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Date:
01-08-17

Author:
Empower

Early Access to Medicines Scheme - three years on

Early Access to Medicines Scheme - three years on

Early Access to Medicines Scheme – three years on, is it delivering on the ambitions of speedy patient access and the generation of real-world data?

The Early Access to Medicines Scheme (EAMS) reached its third birthday in April 2017. When the Scheme was opened there were high hopes that it could result in patients accessing promising new drugs for life threatening and life limiting diseases months, or even years, before they were licensed. There were high hopes too that it would offer what amounts to real-world data on the value of new drugs that could help companies navigate approval processes and secure funded patient access. Three years on, have those ambitions been delivered?

50 applications for a Promising Innovative Medicine designation

By 8 June 2017, the UK regulator – the Medicines and Healthcare Products Regulatory Agency (MHRA) - managing EAMS had received 50 applications for the first step of the EAMS scheme. The first step is to apply for the Promising Innovative Medicine (PIM) designation. Sixty-eight applications were successful, with details available on 22 PIM designations. That the full 34 drugs given a PIM are not available in the public domain reflects the confidential nature of this early stage of EAMS; the MHRA doesn’t reveal the results of PIM applications and it’s up to companies to decide if they want to publicise their PIM, or not.

Those companies and their drugs with a PIM designation in the public domain include:
• AbbVie’s Glecaprevir/pibrentasvir for patients with hepatitis C virus and Venetoclas for leukaemia
• Astra Zeneca’s Ozimertinib for non-small cell lung cancer
• Bristol Myers Squibb;s Nivolumab in five cancers
• Dompe farmaceutici’s Oxervate for neurotrophic keratisis
• Merck Sharp and Dohme’s Pembrolizumab in melanoma, non-small cell lung cancer and advanced lung cancer
• Northwest Biotherapeutics DC Vax for brain tumours
• Novartis’ Certitinib for non-small cell lung cancer and Sacubitril/valsartan for heart failure
• ProMetic’s PBI-450 for Alström Syndrome
• Pfizer’s Palbociclib for breast cancer
• Rexgenero’s Rexmyelocel-T for critical limb ischemia
• Roche’s Atezolizumab for bladder cancer
• Sanofi’s Dupilumab for severe atopic dermatitis
• Santhera’s Raxone for Duchenne muscular dystrophy
• Soligenix’s Dusquetide for oral mucosistis
• Vtesse’s VTS-270 for Niemann-Pick Disease Type C

In some cases the PIM is for sub-groups for patients. So far, cancer and big companies feature heavily in the list.

19 applications for a full EAMS scientific opinion

By June 2017, the MHRA had received a further 19 applications for the full EAMS scientific opinion (SO). Seventy-four per cent of applications were successful. This essentially means that the MHRA - after reviewing the information required from companies to support their application - believe that the benefits of the drug outweigh the risks. Patients can be prescribed drugs given the EAMS SO although their prescribing clinician will do so under the legislation that covers unlicensed use. Companies supply the drugs at no cost to the NHS.

The full EAMS SO has been given to 10 products across 16 indications, including:
• AbbVie’s Venetoclax for chronic lymphocytic leukaemia and Gelecaprevir/pibrentazvir for patients with hepatitis C
• AstraZeneca’s Osimertinib for locally advanced or metastatic EGFR T790M positive non-small cell lung cancer
• Bristol Myers Squibb’s Nivolumab in five cancers
• Dompe farmaceutici’s Oxervate for neurotrophic keratisis
• Merck Sharp and Dohme’s Pembrolizumab in melanoma, non-small cell lung cancer and advanced lung cancer
• Novartis’ Sacubitril/valsartan for heart failure
• Roche’s Atezolizumab for bladder cancer
• Sanofi’s Dupilumab for severe atopic dermatitis
• Santhera’s Raxone for Duchenne muscular dystrophy

It looks like the right drugs are being put forward for EAMS
Together, the high proportion of successful applications for both PIM and the full EAMS SO, suggests that on the whole, the right drugs are being put forward. Of course there could be other candidates out there too.

Earlier patient access, but not years before licensing
The speed of patient access for EAMS drugs is driven by a number of factors; the time that the company applies, the time that the MHRA takes, as well as the complexity of the specific drug, disease and its context. Since it matters to companies that they get paid – they are afterall commercial organisations - they are not only interested in access during EAMS but the speed at which they can secure reimbursed access. The latter relies in large part, on securing a positive recommendation from a Health Technology Assessment (HTA) agency. In England, the relevant agency is the National Institute for Health and Care Excellence (NICE).

It seems that the MHRA is speedy, with the majority of EAMS SOs within their 75-day procedure (figure 1).

Figure 1: Speed of MHRA decision-making on EAMS applications

Source: FOI response from MHRA

Access for patients can be months before licensing too – between 326 days and 18 days (figure 2) with an average of 85 days - although it’s not the years that some may have hoped for.

Figure 2: EAMS access period (days) – time between date of positive EAMS SO and marketing authorisation

Source: Analysis of MHRA data. nsclc – non-small cell lung cancer

Of the seven drugs/indications where a final NICE recommendation is available, it’s taken from 82 to 266 days from marketing authorisation to the recommendation being made (figure 3), and an average of 183 days. They have all been positive though.

Figure 3: Days between marketing authorisation and final NICE guidance

Source: Analysis of data on NICE website: TA357, TA366, TA384, TA388, TA416, TA417, TA28. ipi = ipilumimab, nsclc – non-small cell lung cancer

Real-world data potential seems limited

It’s difficult to know just how much real-world data has been collected on EAMS drugs, but an insight comes from the numbers of patients registered for EAMS drugs in England from NHSE data (figure 4). This will likely undercount the patient volumes as EAMS is nationwide, and NHSE only holds data for the drugs they commission, and not those commissioned by the local NHS.

Figure 4: Number of patient registrations for NHSE commissioned EAMS drugs, up to June 2017

Source: Data from NHSE freedom of information response

The takeaway from patient registrations seems to be that the real world data potential may be limited so far, given relatively small numbers of patients being able to access EAMS drugs. There’s little known about what this early use reveals as size is not the only relevant factor; insight can come from small numbers where they may add in terms of depth of data.

Insights and evidence could be greater in practice, but held within companies and the clinicians involved; they could unlock the potential for real world data arising from EAMS and how that can be used to inform approval decisions with the ultimate goal of getting the right drug to the right patient at the right time and at the right price. This is a potential valuable area for research; this would be relevant to all to try to optimise EAMS for the benefit of patients.

The future

EAMS appears to have bedded down within the system; in England at least based on this review. Yet questions remain about just how speedily patients can get access, how speedily companies can secure reimbursement and just what the potential is from real world data collected as part of the scheme.

Leela Barham is an independent health economist and policy expert.

Follow her on Twitter: @leelabarham

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