Collaboration can ensure access to life-changing innovation

As a pioneering risk sharing scheme has shown, new medications can be the catalyst for a whole new care infrastructure.

But more than 15 years later, we are facing the same dilemma of how to get innovative new treatments to the people whose lives they could change.

When the first disease modifying therapies (DMDs) for relapsing-remitting multiple sclerosis (MS) were licenced in 1996, they offered hope to a largely forgotten group.

With no treatments available, those diagnosed with the debilitating neurological condition were told to go home and wait until they needed a wheelchair.

But Avonex, Beteferon, Copaxone and Rebif offered a chance at something different – until they were appraised by what was the National Institute for Clinical Excellence (NICE).

While they were clinically effective, with an average £10,000 a year price tag, they were not cost effective, the body ruled.

Securing access to ‘non-cost effective’ drugs

“When our original guidance was published in 2002, NICE concluded that, on the balance of their clinical and cost effectiveness, neither beta interferon nor glatiramer acetate would be recommended for the treatment of MS in the NHS in England and Wales,” said a NICE spokesman.

“NICE also requested that the Department of Health and the National Assembly for Wales, along with the manufacturers… consider what action could be taken so that the NHS could obtain these drugs in a way that would be cost-effective.”

The result of these considerations — and some heavy campaigning by patient advocates — was the Risk Sharing Scheme (RSS).  Set up in 2002, at its core was a deal struck between the Department of Health and Bayer, Biogen Idec, Merck Serono and Teva.

All patients eligible for the drugs, under the Association of British Neurologists (ABN) criteria, were given access and monitored.  Should the DMDs not meet a £36,000 per quality adjusted life year (QALY) target, the drug companies would issue refunds.

The final data includes 5,000 patients over 10 years of follow-up and was published in the British Medical Journal this month.

Not only do they show the DMDs have met their cost-effective target, they suggest the drugs can delay the time it takes someone with relapsing-remitting MS to need a stick to walk by four years.

“With knowledge that the Risk Sharing Scheme (RSS) was coming to an end in 2017, NICE reviewed its previous guidance using its usual methods,” said the NICE spokesman, adding that an updated Technology Appraisal had been published in June this year.

Unmet needs

In 2002, just six per cent of people with relapsing-remitting MS (RRMS) had access to DMDs. This compared to 28 per cent in the rest of the European Union and 46 per cent in the USA.

Under the RSS, the number of people with access to DMDs, of which there are now 13, has gone from 2,000 to 18,000.

But the RSS has also helped meet patient need on a scale that few could have, because to deliver the drugs, the whole system of MS care had to be built from the ground up.

Speaking in the House of Lords in 2004, Lord Howe, at the time a Conservative spokesman for health, said: “In many areas there was simply no infrastructure —clinic space, administrative support and appropriately qualified clinicians and nurses were simply absent.”

Echoing these sentiments more than a decade later, Dr Martin Duddy, consultant neurologist in Newcastle and co-author of the data report, explained that today’s system of MS specialist neurologists, MS nurses and MS centres had been made possible by the RSS.

The primary care trusts that were at that time in charge of delivering services were obliged to set up the means to provide the drugs. The funding that came from the RSS was used to set up clinics, design services and recruit and train specialists.

Crediting his own job to the scheme, Dr Duddy said: “People tend to focus on the financial side of the RSS.  But the wall of funding that came from the Department of Health and the drug companies provided our entire infrastructure.”

Building a care infrastructure

According to the MS Trust, which administered the scheme, the number of MS specialist nurses has gone from 80 in 2005 to more than 240 today. There are now more than 70 specialist MS centres across the UK, compared to the 25 that existed in 2002.

The infrastructure the scheme created has improved access to MS services for 100,000 people in the UK living with MS.

“As administrator of the scheme, we are proud to have contributed to what has been achieved,” said MS Trust chief executive, David Martin, “but we won’t be resting on our laurels.”

That’s because not everyone benefited from the swathes of funding and attention, which led to a network services designed primarily to deliver drugs.

History repeating

At the very moment we celebrate what has been achieved for people with relapsing-remitting MS, those with the more severe form of the disease are facing a familiar struggle.

The first DMD for progressive disease was licenced at the start of the year and while NICE has approved it for RRMS, it has been judged not to be cost-effective in primary progressive disease.

There are no approved treatments for this form of MS, which leads to people experiencing disability significantly quicker than those with RRMS.

Jo Sopala talks about risk sharing scheme

“People do everything they can to minimise the impact PPMS has on their lives, but what they really want is access to treatment which will slow down the progression of their disease,” said Jo Sopala, Director of Health Professional Programmes at the MS Trust

“They risk becoming the forgotten people with MS.”

A risk worth sharing

What happens next for people with progressive MS is unclear. There are a number of potential DMDs in the pipeline, and in the meantime groups like the MS Trust and the MS Society will continue to fight to ensure the patient voice is heard.

What we do know, thanks to the RSS, is that game-changing drugs are worth fighting for, and that collaboration on a grand scale can ensure access to treatments and services that can change lives.