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Innovative Licensing and Access Pathway will smooth approval process for rare disease medicines


23 September 2021

The Lily Foundation is actively representing the interests of mito patients in a major overhaul of regulations to speed up the process of how new medicines are approved and made available through the NHS.

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Launched in January, the Innovative Licensing and Access Pathway (ILAP) aims to smooth the complex approval process that drug companies must follow in order to bring innovative treatments from 'bench to bedside'.

ILAP is being hailed as a hugely positive step for people with rare diseases that currently don't have a cure, such as mitochondrial diseases, where new and cutting-edge drugs offer the best chance of effective treatment.

The improved pathway has been put in place by the Medicines and Healthcare products Regulatory Agency (MHRA), working with partners the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and NHS England.

Described by the government as 'a new era in medicines approvals in the UK', ILAP aims to provide a single integrated platform for sustained collaborative working between the MHRA, partners and drugs developers.

Dr June Raine CBE, Chief Executive, Medicines and Healthcare products Regulatory Agency, commented: "Transforming the way innovative medicines reach patients in the UK is not a ‘nice to have’. It’s a ‘must do’. An imperative. And the time to do it is now. We are transforming the MHRA, making the regulator an enabler of innovation. Our new Innovative Licensing and Access Pathway has established new partnerships to robustly and safely support all new medicines at any point in their development, and most important of all, involve patients in all aspects of decision-making."

The Lily Foundation is one of several patient-facing charities invited to join ILAP's Patient and Public Reference Group, set up to help to steer decision-making and ensure patient needs are given priority. Alison Maguire, Head of Research and Finance for The Lily Foundation, said: "ILAP represents a timely and vital improvement to the process of making new drugs available to patients, and our charity is proud to be involved. Research into drugs to treat mitochondrial disease is at a pivotal point right now in terms of emerging treatments. Getting drugs which have successfully made it through clinical trial to patients without delay is our priority."

Commenting on the improvements being put in place, Alison added: "ILAP means that regulatory authorities are involved at a much earlier stage in the drug development process, which can take many years.  ILAP can support drug companies through the long process of bringing a new product to market. That way, when it does reach the trial stage there will be far less obstacles to getting it approved. This is great news for the mito community, because we're at the point in the development of drugs for mitochondrial disease where a smoother regulatory process is going to be vital."

How ILAP works

The 'Innovation Passport', a new medicine designation, acts as the gateway to entry into the pathway and will be awarded to innovative products submitted to the ILAP. This passport step incorporates broad and inclusive concepts of innovation and patient need, allowing the ILAP to encompass a wide range of medicines undergoing development, including Advanced Therapy Medicinal Products (ATMPs), medicines for rare diseases and repurposed medicines.

A successful Innovation Passport designation then triggers the MHRA and partners to create the “Target Development Profile” (TDP) document. This “living document” will set out a unique product-specific roadmap towards patient access in the UK healthcare system. The TDP includes access to tools from a toolkit that can be selected to design an efficient and “regulation- and access-ready” development programme. Available tools include continuous benefit-risk assessment, increased support for novel development approaches and enhanced patient engagement.

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