Is it time to redefine a ‘breakthrough’?

Claudia Rubin 02 January 2018


The meaning of life

For patients to receive life-changing meds, perhaps it’s time to redefine ‘breakthrough’.


What do you think when you hear the term medical breakthrough? Mapping the human genome maybe, face transplant surgery perhaps, the HPV vaccine possibly? These astonishing medical breakthroughs do not come around very often, so perhaps there is a broader definition we could consider.

Less catchy, but more informative in terms of access to medicine issues, is ‘strategically important transformative products’. Recent headlines suggesting NHS patients will get faster access to breakthrough medicines implies that finding the right definition may well become increasingly pertinent. Behind that newsprint lies the official Government response to the Accelerated Access Review (AAR), which outlined a new fast-track route into the NHS for breakthrough medicines and technologies.

Although well received so far, as ever with grand proposals of this sort, it will now take some time to properly identify whether this will actually serve its purpose.

Launching the Accelerated Access Pathway (AAP), a new route to market that will streamline regulatory and market access decisions, the Government made it clear that selecting the best products for acceptance into the AAP is crucial.

It is anticipated that approximately five products a year will receive breakthrough product designation and go onto the new pathway, subject to satisfactory commercial negotiation. With such a small number entering the system, and no guarantee that all five will emerge, it is questionable to what extent this will impact the NHS’s record of rapid innovation uptake.


Changing of the guard

Arguably, NHS England has made significant progress in tightening its grip over commercial negotiations with industry. It’s taken over control of the Commercial Medicines Unit (formerly within the DH), pushed through incremental cost-effectiveness ratio thresholds and triggered a budget impact test for NICE, as well as taking on the role of agreeing future patient access schemes.

The continuing direction of travel is a fundamental downgrading of the primacy of NICE guidance and a considerable handing over of discretionary power to NHS England. Only time will tell whether this delivers a win for both the NHS and industry.

Existing concerns about how the NHS is poised to handle substantial increases in the number of therapies for genetic conditions that need assessing, and how to differentiate those that are truly ground-breaking, are unlikely to go away yet. The cautious welcome offered to the AAR response may be due, in part, to a belief that if an accelerated pathway can be shown to be possible and effective, it can soon become the standard process for more products.

Perhaps the element of the Government response with the potential to have the greatest impact early on is the commitment to invest £39m to improve local adoption and uptake of innovative medical technologies. New ‘Innovation Exchanges’ will be established to foster greater collaboration between the 15 Academic Health Science Networks (AHSNs), innovators, clinicians and patients, duly helping them to navigate the system.

This is intended to increase AHSN capacity and its capability to assess the local value of innovative technologies, while promoting diffusion across the local system. A lack of evidence around the effect of implementation is one of the most common local barriers to innovation. As such, the commitment to continue building expertise at local level and collaborating more effectively with national leads is extremely welcome.

It is always of considerable comfort to industry, and the patients their products are intended for, when the Government makes noises about needing the UK to get early benefit from the science it has played such a big part in developing. With concern mounting around Brexit, however, and how the relocation of the EMA will affect clinical trials and access to medicine in the UK, the Government needs to avoid the impression that every positive step it takes domestically will be undone by the Brexit fallout.

The fact that the Department for Exiting the EU has undertaken impact assessments of Brexit on dozens of economically important sectors ought to be reassuring, but somehow the secrecy surrounding their existence has succeeded in creating a whole new level of anxiety. With little agreement over something as fundamental as how to define, identify and even prioritise a medical breakthrough, much ironing out remains.     


AAP for that?

On the assumption that the right products are identified for inclusion on the AAP, is it a given that it will be in the manufacturer’s best interest to proceed along this route? After all, with other fast-track systems in place; a more streamlined Cancer Drugs Fund, the Highly Specialised Technologies programme, the Early Access to Medicines Scheme and others, it may not yet be clear what the true benefit of AAP inclusion is.

After all, approval of AAP products will still be dependent on their proving to be cost-neutral for the NHS, and it is likely that in return for a place on the AAP, companies will have to be extremely accommodating in their price negotiations. Indeed, NHS England’s newly created Strategic Commercial Unit is rapidly developing its ability to negotiate cost-effective deals.


Claudia is a Director at Decideum. Go to decideum.com




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