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Posted 17/03/2014 by sevans

The pace of scientific discovery cannot move fast enough for many people. And patients with life-threatening illnesses cannot be expected to ignore any potential lifeline that the global pharmaceutical industry can throw them. But they do not have the time to wait for all the trials and regulatory hurdles to be overcome. Ensuring the safety of our medicines is one thing, but should it be at the risk of denying patients a therapeutic lifeline?

A case in point has arisen in the US, where this week the case of a seven-year old boy suffering from cancer and now a life-threatening virus has finally prompted a change in how clinical trials can be arranged and authorised. The boy’s parents had discovered that a small biotech concern, Chimerix, based in Durham, South Carolina, has been engaged in Phase 2 clinical trials of an experimental therapy against lethal viral infections, brincidofovir, that was being developed with US government funding. Naturally they contacted the company and asked to be able to use the so far unapproved treatment.

The biotech was therefore caught between a rock and a hard place – whether it wanted to or not, it could not supply an unapproved drug that had not even completed its Phase 3 trials but was, at the same time, subjected to intense media attacks for denying it, with the ceo and staff members even receiving death threats. 

Fortunately, the US Food & Drug Administration has found a way around the situation by allowing the treatment to be used in this case as a pilot study, with Phase 3 trials to follow at a later date, but this case throws up a major conundrum for the pharma industry, regulators and society. 

While we all expect to have all our drugs complete appropriate clinical trials, regulatory approvals etc to meet the efficacy and safety concerns of all involved, the process can take too long, especially for patients for which the therapy concerned might just be their last hope. In the UK, for example, it can take a year or more for marketing approval to be granted by the Medicines and Healthcare Regulatory Authority (MHRA) even though all the necessary trials etc have been completed with satisfactory outcomes.

To overcome this particular hurdle, the UK government plans to introduce a Promising Innovative Medicine (PIM) designation and an Early Access to Medicines Scheme (EAMS). The EAMS aims to support access in the UK to unlicensed or off-label medicines in areas of unmet medical need. The PIM designation will provide an early indication that a product may be a possible candidate for the Early Access to Medicines Scheme. The MHRA will issue an EAMS opinion if the quality, safety and efficacy data provided in support of the application is sufficiently compelling for a positive benefit: risk balance and added clinical value, and the EAMS scheme will be complemented by the introduction of a new National Institute for Health & Care Excellence (NICE) technology appraisal and NHS England Commissioning process.

As Steve Bates, ceo of the UK BioIndustry Association expressed it, the introduction of the PIM designation and the EAMS ‘shows the UK is committed to an "all hands on deck approach" to speedily progress promising innovative therapies to the patients that need them.’ However he warns that ‘as it is currently envisaged, without centrally funded reimbursement, the scheme runs the risk of being under-utilised’, undermining its attractiveness to UK SMEs and to global corporations choosing the UK as a location for their clinical trials.

‘To ensure that patients receive the next generation of breakthrough therapies in the UK, while supporting overarching industry objectives, under the EAMS companies will need to be reimbursed at an earlier stage in development at a price that recognises the uncertainty of the effectiveness of early stage products,’ he added. So this adds yet another dimension, this time economic, to the already complicated health and safety debate.

When suffering from a life-threatening disease, patients do not appreciate any obstacles that might stand in the way of them receiving a therapy that may save their lives. There is a balance that needs to be struck between health and safety in all aspects of our lives, but for such patients, where that balance falls is crucial. While small steps are being taken, this debate will continue to run and run.

Neil Eisberg - Editor

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