Discoveries and Developments: Highlights of the Plasma Protein Forum (June 2013)

The 2013 Plasma Protein Forum was held in Reston, Virginia on June 11-12.

The conference was opened by PPTA President and CEO, Jan M. Bult who expressed the need for industry to meet needs of patients worldwide. To do so, Bult stressed the importance of working with the not-for-profit sector, stating that, “We have more commonalities than differences,” and that “peaceful co-existence” will benefit patients.

In his first address to the membership as Chairman of the Global Board, Paolo Marcucci set forth his vision, “We do something amazing and unique and we should be bold and ambitious in providing treatment for all on any continent, in any country.” Marcucci also applauded the Association for its efforts on behalf of the IVIG Access Act, launching International Plasma Awareness Week, and succeeding in having its research published in peer review journals.


The Orphan Drug Act

The 30th Anniversary of the Orphan Drug Act (ODA) served as a backdrop for a conference program that focused on rare diseases. In the keynote address, Stephen Groft, Pharm.D., Director, Office of Rare Diseases Research, National Institutes of Health (NIH) provided an overview of the ODA’s impact on rare disease populations which constitute about 6-8% of the world’s population. Dr. Groft noted that NIH has a global approach and actively seeks global collaboration with research partners including patient advocacy groups, the private sector and academics. He devoted time to the NIH infrastructure, how to identify program managers, grant opportunities and incentives for research. Groft discussed the value in patient registries, the human genome sequencing and emerging developments in nanotechnology all of which are advancing knowledge regarding rare diseases. Under the auspices of the ODA, NIH seeks to develop an additional 200 diagnostics by 2020.

Other panelists provided industry and patient perspectives on the ODA, noting that it was a landmark piece of legislation that contributed significantly to the development of therapies. While recognizing its many benefits, suggestions for improvements were put forth such as strengthening orphan drug policy, including increasing the rare disease threshold from 200,000 to a percentage of total population, and providing regulatory predictability by having open dialogue for post market studies early in the development/approval process. Further, several leading biologics and pharmaceutical trade associations agree that delinking orphan drug designation from clinical superiority is sound public health policy because the intent of designation is to broadly encourage manufacturers to explore developing therapies for unmet medical needs.


Perspectives from Patient Organizations

Since the ODA was signed into law in 1983, there have been 430 orphan products approved. According to the National Organization of Rare Disorders, (NORD) there are over 7000 rare diseases, 80% which affect children and 27% of those who die before their first birthday. Despite progress, there are thousands of orphan diseases for which there is no treatment available. Patient advocates articulated concerns regarding imminent challenges such as the impact of sequestration on the Food and Drug Administration Safety and Innovation Act (FDAISA), the pharmaceutical excise tax, fail first policies, specialty tiers and off label use of therapies, Essential Health Benefits and the fact that Food and Drug Administration (FDA) approval does not translate into reimbursement. In spite of these concerns patient advocates are encouraged by collaboration among stakeholders, the research, development and regulatory communities and the commitment of industry. It was suggested that the key to future success will be in “finding balance among innovation, sustainability, access and affordability.” J. Russell Teagarden, M.D., NORD’s Senior Vice President of Medical and Scientific Affairs provided humanistic perspectives on illness, disease and sickness, stating that, “Illness is part of the human condition,” and that the rare disease community is particularly vulnerable to the utilitarian philosophy of “the greatest good for the greatest number.” Teagarden argued that conventional perceptions and established protocols for clinical trials to not accommodate rare diseases and suggested that alternative value determinations must be identified.

Patient advocacy representatives also highlighted the need to include patients as decision-shapers in outcomes research, discussed the Patient-Centered Outcomes Research Institute (PCORI) and that that A-PLUS, NORD and other organizations objected to PCORI’s plan for single rare disease advisory panel, and succeeded in including in the legislation a provision to convene disease specific ad hoc panels to complement research. Panelists from the hemophilia, immune deficiency and alpha-1 communities shared meaningful insights into their histories, and current policies and research concerns.


Rare Disease Advocacy Strategies in Developing Countries

A panel discussion on patient access in developing countries proved to be a Forum highlight. As the Association and industry seek to expand diagnosis and treatment, throughout the world, it was instructive to listen to the practical experiences of experts working across the globe, with a focus on Africa and Latin America. Over 80% of the world’s population lives in low resource countries and have limited or no access to plasma protein therapies. The panel discussed how to identify local leaders and develop advocacy strategies. Developing societies are often hierarchical in nature and successful leaders must be individuals who are willing to challenge the status quo, question authority, believe in themselves, represent their people and most importantly, act. Barriers to success include disease, politics, logistics, and cultural norms all of which may result in a decade or more to create an effective hemophilia patient organization and advocacy success. In many countries there is no effective regulatory authority and the inability of local hospitals to meet demand poses particular challenges. There was agreement among the panel that developing solutions specific to the political, economic and cultural climate of developing countries are essential if they are to be sustainable. In Latin America, hemophilia organizations work to develop national plans and are increasingly relying on Quality of Life (QALY) models to bolster their positions.

It was also reported that South Africa is the only country that has fractionation facilities but only produces enough for its own needs.


Donor Safety

A pre-conference workshop: “Industry’s Commitment to Safe and Healthy Donors” was described by Shinji Wada, Chair, Source Board of Directors as “a monumental success.” The workshop covered a range of donor issues including profiling today’s centers and donors, reviewing donor eligibility requirements, exploring literature on donor safety and managing donor adverse reactions. The workshop highlighted the contributions of the Medical Policy Committee in drafting uniform definitions for documenting donor reactions.

On Wednesday, Dorothy E. Scott, M.D., Chief, Laboratory of Plasma Derivatives, Office of Blood Research and Review, Center for Biologics Evaluation and Research, Food and Drug Administration addressed the conference assembly on the long recognized complication of hemolysis resulting from immunoglobulin infusion. Risk factors from anti-A and anti-B antibodies may result in severe anemia, renal failure and other complications and affect both men and women equally.

A draft concept for a PPTA/FDA sponsored regulatory workshop to be held in January 2014 was presented. The workshop will examine epidemiology, pathogenesis and patient and product related risk factors and manufacturing and risk mitigation strategies.

In a broader effort to address donor hemovigilance, the final panel discussion set forth an imperative for standardizing donor adverse event definitions. Panelists provided an overview of the government’s efforts to standardize reporting and definition and willingness to share programs,. industry’s commitment to and efforts on behalf of and the complexities involved in gathering aggregate data from companies. Descriptive terms such as severe and moderate work well within individual companies because everyone accepts the same definition. Among various companies, however the descriptions may differ widely and be subject to interpretation. In order to move towards a unified PlasmaVigilance effort, the Medical Policy Steering Committee has been working to create standardized donor adverse event definitions that would be uniformly be used by all. This will facilitate quality aggregate data collection that supports research and analysis.

This year’s Forum was a vibrant event well attended by patient group representatives, industry, regulators and academics. The event provided substantive and thoughtful discussions while highlighting key challenges facing patient access to plasma protein therapies.

Please Save the Date for the 2014 Plasma Protein Forum to be held in Washington, D.C. June 26-27.  






















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