Manifesto for ‘patient driven’ innovation launched


A major cultural shift is required to make medical innovation 'patient driven', according to a new manifesto.

The call for change comes from Empower: Access to Medicine, an alliance of UK-based patient organisations, the Centre for the Advancement of Sustainable Medical Innovation (CASMI), which wants to develop new models of medical innovation, and the Genetic Alliance.

The manifesto was launched on Tuesday in London at a reception in the Houses of Parliament, with a number of MPs and health minister Earl Howe endorsing the aims of the group.

The driving force behind the movement are campaigners urgently seeking new treatments halt for deadly and debilitating diseases, such as Duchenne muscular dystrophy (DMD), for which there is currently no treatment.

The genetic disease affects only boys, typically first appearing before the age of six, then causing severe muscle degeneration which disables and eventually causes death around the age of 25.

Three mothers of DMD sufferers spoke at the launch of the manifesto, and called on fast action from medicines regulators and governments in the US and Europe to accelerate access to potential breakthrough drugs.

One of the campaigners, Alex Johnson, called for "bold, compassionate and dynamic" action from regulators to allow greater access to early-stage medicines in cases of exceptional need.

The Empower and CASMI manifesto comes just weeks after the UK government unveiled the Early Access to Medicines scheme, a fast-track access system which has come about in direct response to rare disease campaigners. European regulator the EMA has just launched a similar project, the Adaptive Licensing pilot, which would grant an early licence to medicines for a small patient population to help speed access.

Alex Johnson spoke of her frustration at the FDA's refusal to fast-track one pipeline treatment for DMD, but was hopeful the UK scheme could help.

"I believe if established correctly, it has the potential to save my son's life," she said, adding: "Let's show that we are world leaders."

Empower says that the UK Early Access scheme, which is to be fully launched next month, and the Adaptive Licensing Pilot in Europe are "hugely positive steps forward", but that more still needs to be done to make drug development more responsive to patients' needs.

Cultural and legal change

The joint manifesto, produced by CASMI, sets out a 10 point plan which would create an environment where patients would have a greater voice in their treatment, and more adaptive pathways for clinical research would be developed.

The campaigners are also supporting Lord Saatchi's Medical Innovation Bill, which proposes changes in the law aimed at freeing doctors to give novel but relatively untested treatments to patients without fear of being sued.

The government had originally blocked the bill put forward by Lord Saatchi - who lost his wife to ovarian cancer in 2011 – saying changes were unnecessary. But now the government has bowed to pressure, and the proposals are now out to public consultation, which closes in a month's time. Campaigners are drumming up support for the Bill via an online petition.

Lord Saatchi and his supporters say the changes would end the "culture of defensive medicine in the NHS", whereby doctors are still liable to be sued even if patients give their full consent to untested treatments.

The Bill is being supported by Conservative MP George Freeman, who was previously involved in bioscience venture capital and is now the government's life science adviser.

He and fellow MPs say the bill will allow for responsible scientific innovation, whilst still protecting patients against reckless experimentation.

Addressing the Westminster meeting, Freeman said the old models of pharma and biotech R&D had proven to be wasteful and slow, and that 'patient driven innovation' would accelerate breakthroughs.

Early Access scheme and 'Adaptive Licensing' pilot

The potential of the recently announced Early Access to Medicine scheme in the UK has been welcomed by patient groups, pharma and biotech companies, but full details of how it will operate are yet to be released.

The formal launch of the scheme comes in April, and will include details of what kind of evidence a pharma or biotech company would have to put forward to gain the 'Promising Innovative Medicine' (PIM) status.

Health Minister Earl Howe was also present at the House of Commons meeting, and said the PIM status could be given several years before full regulatory approval, and thereby opening up access to patients in urgent need of treatment. Earl Howe said companies would not have to generate an "enormous dossier" but put forward a convincing case based on evidence.

He said the co-operation of the NHS would also be vital to the success of the scheme, and he said the regional Academic Health Science centres would play a pivotal role in this.

Comparing the UK Early Access to Medicines scheme to the Europe-wide Adaptive Licensing pilot, Earl Howe said of the EU plan: "I think this is very promising, but I envisage it moving at a much slower pace [than the UK scheme]."

He pointed out that the European pilot would give access based on a provisional or adaptive licence, whereas the UK scheme would grant access to drugs without any licence.

GW Pharmaceuticals – 'the devil is in the detail'

Also speaking at the event was Dr Geoffrey Guy, chairman of GW Pharmaceuticals. He voiced his agreement that UK doctors no longer felt comfortable operating in experimental fields, and said local drug and therapeutic committees were stifling innovation.

Asked by pharmaphorum for his thoughts on the Early Access scheme, he said: "The devil is in the detail," but added: "If it's good, we'll go for it. We have 4 or 5 drugs in development which are in that rare or ultra-rare category."

Nevertheless, Dr Guy said Europe and the UK had fallen far behind the US in terms of their willingness to pay for new medicine, which meant that America was still the place for small pharma and biotech companies to focus their efforts.


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