*IQVIA. EFPIA Patients W.A.I.T. Indicator 2023 Survey June 2024 Page 22

Key Findings

As of October 2024, 19 Orbis-designated products have been granted GB marketing authorization (MA) or had an existing authorisation extended. Interestingly, the majority (56%) were indicated for non-small cell lung cancer (NSCLC). This observed trend may reflect large pipeline in NSCLC products, the high unmet need in NSCLC characterised by consistently poor outcomes with existing NSCLC treatments and the high prevalence of the disease.

Of the 19 Orbis-designated products, NICE was less likely to provide a positive recommendation versus SMC, with 61% versus 72% of Orbis products receiving a positive recommendation, respectively. SMC was, however, more likely to not receive a company submission versus NICE, with 22% versus 11% of products not being submitted for review, respectively.

We also observed that regulator and HTA decisions were not always aligned, with more Orbis products achieving access in Scotland than in England. There were instances of two and four products that did not submit an evidence package to NICE and SMC, respectively, despite prioritisation for approval.

Another finding was that following HTA approvals from NICE and SMC, mobocertinib did not meet the approval conditions of the conditional marketing authorisation, with its MA having since been withdrawn. This case demonstrates the risks of conducting HTA for products with conditional approval, and not necessarily a risk with ILAP.

In terms of time to access, the overall mean time to decision since 2021 was longer for NICE (306 days) compared to SMC (257 days). The overall mean time to availability since 2021 were similar in England (261 days) and Scotland (257 days).

Our review observed that the timelines for HTA approval in Project Orbis (≥2022) were slightly longer in England (261 vs 235 days) and faster in Scotland (257 vs 387 days) compared to all oncology products reviewed in the 2023 EFPIA W.A.I.T. indicator survey.

Further, more recent Orbis approvals show quicker time to decision and availability by NICE compared to 2021 approvals, reflecting the first year of ILAP. However, time to decision and availability has significantly increased for SMC in recent years compared to 2021, suggesting divergent views on acceptability of evidence for Orbis products.

Takeaways

ILAP, launched in 2021, offers early engagement for manufacturers, but benefits remain unclear due to limited publicly available documentation. There is a decreasing trend in products going through ILAP, and instances of MA being withdrawn despite positive HTA indicating that ILAP participation may not guarantee commercial success . It is important to note that Orbis products may not fully represent ILAP, and transparency on non-Orbis products would aid assessment of participation benefits. Nonetheless, data available suggests it may not offer the benefits that were intended. This may explain why there is a proposal to alter the program, with recent communication from MHRA of its intention to revise the broad definition it applies to drugs entering ILAP so that the scheme resembles similar offerings by other regulators, such as the EMA’s PRIME and the US FDA’s Breakthrough Therapy Designation.

In the meantime, companies may want to consider alternative pathways to harmonise their regulatory and HTA timelines and requirements in the UK. Recent attention has also been given to the new international recognition procedure (IRP) allowing companies to request MHRA recognise the evidence submitted for products approved from one of the specified reference regulators. Note that this does not imply automatic MA and that MHRA retains the authority to reject applications if the evidence provided is considered insufficiently robust. NICE has recently posted communication on what this means for HTA. For companies seeking to benefit from the IRP, NICE encourages companies to engage early which can be done via NICE Advice, to ensure product information on UK PharmaScan is kept up to date, to regularly provide updates on regulatory plans to both NICE and the MHRA and to allow NICE and the MHRA to share operational and planning data.

Abbreviations: EFPIA: European Federation of Pharmaceutical Industries and Associations; HTA: Health technology assessment; ILAP: Innovative Licensing Access Pathway; NICE: National Institute for Health and Care Excellence; N/A Not applicable; NSCLC: Non-small cell lung cancer; SMC: Scottish Medicines Consortium; UK: United Kingdom.