Authored by Karl Simpson
Well, the pharma industry’s rare disease pioneers gather again this week for the World Orphan Drug Congress in Geneva. It is a two day seminar that focuses on three separate tracks, Clinical Development, Market Access/Pricing and Reimbursement as well as Corporate Development and Partnerships.
So here we are, end of day one, reflecting on some of the issues discussed today. It is always good to represent Liftstream at these events, it just reminds us what challenges and opportunities lie ahead for the life sciences sector, and in rare diseases they are numerous.
Sandwiched in between the usual networking opportunities and catching up with contacts you haven’t seen for a while, it is a great way to hear what is really on the minds of some of the key industry participants shaping opinion in the rare diseases market.
The meeting kicked off with a great energiser from Theresa Heggie, the SVP, Global Commercial Operations at Shire HGT, who are the lead sponsors for the event, somewhat logical given their new shiny Nyon facility almost casts a shadow over the conference venue in the low winter sun (actually it is raining cats and dogs). Theresa reminded us that we’re in this together – sharing ideas, successes and failures would all be important in delivering the results for the industry. It is interesting, this year I would say that openness and collaboration have been constant themes across most of the events I have attended.
Theresa gave the floor over to Yann Le Cam from Eurordis who in his enigmatic and authoritative style walked us through the European outlook from the Eurordis perspective. So what were the take away messages. Well, undoubtedly we are well served by being reminded that while investors, companies and patients are all very much global in context, regulation and pricing and reimbursement are local decisions and that the industry must appreciate this very clearly. He outlined that within Europe, time is nearly up on the need for national level plans for rare diseases, with 2013 being the cut-off. There continues to be a strong agenda to deliver the therapies to patients and this is message is a consistent theme. Patient access, how can it be best achieved. However, if I convey to you every aspect of industry, regulatory and patient action presented in this talk, I would have lost you be now, so I’ll try to keep it brief and to point, after all, had you wanted to know that much, you’d be here I guess.
> The important aspects of this presentation focused on the need for patient and disease registries and the advantages they could offer.
> IRDiRC featured strongly and how this consortia is trying to bring about a far more coordinated approach to the investment and development of therapies in the rare disease area. Lofty goals but drive and determination evident for sure.
Clearly the HTA environment continues to dominate thinking, planning and discussion. There still remains this challenge of centralised Regulatory approval but HTA decisions taken at the country level and so deeper integration of this HTA methodology was called for. Clearly part of this discussion centred on the move towards adaptive licensing and the notion that evidence-generation in rare disease therapies is a continuum. There must be a focus towards building the evidence in the pre and post approval phase. Also, that the value assessment must switch from a pure clinical value to one which is judged on real life benefit, taking societal and the myriad of other factors into account in assessing whether a new drug for rare disease therapy has value.
As all good keynote speakers should aim to do, Yann Le Cam gave the audience a framework or agenda for the rest of the day. He set up the issues which dominate the landscape and which each track could discuss and debate.
Here are some of the things I feel everyone here is thinking about:
1. Need for greater focus for patient access
2. Stronger collaboration across the stakeholders in rare diseases
3. More focus towards integrating HTA considerations into clinical and regulatory programmes earlier
4. Working towards new novel clinical trial design
5. The commitment towards looking at evidence building in the pre and post value license environment
6. Use the framework in existence to explore the options for adaptive licensing
7. Greater transparency across all aspects of rare disease
8. Continue to raise awareness about the patient needs and the importance of these therapies
9. Value is more than just cost in the austerity environment