No 193 March 2016

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WFH Global Forum

Sharon Caris

Sharon Caris is Executive Director, Haemophilia Foundation Australia

The World Federation of Hemophilia Global Forum held in November 2015 was packed full of information, in fact so much, that it was hard to keep up!

TREATMENT FOR ALL

As always, it is confronting that despite increased global supply of recombinant clotting factors and some innovative humanitarian programs for plasma derived clotting factors, there remains significant unmet need in the developing world. With only 30% of the global haemophilia community accessing appropriate treatment, there is huge scope for both recombinant and plasma derived factor VIII, in particular, but the ongoing and huge challenge is to find ways and motivation for the manufacture and supply of treatment that is affordable and accessible to those who need it. It is our responsibility in Australia to try to do something about this, and I urge you to participate in activities that will help achieve the WFH vision, and ours, ofTreatment for All.

TREATMENT SAFETY

Safety is linked to supply. For those without access to a supply of safe treatment, there is a need for both of these aspects to be addressed. While the historical concerns about blood product safety are somewhat more reassuring now we have appropriate viral inactivation and donor selection strategies in place, there is no place for complacency – there are new viruses capable of emerging in biologicals at any time. Further, the ongoing challenge and worry remains the complication of inhibitors developing in people using clotting factor and whether these can be prevented or reversed. It is pleasing that there are new treatments in development and some not very far away from the market.

EVALUATING NEW PRODUCTS

The discussions around new products and longer acting clotting factors and gene therapy are very interesting and challenging. There are more treatment products for bleeding disorders in various stages of development globally than ever before. The holy grail of gene therapy continues to be elusive on a large scale, although there have been some successes with small groups of patients.

There was great interest at the Forum in how to evaluate the benefits and costs of longer acting clotting factor therapies. Clotting factors with longer or extended half-lives stay at higher levels in the body for longer and less frequent/fewer infusions are needed to achieve the same clotting result as for the regular clotting factor products.

These new products have led to international and local discussion about how prophylaxis treatment should be given, what doses should be given, how often and what % clotting factor levels should be aimed for in a person’s blood. Many agree there is potential for better treatment outcomes and better adherence to treatment. A prophylaxis protocol can be tailored to the individual based on his/her bleeding patterns and clotting factor levels as well as taking into account factors such as age, joint health, and physical activity.

PATIENT PARTICIPATION

The benefits would need to be justified by governments who pay for treatment products, and the patient community will have an important role in providing some of the data on outcomes of such treatments for this. MyABDR could become a very important tool in Australia as the data generated from this helps doctors and their patients understand and evaluate the outcomes of treatment. The HFA Council is considering the HFA Treatment Policy in the context of longer acting clotting factors and other novel treatments and how these are evaluated in Australia for purchase and supply.

At the Forum, I reflected on the benefit for both clinicians and patients where clinical trials are available, and the value of more trials having been available in Australia in recent years. It is important for clinicians to have experience with a range of therapies, but our community also benefits from the courage of individuals who join clinical trials that later result in new products being manufactured, registered and ultimately available to others. Keep in touch with your Haemophilia Centre to hear about new opportunities for new treatments or clinical trials.

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Date last reviewed: 15 March 2016