotin through supplements.
Session six covered some regulatory aspects of the plasma protein therapeutics industry. Dr Karmin Saadat talked about the implementation of Risk-Based Approach (RBA) on inspections of blood establishments, outlining the legal background, the Risk Based Inspection (RBI) planning as well as the related tool and control measures. Dr. Brian Custer provided a comprehensive overview on MSM blood donation deferrals, with particular focus on the current status, global perspectives and future developments.
Before the start of the next panel, Alex Dowsett, who is a world-champion cyclist with hemophilia and who shared his story as part of the “How Is Your Day?” initiative, talked about his personal experience living with hemophilia, stressing the incredible role that plasma protein therapies played in his life.
Session seven provided a look at developments in a selection of BRICS countries: Brazil, China, and South Africa. Dr. Silvano Wendel presented on the Brazilian experience in supporting plasma self-sufficiency in developing countries, observing the major bottlenecks and obstacles in the country. Mr. Xiangjun Du talked about plasmapheresis in China, noting the main challenges regarding plasma collection and explaining his suggestions to governments, academia, and WHO, as well as to plasma centers and the Chinese plasma fractionation industry. Mr. Ravi Reddy described the South African experience in supporting plasma self-sufficiency in developing countries. His presentation showed the blood transfusion and blood donation processes in South Africa, highlighting the benefits of local fractionation to the country but also the challenges.
The final session of IPPC dealt with health technology assessments (HTA), particularly the value assessment of therapies to treat rare diseases and the approaches within different reimbursement systems and emerging frameworks. Prof. Matthias P. Schönermark presented on the German approach to the value assessment procedure, outlining both the benefits and challenges in the process in Germany for manufacturers as well as payers. Mrs. Sheela Upadhyaya then provided an insight into the UK approach to the procedure, describing the topic selection process and criteria, the elements and methods of the Highly Specialized Technology (HST) Program for rare diseases, as well as the role of patients and clinicians. Mrs. Carina Schey talked about the application of Multicriteria Decision Analysis (MCDA) to assess the value of rare disease therapies, illustrating the related method, criteria and framework.
Jan M. Bult, PPTA President & CEO, closed IPPC 2018, thanking PPTA staff, the speakers, and the audience. He also announced that the 2020 IPPC will be held in Berlin!!!