November 20, 2012

Dr. Kouki Takahashi, Leader, Blood Services Section, Pharmaceutical Affairs and FoodSanitation Council
Dr. Makoto Handa, Chairperson, Blood Services Section Steering Committee
Dr. Masami Kato, Manager, Blood and Blood Products Division, Pharmaceutical andFood Safety Bureau, Ministry of Health, Labour and Welfare

Position Paper concerning “Basic Policy for Improvement of the Safety ofBlood Products and Securing Stable Supplies”

Introduction
We have shown the challenges faced by the Japanese plasma derivative business and theircountermeasures in the proposal 1) concerning the “State of Plasma derivative business inJapan,” submitted on September 9, 2011 to the Blood Services Section of the Pharmaceutical
Affairs and Food Sanitation Council and the Blood and Blood Products Division,
Pharmaceutical and Food Safety Bureau, MHLW. We have also submitted an opinion paper
concerning the “Review Committee of the State of Supply of Plasma Derivatives: Final
Report” 2) on July 23, 2012, presenting specific opinions and countermeasures based on thecontent of the committee’s final report. At the same time, we noted the discrepancy between
the documentation quoted as an indication of citizens’ and blood donors wishes concerning
blood services 3) 4), and the outcome of our own studies of citizens and donors, and, presented
our report of the study outcomes 5).
We have pointed out shortcomings in the Japanese blood services policy’s information
sharing with citizens, blood donors, patients, healthcare professionals and service providers,and as part of our efforts to improve the said situation, hosted a seminar on blood plasma
manufacture for the media on July 23, 2012, where patients, clinicians and service providers
met under one roof to exchange views with the media. As a result, this event was reported in

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the media in a simple article stating the citizens’ point of view of the current challenges forplasma derivative services.
The social and healthcare environments that surround plasma derivatives are now changing
considerably, while R&D in plasma derivatives and gene recombination products
manufactured with the latest technology have advanced both in Japan and overseas. We
believe that we have to urgently review the current issues of plasma derivative business
policies, from the viewpoints of citizens, patients and donors.
Following the revision of the basic policy in the title, an MHLW-proposed revision waspresented to the Blood Services Section Steering Committee. This proposed basic policy
directs the government policies on blood services for the next 5 years. However, it wasunfortunately difficult to see any evidence of the issues and solutions we had included in that
proposal. In addition, we could not grasp the details of the discussions that precluded theformulation of the proposal. Therefore we have decided to submit this document to request
more specific discussions with the blood service providers.

Gist of our requests
The plasma derivatives and blood substitutes gene recombination products we supply makeup 41% of Japan’s plasma derivative market, based on drug price. These include uniquepreparations in certain classes or with specific effects used to treat orphan diseases such as
Factor VII deficiency, thrombasthenia, Factor XIII deficiency, hereditary angioedema andinhibitor-induced hemophilia, etc. We also supply gene recombinant Factor VIII used inhemophilia A treatment in Japan. We are proud of our sizable contributions to the Japaneseplasma derivative business, including the treatment of orphan diseases.
Furthermore, reviews of yet to be approved or off-label use drugs with high clinical needs
strongly requested by clinicians’ societies are ongoing at the government sponsored “ReviewCouncil On Yet To Be Approved or Off-Label Drugs With High Clinical Needs,” including
blood products: three yet-to-be-approved drugs and two off-label use drugs that concern us.
We endeavor to eliminate the drug lag so that Japanese patients can have access to the latest
treatment. In addition, we are preparing for the early introduction of next generation generecombinant coagulation factor substitutes, expected to improve the QOL of hemophiliac
patients, to the Japanese market.

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We consider it our duty to take part in the discussions on building a framework of sustainable
and stable future supply of safe and efficacious drugs from the patients’ point of view.
That is why we make the following two requests on the proposed basic policy.
1.

To clearly state, “Specific review of the exclusion of plasma derivatives from thoselisted as subject of the provisions of the Export Trade Control Order shall commence.”

2.

To clearly state, “As a specific measure to secure domestic self-sufficiency and a stablesupply, as stated in The Blood Act (Act on Securing a Stable Supply of Safe BloodProducts), fundamental review, such as the introduction of a new blood collection
organization specialized in harvesting source plasma for plasma derivatives, in addition
to the current blood collection organization, which is a single company, shallcommence.”3Details of our requests
1.

To clearly state, “Specific review shall commence concerning the exclusion of plasma
derivatives from those listed as items subject to the provisions of the Export Trade
Control Order”<Main reasons>i.

