The European Medicines Agency (EMA) has selected six medicines to spearhead its adaptive pathways project, which is trialling a progressive licensing model for new drugs.
The aim of the project is to develop ways to accelerate the registration of new drugs – with close consultation between the regulator, drug developer and other stakeholders including patient groups and health technology assessment (HTA) agencies – so they can reach patients more quickly.
The EMA asked for volunteer companies earlier this year when the pilot project kicked off and received 34 applications with the list currently whittled down to six candidates and entries still open until February 2, 2015.
Applications included six advanced-therapy medicinal products (ATMPs) and 12 orphan drugs, while 14 of them were for new cancer therapies. Discussions have now started to see how the adaptive licensing approach can be implemented in practice.
“The first of these in-depth discussions, on the quality aspects of an ATMP, took place in December 2014, with others already planned during 2015,” said the regulator in a statement.
A report on progress with the pilot – now renamed ‘adaptive pathways’ to emphasise its role as a tool to encourage earlier dialogue between parties rather than establishing a new licensing pathway – was published earlier this month.
In the document, the EMA notes that it is considering two ways to hasten patient access to drugs. The first model would involve approval in a smaller, well-defined patient group with use possibly expanded later to a larger population. The second would involve an early – possibly conditional – approval based on surrogate markers with obligations to generate more clinical data later on.
A key part of both models is early involvement of the HTA to try to streamline the health economic appraisal of new drugs. Discussions with 13 HTAs from across the EU took place earlier this month in order to gauge their interest in taking part in the process.
“A full review of the outcome and impact of the adaptive pathways pilot project will be conducted once at least six medicines selected for the pilot have completed a procedure of parallel scientific advice with HTA bodies,” said the EMA.
A safety mechanism
The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has also been looking at adaptive licensing models and after an information gathering and sharing exercise has started preparations for its own pilot project.
Last year, UK Parliamentary Under Secretary of State for Quality Lord Howe told a workshop on adaptive licensing that if the approach is successful it will demonstrate that regulation is “not a noose which stifles but rather a safety mechanism which can release medicines to patients in need”.
The workshop – convened by the Association of the British Pharmaceutical Industry (ABPI), the BioIndustry Association (BIA) and the Centre for the Advancement of Sustainable Medical Innovation (CASMI) – discussed a number of issues related to adaptive licensing, including the capacity of regulators to tackle the extra workload.
Another concern was how adaptive licensing pricing will fit in a global product development strategy, and whether it is realistic for all European payers to accept greater uncertainty.
A long time coming
The EMA has been talking about adaptive licensing for some time – it was featured in the Road Map to 2015 document adopted in 2010 – and reiterated the need for changes to its licensing framework in the wake of the approval last year of UniQure’s gene therapy Glybera (alipogene tiparvovec) for lipoprotein lipase deficiency.
Glybera was eventually approved for a narrower group of patients than originally sought but could have been made available sooner if an adaptive licensing framework was available, said the EMA. It had been due to start a pilot in 2012 but the programme was delayed by other agency priorities.
Meanwhile, other regulators around the world are also exploring the adaptive licensing model. Singapore’s Health Sciences Authority (HSA) launched an initiative called New Drug Development Paradigms (NEWDIGS) in 2013 in collaboration with the Massachusetts Institute of Technology (MIT) in the US, while Health Canada has also been developing the concept under the banner of ‘progressive licensing’.
The community’s approach to this was clearly expressed in a recent Clinical Pharmacology and Therapeutics article. ‘From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients‘ was jointly-authored by the likes of the EMA’s Hans-Georg Eichler and Finn Børlum-Kristensen from the European network for health technology assessment and Danish Health and Medicines Authority. It also had wide-ranging industry input from companies including Amgen, BMS, GlaxoSmithKline, Novartis and Sanofi.
In it the authors examine “the changes in the scientific and political environment that we believe will make adaptive licensing the preferred approach in the near future”.
“Regulators have just begun to explicitly address and communicate ‘uncertainty’ in their templates for benefit-risk assessment,” the authors note, but acknowledge: “Further important steps towards enabling adaptive pathways are currently being taken.”
On a European level the EMA has certainly quickened its pace over the last 12 months, giving its project a real sense of momentum as it assesses adaptive licensing’s promise.