Update Summer 2000

WORLD FEDERATION OF HEMOPHILIA
Montreal, Canada - 10/11 April 200

I was privileged to attend this Forum on behalf of IPOPI. It was a unique international forum, which brought together patients (with haemophilia), treaters, manufacturers and regulators from around the world, under one roof. Those attending in all categories came from developing as well as developed economies.

The subjects addressed came under four broad headings:

• Concepts in Risk and Safety
• Resource Utilisation
• Regulation and Harmonisation
• Costs, Economics and Production.

While the thrust of discussions centred on coagulation materials, I was able to keep Ig in the picture as well, and widen the perspective from time to time. While it is very clear that Ig is becoming the market leader in plasma derivatives, I brought the message that there is no comfort or solace in that for us.

The haemophilia community has succeeded in having a high profile with the producers and if we are to get anywhere at all, we must all (nationally, regionally and internationally) alter the impression we give to industry. The world and the environment of blood products is changing rapidly and radically. While the increased demand for Ig will run in tandem with a fairly constant demand for coagulation products in the immediate future, we need to look beyond the next 5 --10 years and prepare ourselves for other changes. The impact of recombinant clotting factors (rF) will not have an adverse impact on cost - and hence supply - in the short term. We need to look at and consider the implications of gene therapy and transgenics as well. The international demand for Ig will grow and grow despite a future decreased demand for plasma derived coagulation products. This has very real cost implications for Ig - and hence potential supply implications.

It is of the utmost importance that all NMOs of IPOPI monitor very closely the situation in their own countries and regions and report regularly to a central IPOPI location. No one else will do this for us and it is vital that we (as WFH) are on top of statistics surrounding cost and supply issues.

In that context, I recommend most strongly that we invite PPTA to make a presentation to the IPOPI meeting in Jerusalem so that we are all better informed and alert, not only to today's situation, but also to the unfolding position over the next horizon.

Other points that emerged:

On the harmonisation and regulation front, we must all continue to maintain close collaboration with our regulators at national and regional levels. This helps to make sure that they are cognisant of our needs and of our expectations. I believe that at a European level, for instance, our regulators understand the needs and demands of haemophilia but that they are woefully unaware of the needs of people with primary immunodeficiencies. We need to make sure that all our regulators are aware of our needs in the same way that IDF have already succeeded in the US.

On the safety and risk front, the haemophilia patients organisation in the USA (NHF) have an absolute standard of safety - zero tolerance of anything that might pose a threat or a risk. This means they find only synthetic coagulation materials - recombinants - acceptable. While their position commanded little or no support from other patient groups, it is a highly dangerous position from our point of view and from the view of people with primary immunodeficiencies in developing economies. I told them so! On the other hand there was a distinct move towards cheaper coagulation products with acceptable rather than extreme levels of safety.

A further recommendation is that we should work much more closely with the haemophilia community nationally, regionally and internationally. Here in the UK we have established a plasma users group with the haemophilia community and other users of plasma derived products. At a European level we have met once with the European Haemophilia Consortium (EHC) and I think that we need to work more closely with WFH at an international level - both to enable our voice to be heard and, where necessary, to support the demands of the haemophilia community.

In all this we will not always necessarily be friends but common goals are achieved through recognising ones differences rather than being in total agreement at all times.

The main lesson from this two day conference is that we must achieved the same level of perceived importance from industry that has already been achieved by the haemophilia community. Let's go for it!!

David Watters
London, UK
Wednesday, 03 May 2000