This is why the Repurposing Bill will struglle....and the reason being it would not be innovative and pro business.
THE NEW REPURPOSING BILL
The repurposing Bill should have encouraged pharma to stand up and come to the party, they can take the risk and they can take the money.
A template already exists about how to do this and this is via the Orphan Disease Act, Rare Disease Bill etc
In the United
States, the Rare Diseases Act of 2002 defines rare
disease strictly according to
prevalence, specifically "any disease or condition that affects fewer than
200,000 people in the United States, or about 1 in 1,500 people. This
definition is essentially like that of the Orphan Drug Act of 1983, a federal law that was
written to encourage research into rare diseases and possible cures. In Japan,
the legal definition of a rare disease is one that affects fewer than 50,000
patients in Japan, or about 1 in 2,500 people
However, the European Commission on
Public Health defines rare diseases as "life-threatening or chronically debilitating
diseases which are of such low prevalence that special combined efforts are
needed to address them. The term low
prevalence is later defined
as generally meaning fewer than 1 in 2,000 people. Diseases that are
statistically rare, but not also life-threatening, chronically debilitating, or
inadequately treated, are excluded from their definition.
The definitions used in the
medical literature and by national health plans are similarly divided, with
definitions ranging from 1/1,000 to 1/200,000.
Orphan Diseases are rare diseases and you only have to do one phase III trial and you don't even need a patent but you get market protection so other company can not come into the space to compete.
First thing would have been to make progressive disease an orphan disease. I believe it was tried and it failed. The apparent success of the anti-CD20 may have killed that option.
The thing that would encourage
companies to try and repurpose their drugs is the prospect of making money.
Pharma have a vast array of drugs on their shelves if they could repurpose and
make money maybe they will take some risks to fund and monitor the phase III
trial and hold the licence etc.
At present with innovative drugs
the protection is the patent, but with the orphan disease act you don’t have to
have a patent but you have a system in place that gives the company market
exclusivity for x number of years. You could allow them to put up the price by
x% as an additional carrot to allow them to recoup their investment.
Can this be done yes it can.
You
have all heard how pharma have sued the NHS for using the cheap licensed drug
in place of the expensive drug with avastin and leucentis which are two differnt drugs but currently in
place we have the case where a licensed drug is available for one condition but
the generic in available for another condition and the NHS is only allowed to
buy the expensive version for the condition for which it is licenced. Therefore you could have a statin a £2.70 sold for cholesterol next to a statin at £27 or £2700.
Therefore everything is in place already you just
have to morph the orphan drugs act into the repurposing drugs act.
Orphan diseases encourage smaller size companies to try their luck
This would encourage pharma to do this
and involves no state money so a simple plan.
I am no great brain but if you got pharma (e.g. GSK) together they would tell you how they could and would do this to benefit
themselves and UK plc.
What if pharma
does not step up to the plate?
Then we need a route that academics can drive
this forward. Rather than being the licence holder and the purchaser (NHS)
which may create a conflict of interest. The Government could set up (a not for profit) independent company to create an infrastructure to get things going.
Following success of
the first few it should start to become self-financing as it will make on the additional revenue when they distribute the drug to the NHS. If they want, it could be
something that could be sold once it is on its feet making money for UK plc.
This could then be a vehicle to act as the licence holder and drug distributor and the vehicle to
distribute its profits, such that the profit could be used to support academics/small pharma to try and repurpose new indications and so use its profits for the good
of the company, industry and the country by supporting British Businesses. In a more capitalist structure is needed so be it.
If more countries enter into this it could spun out European wide and globally. This could feed the trial monitoring companies too and do stuff the proper way so a level playing field. Maybe a bit of thought on this as this was part for the repurposing bill.
There are lots of charities wanting this to happen. Maybe they can come up with a unified view that is acceptable to pharma and government and then get this debated in parliament again by using the 100,000 people petition.
The MS register has 15,000 people if they are mobilised you are a tenth of the way there get Panorama behind this and you never know what may happen.
These are just ramblings that can be finessed or rubbished but the current system is broke and we need a better deal for many people with conditions that have no treatments