A recent review of UK’s National Institute for Health and Care Excellence (NICE) technology appraisals in development reveals a remarkably sharp increase in the number of terminated NICE appraisals between 2021 and 2022 compared to the most recent ABPI analysis restricted to 2016 and 2020. Between 2021-2022, there were 39 terminated technology appraisals (or 19 per year) versus 48 across the previous 5 years (or 9 per year), resulting in double the amount of the total number of appraisals terminated annually. The recent data on terminated appraisals also reflects the growth in precision medicine and complex molecule trends in clinical development as these products highly feature in the list of terminations. Such a significant increase in terminated appraisals will mean fewer innovative healthcare technologies will reach patients treated by the NHS.

There are a number of factors that could explain the sharp increase, including the rise of appraisals that NICE must conduct, which was anticipated to have grown from 30 per year in 2015 to more than 75 in recent years. Contributing to this growth is divergence in the evidence requirements for regulators and HTA decisions – for example, regulators are, in some cases, willing to take decisions based on surrogate endpoints for clinical outcomes while HTA process often rely on clinical outcomes to estimate long-term health benefits, and companies need to ensure that their development programme is designed to cover the needs of both.  Although benefit-to-risk ratio will have been demonstrated for regulatory purposes, the evidence available at the time of the appraisal for many products leads to high uncertainty of value for money, increasing the risk of a managed entry agreement, or worse, a negative recommendation. With the UK’s decision to leave the European Union, companies will also need to file for regulatory approval with the UK authorities (MHRA). For these reasons, new policies have been implemented to provide companies the opportunity to engage with UK stakeholders at earlier points in the clinical development pathway to help manage evidentiary uncertainty and deliver financially sustainable patient access to innovation, such as the Innovative Medicines Fund (IMF) and Innovative Licensing and Access Pathway (ILAP).

These new access initiatives could also be a contributing factor to the increase in terminated appraisals. Some companies, including some routed via the ILAP, may find themselves undergoing the NICE HTA process with uncertainties, suddenly realising their product is on the path toward the IMF or Cancer Drugs Fund (CDF) without perhaps having considered the consequences. After reflecting on the risks and ongoing responsibilities, companies might decide to terminate the appraisal due to financial risk of an IMF/CDF recommendation or the consequences a negative NICE recommendation might have for other HTA markets. This emerging trend might be signalling the missed opportunities to engage at an early phase of the development as many of the ILAP products approved by the regulator so far were late in their development. Hence a review of the products that have been approved in ILAP in terms of how they have fared at NICE is warranted.

The right market access strategy for an innovative medicine may include the ILAP and/or the IMF. Preparing to engage with new policies requires careful planning in the very early stages of clinical development, involving multiple internal stakeholders including medical, market access, health economics, clinical development and regulatory teams, some of which may have limited time/resources to commit to discussion at such an early stage. Companies planning to engage with UK stakeholders through the ILAP process first need to define which pipeline products would such a process bring value. Defining the attributes of an ILAP-appropriate product is complex and requires broad internal endorsement. For those products deemed appropriate, further alignment on the evidence generation strategy and the relevant tools for engagement is similarly complex, requiring muti-stakeholder agreement. For products where the right market access strategy does not include ILAP but might include the IMF or CDF, a similar approach is required, involving multiple stakeholders to align and endorse the evidence generation strategy early in the process.

To support companies developing innovative medicines across a broad range of therapy areas, IQVIA’s Accelerator for Innovative Medicines (AIM) (Figure 1) services addresses a key set of UK access and evidence needs for its clients by providing cross-functional support throughout all clinical development and market launch phases drawing from its local UK expertise in market access, health economics, real world evidence generation, regulatory affairs, and commercial services tracking access and uptake. Through AIM, IQVIA provides support at the following phases:

Phase 1: Pre-NICE engagement –

Plan Early: During R&D phase, pro-actively plan your local HTA, RWE, pricing and access strategy for the coming years. Understand the options and navigate access strategies, with the primary aim of avoiding the IMF/CDF route and proceeding to routine commissioning. Consider whether ILAP is right for your product.

Phase 2: Preparing for NICE & MHRA engagement –

Engage pro-actively: Prepare for NICE discussions with our years of proven experience, and provide strategic options to meet and shape NICE criteria. In this phase, it is important to outline RWE approaches for data collection. As experts in RWE data sourcing, study design and analysis, IQVIA can provide a market-leading options in primary data and secondary data. If ILAP is an option for your product, IQVIA can also provide an experienced multi-disciplinary team of local NICE, MHRA regulatory and market access experts to support the Innovation Passport (IP) application process and preparation for stakeholder engagement beyond the IP phase if successful.

Phase 3: Negotiation –

Informed negotiation: Leverage the strategy developed in Phase 2 to maximise consensus on an achievable Data Collection Agreement (DCA) and a reasonable Commercial Access Agreement (CAA). Establish the RWE programme: Activate your RWE programme with NICE, data controllers, study teams, external stakeholders and operational / contractual management

Phase 4: Resolving uncertainty –

Efficient evidence generation: IQVIA’s set of solutions can improve the timeliness, quality and value of evidence generation to support your innovative medicine. Informed negotiation: IQVIA’s team of HTA experts can ensure your value dossier and NICE negotiations put your medicine in the best position for routine reimbursement post-IMF/CDF.

Phase 5: Medicines update  –

Maximise your medicine: In response to NICE IMF/CDF guidance, IQVIA can help maximise the access and impact of your medicine. This is through not only data-driven monitoring of your medicine but also optimisation of your internal resource and the healthcare system pathways which your medicine needs to impact.

For companies considering whether the ILAP and/or IMF is right for their products, IQVIA can leverage its connected capabilities to facilitate internal alignment and endorsement of the decision to pursue the ILAP or IMF/CDF process by helping companies to answer the following key questions:

• Is this process appropriate for this product?
• What evidence gaps exist and can the gaps be addressed within this process?
• What are the perceived benefits and challenges of engaging with this process?
• What are the financial risks and wider risks in pursuing this process?
• What other access strategies should be considered?

IQVIA is committed to supporting its life sciences partners in ensuring patient access to innovation through its tailored regulatory, market access, HTA, RWE and commercial solutions via AIM. For more information about IQVIA’s AIM services, please contact askiqvia@iqvia.com.

FIGURES FOR AIM:

Figure 1. IQVIA’s Accelerator for Innovative Medicines (AIM)