UK bioscience companies form Rare Disease Industry Group
European Pharmaceutical Review, Dr Zarra Kassam, 24 July 2017

A group of bioscience companies who focus on pharmaceutical development for rare and very rare diseases have formed an industry group. Entitled the Rare Disease Industry Group (RDIG), it is committed to inform the public about rare diseases and offer pragmatic solutions to ensure access to orphan and ultra-orphan medicines. Orphan medicines are generally research intensive to develop, and usually treat a very limited group of rare illnesses each. In the UK, 1 in 17 people are expected to be affected by a rare disease in their lifetimes. There are currently 143 orphan medicines available in the UK, but only 68 are reimbursable on the NHS. By contrast, 116 orphan medications are reimbursed in France, and 133 in Germany. The RDIG will exist under the auspices of the BioIndustry Association.

NHS should pay for medicines based on outcomes, argues Novartis-backed report
Pharmaphorum, Neil McConaghie, 24 July 2017

Pharmaphorum further covered on the July 21st report from the Social Market Foundation, which was funded by the UK-based operations of Novartis. The report’s findings endorse a results-based approach to drug funding and pricing. The major UK drug pricing scheme, the PPRS, is due for renewal at the end of 2018, and many companies have expressed their dissatisfaction with the current model. Novartis has pioneered the results-based model in the United States for heart medicine, and Italy currently employs a variation on the pricing system for the majority of pharmaceuticals. The ABPI is currently consulting with members, and there is growing appetite in the industry for a greater use of outcome based mechanisms.


There is no parliamentary coverage.


UK bioscience companies form Rare Disease Industry Group
European Pharmaceutical Review, Dr Zarra Kassam, 24 July 2017

A group UK bioscience companies that specialise in treatments for rare and very rare diseases have launched a Rare Disease Industry Group (RDIG).

Formed under the umbrella of the BioIndustry Association (BIA), the RDIG is committed to developing recommendations that can pragmatically inform and improve the challenge of ensuring patient access to treatments for rare and very rare conditions, sometimes referred to as orphan and ultra-orphan medicines.

In the UK, it is estimated that 1 in 17 people – approximately 3.5 million people – may be affected by a rare disease at some point in their lives. The impact that rare diseases have on patients, their families, and society is profound, as many are severe, chronic and progressive, with high mortality rates.

In England, there is no dedicated process to assess orphan medicines and uptake of these medicines is varied. According to the Office of Health Economics, of the 143 orphan medicines that were available in the UK, only 68 of these medicines were reimbursed and made available to NHS patients in England. In contrast, 116 orphan medicines are reimbursed in France and 133 are reimbursed in Germany.

Commenting on the launch of the group, BIA CEO and Chair of the Rare Disease Industry Group, Steve Bates OBE, said:

“The BIA has long been a vocal advocate of the need to improve access to treatments for patients with rare and very rare diseases. In 2014, the BIA published the report Very Rare Diseases, Complex Issues, calling for a separate approach for evaluation and commissioning of ultra-orphan medicines. The RDIG will now continue and build on the BIA’s previous work in this important area.”

Peter Kuiper, General Manager UK and Ireland at Sanofi Genzyme, the speciality care global business unit of Sanofi, and Vice-Chair of RDIG said: “There is strong public support for treating patients with very rare diseases as part of the comprehensive offer of the NHS. Almost three quarters of the UK public agrees that patients with a very rare disease should have the same access to treatment based on clinical need as patients with common diseases. The RDIG looks forward to developing recommendations and initiatives that can help move the dial on this important issue.”

NHS should pay for medicines based on outcomes, argues Novartis-backed report
Pharmaphorum, Neil McConaghie, 24 July 2017

A new report from the Social Market Foundation think tank argues that the UK’s National Health Service should switch to paying for medicines based on patient outcomes.

The report, Outcomes-based reimbursement of medicines, was sponsored by Novartis’ UK operations, but was independently authored by the think tank.

Its message is certainly in line with Novartis’ thinking in the UK and beyond however, as the company is among the leading pharma exponents of ‘pay for performance’ and similar deals.

While outcomes-based payment clearly has some potential risks for pharma, Novartis believes the switch could save the NHS money, and allow patients to access new medicines faster.

The UK has one of the slowest uptakes of new medicines among developed nations, but the report suggests it could learn from pioneers in outcomes-based payment, such as Italy.

Report author and SMF research director Nigel Keohane said:

“With pressure on the health budget growing, the NHS should adopt the principle of only paying for what works when it buys medicines. This would simultaneously drive better value for money and mean that patients could be given earlier access to new treatments.”

“Given the NHS spends £17bn on medicines each year, the reforms would also steer manufacturers to develop innovative drugs that can make the largest improvements to patient health.”

Drawing on international experiences in countries such as Italy, lessons from other UK public services and the wider NHS reform agenda, the report recommends that:

  • New drugs that meet specific criteria – such as addressing ‘unmet need’ among patients – would be eligible to go through the new purchasing scheme and fast-tracked to patients.
  • The NHS and the manufacturer would agree the health outcomes that should be achieved and the basis on which the manufacturer would be paid if and / or when the drugs are successful.
  • The NHS should lead an exercise towards better outcomes measurement and data to enable performance to be tracked properly. The report says this would be of wider benefit to NHS commissioning reforms.

The report comes at a confusing time for the UK pharma industry, its future clouded by the many uncertainties of Brexit.

Theresa May’s government has stressed that the life sciences sector is one of UK’s most important, but new budget control measures enforced by NHS England are causing consternation in the pharma industry.

The key UK pricing agreement, the PPRS , is due to renewal from December 2018, and many pharma companies have expressed their dissatisfaction with the system, which has capped spending but not delivered on promises to accelerate new medicines uptake.

Meanwhile the next few months are due to see the Government belatedly publish its response to the Accelerated Access Review, while Sir John Bell is set to unveil the Life Sciences strategy.

Commenting on the report, Barak Palatchi, Oncology General Manager, Novartis UK & Ireland, indicated that outcomes-based reimbursement was the way forward.

“We know that our health system is incredibly stretched. The NHS is being asked to do more with less funding, and patients are, rightly, demanding swift access to the effective, innovative treatments, they deserve. We need a different approach so the health system is sustainable, delivering better economic value over the long-term, and ensuring better patient outcomes”, said Barak Palatchi.

“We are working in partnership with the medical and patient communities to bring our science-based innovation and pioneering approach to deliver novel treatments to more people when they need them”, he concluded.

Novartis has pioneered outcomes-based payment in the US for its heart failure drug Entresto, with many other companies agreeing similar deals with health insurance companies there.

The report also gathers insights from Italy, which already uses two different outcomes-based agreements: since October 2012, there have been16 ‘Payments-by-Results’ deals and two ‘Risk Sharing’ contracts.

The UK industry association the ABPI is currently consulting with its members on what they want from the next PPRS agreement. It is far from certain that the industry would back a move to an entirely outcomes-based system, though there is growing appetite from the NHS and the industry for a much greater use of such mechanisms.

Read the full Social Market Foundation report here


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