Sunday, 8 November 2015

Alternative ideas for the Repurposing Bill

The repurposing bill appears to be there against the wishes of the Government 

Private members bills in the House of Commons are like members privates and usually wither as quickly as they appear after a weak stand:-) 

Central to the bill was we won't do anything to the currently licensing system but we will break every hoop that we have made pharma jump through to get you drugs and the government will fund it all. 

Pharma have a laugh at delusional academics who think they can really deliver an MS drug to you. They think they can do the work and that the system will crumble in response to their ego and pressure. However it could also be a bottomless pit for Central Government

Despite many attempts to find something useful Academic Neuros have created a lot of hope but not much else. 

Yes their are some big names out their that surf on pharma's coat-tails and are wheeled out to present data that a group of other people have worked hard to produce, but that is marketing....Can they do investigator led studies and bring you drugs without pharma involvement.  

Can it be done without pharma. Some egos will say it can but however to date I would say there are people in high positions that are totally clueless about the way business works and develop drugs.

They think that they can go ahead and do something useful and then bully the system so that it will change and everything will be hunky dory. This could perhaps be the case where there is nothing, but this is not going to fly for something like MS where there is so much company interest.

This success makes it uninteresting. The decision makers are not MS experts and they cannot see that there is nothing for progressive MS as there are lots of MS drugs available and those drugs are there so there is no point in doing something better and cheaper. 

Whilst  a trial in cancer has a quick easy endpoint, trials in Neurological diseases are incredibly slow and expensive. Something needs to be done to make getting treatments for neurological diseases easier. 

I will be happy to be proved wrong. That is my challenge to all the Neuros out there. Prove Me Wrong

Whilst I may be chastised for being negative, the question what is the reality?

However, this is the approach that neuros are taking in MS. Do a trial and hope for the best and juggle with the power and numbers to come in cheap. I ask then "What Next?" and all too often I just get those blank puppy-dog eyes starring back at me. 

"We will cross that bridge when we come to it. However, what comes after the first bridge is another bridge". 

The simvastatin trial is held as an example for the repurposing...phase II trial positive result....then what?. 

No immediate plan to do anything so a few years wasted whilst we have a think.....so far thinking for 5 years and ticking.

Let's do another phase III trial...but oops in academic costing it is over the magic £2,000,000 figure and is too expensive so have to have a rethink...maybe the alliance can fund......maybe we drop the power down and make the effect of the drug so big (as a primary endpoint) that we only do this many people. 

Seven years on and trial is......positive.... and off we go....but who is going to shell out to hold the licence at £100,000 a year for a £2.70 drug....Cross that bridge when we come to it?

Then pharma who have been having a laugh say "Exqueeze me mister regulator" but you make me do two trials so do you make them spend another 7 years and this is 14 years on since you started, by which time pharma will have found something and this means that you have to get a licence in Europe to prescribe it as it is no longer legal to prescribe off-label. 

The funding agencies say why do we want to fund another phase III trial? There is no merit academically in this because we know the answer and there are much better things to do with our cash. Plus this isn't sexy science so why would you do such a thing, cos we fund sexy science not stuff that is going to make a meaningful difference to peoples health. Furthermore, why should be we using taxpayers money to help industry as surely there is someone making the drug you are using...why don't they pay for it? 

Next pharma say "exquueeze me" again, you make us do all this regulatory stuff that costs us millions but it is OK to have the trial data based on ten page publication published in the New England Journal. You make us submit a 100,000 page dossier about every aspect of the drug, but its OK to have a academic group submit something paltry. Some may say ProfG has spent too long writing a 10 page paper on the Charcot project, but just think if he had to produce a 100,000 page document. We'll have all retired by the time he has got his act together:-). If it takes a company a year to get the documents in order, how long will it take an academi?

You make us go to NICE and submit another set of documents and we have to collect all sorts of extra data to please the people in committees that are now saying we'll just bend the rules because we are academics.

What happens if the MHRA do not bend

Will pharma question the double standard if the repurposing studies are not done to the same standards as done by pharma. 

However, how can this every happen? An academic trial comes in at less than £2,000,000  pharma are paying a lot more just to have the trial properly and independently performed and monitored and documents written this could be easily 20 times more. So who has a spare £50,000,000 to do an independent trial plus the cost of the actual trial. So if they have to go head to head and buy the comparator this will add another £50,000,000-£100,000,000. So this means you can never repurpose a DMT or do any studies once something is found.

Am I bursting bubbles, selling you false information to get a discussion going?. Yes there is an element of melodrama here, but I am playing devil's advocate as people need to think.

It has been consistently said that drugs can't be developed without pharma, but we perpetuate the myth that they can. What is the Alliance for Progressive MS doing to convince us that they can achieve something. They have got pharma on board but can they repurpose the academic way or do they have to invent and do it the old pharma way or can we be inventive and come up with something new?

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

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