Tumour agnostic or histology-independent therapies target the genomic alteration within a tumour, regardless of where in the body it has formed. Currently, there are two tumour agnostic therapies being assessed by the European Medicines Agency (EMA), Bayer’s larotrectinib and Roche’s entrecitinib, both of which are expected to receive marketing authorisation later in the year. These drugs target neurotropic tyrosine receptor kinase (NTRK), a genetic alteration most commonly found in rare cancers such as salivary tumours and infantile fibrosarcoma, but can also be found in low levels in the more common cancers. There is no doubt that affordability remains a key factor for NHS England and healthcare services, and a high price coupled with the uncertainty of long term efficacy for the variety of tumour types will be the greatest challenge for tumour agnostic therapies in gaining market access. However, these types of therapies face additional challenges in adoption due to how these new technologies will be assessed for value and reimbursement by the Health Technology Appraisal bodies (historically existing cancer drugs need to be approved by NICE for each individual type of cancer they treat). The key issue is the differential expression of these mutations across tumour types combined with differing outcomes of treatment, depending on the site of the tumour. Both factors mean that determining an ICER, and therefore a price, across tumour types is challenging. The NHS also needs to ensure that the right diagnostic pathways are in place so that the patients most likely to respond to these treatments are identified and able to gain timely access. The rare incidence of these cancers poses an added challenge where patient populations and clinical programmes are naturally smaller making it harder to demonstrate meaningful value with limited evidence.
The new NHS Genomic Medicine Service is expected to provide the vital infrastructure for testing, ensuring patients can be identified and tested for the NTRK gene. The Accelerated Access Collaborative is expected to discuss how it can also support the implementation of these new treatments.
There is scope for innovation in pricing. With good, proactive engagement, pharma has an opportunity to partner with payers to co-create different pricing and contracting models such as pay-for-performance contracts or rebate contracts alongside real world evidence collection as strategies to mitigate the uncertainty and high costs.
With earlier detection and treatment of cancer forming a central part of the NHS Long Term Plan for the health service, timely reimbursement and access to these therapies will be another welcome step forward for precision medicine and personalised patient care.