Of tortoises, hares, and future hopes and fears

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Mike Holl...

Mike Holland

Of tortoises, hares, and future hopes and fears...

Good report in Nature Medicine this week summarizing how the future landscape in haemophilia care is likely to change as the new long-acting products come through. (It gets the report for bad headline pun of the week: “The clot thickens for long-lasting drugs that stop hemophilia short”). It contains a table showing the current US regulatory status of the main players.
  Company Product Product type Stage
Haemophilia A Biogen BIIB031 Recombinant factor VIII Fc fusion protein Awaits filing
  Novo Nordisk N8-GP GlycoPEGylated recombinant factor VIII Phase 3
  Bayer BAY 94-9027 PEGylated recombinant factor VIII Phase 2/3
  Baxter BAX855 PEGylated recombinant factor VIII Phase 2/3
Haemophilia B Biogen BIIB029 Recombinant factor IX Fc fusion protein Filed
  Novo Nordisk N9-GP GlycoPEGylated recombinant factor VIII Phase 3
  CSL Behring CSL654 Recombinant factor IX albumin fusion protein Phase 3
There’s no doubt change is needed. Steven Pipe from the University of Michigan Medical Center in Ann Arbor is quoted as saying: “We've been using the same products for decades. Now we see this breadth of clinical trial activity and novel strategies.”
In the US at least, Biogen (in this country we know SOBI their Swedish partner) seems to be leading the pack with two products that have completed phase 3 trials and are now either already filed (haemophila B) or awaiting filing (haemophila A). With these, prophylaxis could be given as infrequently as every two weeks (BIIB029 in haemophilia B) or every 3.5 days (BIIB031in haemophilia A). That could make a huge difference to patients who currently take prophylaxis two or three times a week. The half-life and safety data also look pretty good.
For drug companies, the experience in many other therapy areas suggests that being first doesn’t always mean cleaning up. In haemophilia, Biogen/SOBI will certainly face fierce competition from the companies with a more established infrastructure in the UK market. Baxter, Bayer, Novo Nordisk and CSL Behring all have long-acting products in late stage development.

But what will it mean for us?

The companies will put huge efforts into detailing small differences between products. How these differences affect the haemophilia care team is clearly of great interest to Haemnet, both in terms of individual workloads and in how commissioners manage the use of these products.
Last year, we put up a Survey Monkey questionnaire for members of the HNA and asked for comments on the future of haemophilia care. We will share the results of that survey at the HNA meeting in Oxford and on Haemnet for those of you unable to come). Its clear that many of you recognize the impact these new treatments will have on the care you provide.
You can read the full article here http://www.nature.com/nm/journal/v19/n2/full/nm0213-121.html


Mike Holland is the founder of Haemnet and SixVibe. He is a freelance medical writer, editor and meeting organiser - find him at Google+ and Driftwood.

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