Re: Trade - EIGR
Part II(in response to a report put out that I will post shortly):
Just the other side of the arguments put forward by your posted report(which by the way is very much appreciated):
1)A very sharp hematologist on our private board has noted that a great deal of his patients refuse to take hydroxyurea. It does have IMO side effects that would most probably exceed the drug from GBT. If you already have low platelets then hydroxyurea will not be the medication for you. With voxelotor IMO the drug is not given the potential that it deserves. True they are quoting KOL's in the field, but like anything, if you see 20 physicians on a lot of things you will get at least three opinions. The KOL's that GBT had on their latest CC were no slouches IMO, especially Dr. Robert Adams. GBT noted that their trial was not powered to look actually check effectiveness for preventing VOC's but all experts agreed that the data was pointing in that direction.
The point about physicians and insurance carriers not wanting to pay for both hydroxyurea and voxelotor, that is fine and to be expected. But if you look at the patient's refusing hydroxyurea, and the ones that have problems with it.....still leaves plenty of patients to be able to get the benefit of voxelotor. The other situation and some of it is conjecture, but again if the red cells are more closely shaped the way they are supposed to be then at least it warrants consideration that will greatly reduce the risk of VOC's. Again IMO putting pressure on the NVS drug to show its value of need if that is the case. In other words, it might be relegated to third line therapy. Not to mention, the improved oxygen carrying capacity that you will get with a greater hemoglobin and with better shaped rbc's...more oxygen carrying capacity. This could thus help in decreasing risk or slowing the development of pulmonary hypertension.
2)Patient's on voxelotor, if they do well and not have two VOC's per year may prove that many people don't need the NVS drug. Just my opinion but from real world experience in dealing with insurance carriers there will be really hard times for the NVS drug if voxelotor gets approved and performs like I believe it will....and as those other KOL's had mentioned.
3)As for the stem cell transplants and gene therapy, there are huge unknowns and risks. Sure if I had a jetpack and could just put it on and go directly to the office I would save a lot of time and my world would be better. Trouble is a jetback isn't practical at this point in time. If someone gets a stem cell transplant I can't see them charging less than $500 K for that upfront and who knows what the extra costs will be? For gene therapy, from what I've seen the patient's may not get the durability that many espouse and would maybe require something like voxelotor too. The pricing of this drug is so important to get it's foot in the door. Charging just $1200 per month and capturing on 35% of the US market would make this a $500 million a year drug. t