aHUS Patients have a value beyond the balance sheet

It was little known, but Alexion decided years ago that it would reinvest 40% of any of its profits from the sale of eculizumab into further research and development of its therapies for rare diseases. That worked out at about 20% of its sales income. So roughly one fifth of the price of eculizumab was spent on further research.
In Alexion’s financial report for 2016, just over $750m was spent on R & D, or around 25% of its $3 billion income. Its operational profit was just $600m. The R & D spend is likely to be a similar  in 2017 (it had reached $600m by the end of Q3), albeit that its sales income could be much higher than 2016.*
Whether it is the need to show a greater profit, or the original policy is not sustainable with a multi- product portfolio, Alexion has decided to cut back on its R & D investment. R& D embraces a range of activities from pure science research to tracking the effectiveness and safety of a drug. This would include the cost of running a Patient Registry, which Alexion is obligated to do as part of its marketing licences given  by the FDA and EMA.
Readers of the aHUS alliance website will probably be aware that the alliance has been a part of the aHUS Registry for the past three years, and it the only one where patients are represented on a Scientific Advisory Board, which governs the work of the Registry, beyond using it for tracking safety of eculizumab.
The obligation to FDA and EMA was fulfilled after  five years of tracking by the Registry which was achieved  in 2017. A report on the safety of the eculizumab will be published by Q3 this year, it had been expected in December 2017.
The aHUS Registry will continue however, but Alexion has needed to reassess its non-obligatory commitments. Originally it had intended to enroll 2000 aHUS patients by 2023. Having begun recruitment in 2012, there were 1744 patients recruited by 31 December 2017 at over 300 hospital sites. However, a significant number of sites, although set up to do so, have not enrolled patients, and some which did enroll some patients did not provide follow up data, and remained inactive. There is no value in continuing these sites in the Registry; and understandably it is not a good use of R & D resource.
Sadly, Alexion chose not to stop there. It also  reassessed the economics of having sites where only a few patients were enrolled and closed them too. It also decided to stop enrollment at sites which are to continue to updating  existing patients data (apart from one country).
The effect of this is to reduce the size of the active Registry to 700 patients (although that will increase with recruitment in the one new country).
All hospital sites affected have been told, it will be left to those sites whether to notify the  patients being excluded.
The alliance supports registries because particularly for rare diseases one patient’s experience is so very important to research. Its participation in the SAB has made “Patient Included” a reality and it now permeates the work of the researchers using patient’s data. So, it is a blow to the alliance’s efforts to see 1000 patients excluded, when 99% of aHUS patients told us in the 2016 Global Poll how they welcomed being in a Registry, and those who were not, expressed a desire to participate.
It was disappointing, however, to find that so many sites, which had aHUS patients, had not collected data about them, that adds no value.
The alliance will be reviewing its relationship with the Registry, and will consider the benefits to patients in the new size model to be deployed in future. It is important to let researchers know what matters to patients and it is important that valuable resource is respected. It is something that does not show in the balance sheet but does affect corporate perception.
If, as is likely, Alexion make a financial gain from its decision to exclude patients from the Registry ; then some of the gain should be put towards helping patients, who cannot access the drug they need, get it.
It is one way for Alexion to respond to the alliance’s call on them on 1 January.
* the 2017 result for R & D was actually $100m more than 2016, and the sales income was $3billion. Alexion is expecting income in 2018 to be possibly as high as $4 billion ,and it plans to spend 20%  on R & D which would be about $800m , a fall of over $50million on 2017; but around $200 million less than if it had continued to invest at the same rate as in previous years. Quite a cutback.

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