There has been the situation that part of the active blood components obtained
through blood donation becomes excessive and is not effectively used, because of
the increased supply of gene recombination products and some products that cannot
be produced with the manufacturing technologies available to the domestic service
provider, etc. By exporting the excess blood components and products that are notneeded in Japan to overseas, will be significant to patients who have no current
access to the products or sufficient treatments, also in terms of the international
contribution our donors make, as well as from the viewpoint of promoting effective
use of donated blood and ensuring the level of manufacturing capacity of thedomestic service provider. Therefore, plasma derivatives have to be excluded fromthe list of subjects of the said Export Trade Control Order.

ii. The backdrop of listing of plasma derivatives in the 1966 Export Trade Control
Order was the then controversy surrounding the alleged military use of citizens’
blood for the Vietnam War and other conflicts, which raised an ethical issue,
leading to their inclusion in the said list of Control Order items. The current social
environment is considerably different from those times, and current Japanese blood
services are already implemented under the State’s management. It is hard toimagine that such an issue would arise again.
2.

To be specific, “As a specific measure to secure domestic self-sufficiency and a stablesupply, as stated in The Blood Act, a fundamental review, such as the introduction of a
new blood collection organization specialized in harvesting source plasma for plasma
derivatives, in addition to the current blood collection organization, which is a singlecompany, shall commence.”<Main reasons>i.

It has been pointed out that the low birth rate trend and population aging will leadto future shortage of, not only the blood components for transfusion, but also thesource plasma for plasma derivatives.6) We need fundamental discussions.
4ii. The domestic “self-sufficiency” stated in The Blood Act does not require thatdomestic demands should be supplied by products manufactured by domestic
manufacturers, but that the demands will be met with products manufactured fromblood donated within the country. At present, there is a demand for 1,500,000 L.
We believe that it is necessary to secure further stock of some hundreds ofthousands of liters of extra source plasma all the time, as a risk mitigation measure
to secure a stable supply during a crisis.
iii. One blood collection organization collects the blood as a source of blood products
for transfusion and of plasma derivatives. This approach poses an issue for a stablesupply at normal times or in a crisis. We need to avoid the situation whereby ashortage of the source of blood components for transfusion coincides with ashortage of source plasma for plasma derivatives by taking risk mitigation measures.
iv. One approach to dramatically improve the domestic self-sufficiency is to export anequivalent amount of the source plasma collected by domestic blood donation as
the currently imported products for processing at overseas plasma derivative
business plants. The manufactured derivatives are then re-imported for domestic
use. In short, by replacing the source plasma for imported derivative products withdomestic sources, the purpose of domestic self-sufficiency is almost attained. We
believe this is the most realistic approach. It also secures multiple routes of supply,
another measure to mitigate the risk of a stable supply. We believe, however, thatthe Export Trade Control Order currently prevents this approach from being taken,
making it difficult to achieve domestic self-sufficiency.

Thank you
Philippe Fauchet
Chairman
EFPIA JapanReference(1) Proposal concerning “State of Plasma derivative business in Japan”(2) Opinion concerning the “Review Committee on the State of Supply of Plasma
Derivatives: Final Report”
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(3) Michio Matsuzaki “Study on the establishment of a system to monitor blood transfusion
adverse reactions at medical institutions,” an assigned study as part of “Research into
collaborations by medical institutions at discovery of an infection from a product derived
from one of the specified organisms,” MHLW Science Research Project for 2005
(4) “Blood donation and blood products study on the recognition and awareness of livingcitizens” (QLife, Inc., August 25, 2010)(5) Toshiki Mano, “Challenges for Japanese blood services – discussions based on the resultsof a questionnaire survey of citizens and blood donors” Japan Medical Journal No. 4570,
November 26, 2011(6) “Future population of Japan and chronological changes in the potential blood donatingpopulation” Resource for Blood donation promotion investigation team, Blood Services
Section, Pharmaceutical Affairs Working Group, Pharmaceutical Affairs and Food
Sanitation Council, first meeting in 2010

6Contact
Noriaki Kanemoto
Secretary General
European Federation of Pharmaceutical Industries and Associations (EFPIA) JapanZIP code 151-8566
GSK Building, 4-6-15, Sendagaya, Shibuya-ku, Tokyo
Phone:
Fax:

03-6301-3066 (representative & secretary general)
03-3479-4782

Makoto Miyakawa
LeaderBlood Product Taskforce(Operation and Planning, President’s Office, CSL Behring, Co. Ltd.)
Phone: 03-3534-5906
Fax:
03-3534-5864
Email: Makoto.Miyagawa@cslbehring.com

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