Focus Area Oncology

The Client

Leading global biopharmaceutical company, MSD.


MSD’s pembrolizumab is a type of cancer immunotherapy which has been approved in the UK as a treatment for several indications. To expand the availability of this effective cancer treatment, the team at MSD identified a lack of effective treatment options for gastrointestinal cancers. In particular, they identified a lack of targeted therapies, such as immunotherapies, resulting in a significant unmet need for patients with gastrointestinal cancers.

The aim was to develop a successful health technology assessment (HTA) submission to the National Institute for Health and Care Excellence (NICE) in the UK, for pembrolizumab’s use in treating microsatellite instability high/DNA mismatch repair deficient (MSI-H/dMMR) cancers in five sites: colorectal, endometrial, gastric and small intestine cancers and cholangiocarcinoma.

The Challenge

The challenges in this case were varied and complex. Most notably, only two other NICE appraisals have featured similar multi-tumor treatments and in both cases a Managed Access Agreement was required, limiting the initial roll-out of the treatment on the National Health Service (NHS) while more data is gathered on a treatment’s efficacy and cost effectiveness. Other key challenges included a large number of potential comparators (some of which lacked published data), low patient numbers in a number of tumor sites, and a lack of published data for the MSI-H population in general.

Finally, the pivotal trials used in this appraisal, one of which was a basket trial, were both Phase II, open-label, non-randomized studies. Therefore, indirect comparisons were required to model the survival benefit of treatment with pembrolizumab. These challenges combined to create an enormous obstacle for MSD to overcome to gain patient access to this innovative treatment.

The Approach

Lumanity’s Health Economics and Outcomes Research (HEOR) team was engaged by MSD in 2020 to support in overcoming these challenges. Our team worked with MSD at multiple stages, from global modeling through to HTA submission. This included conducting targeted reviews of previous appraisals in each tumor site separately, supporting with statistical methodology and indirect treatment comparisons, and  developing cost-effectiveness and budget impact models. The HTA submission itself posed a significant challenge regarding the effective communication of complex-to-model survival extrapolations to NICE. It was important to convey this information in a way that was robust enough for the NICE appraisal process.

The Outcome

Pembrolizumab received a full recommendation from NICE after a single appraisal committee meeting. This included all five tumor sites and was the first time NICE has approved a therapy for routine commissioning based on data from a basket trial. Patients in England and Wales with MSI-H/dMMR, who often have poorer survival outcomes than other cancer patients, will now have access to a targeted new treatment on the NHS.

“The Lumanity team was integral to the success we achieved with this HTA submission, thanks to their professionalism, responsiveness and collaboration with MSD on this highly complex project. This is a great result for patients in England & Wales, with the Lumanity team being instrumental in helping MSD to address numerous methodological and strategic challenges faced during this appraisal, enabling access via routine commissioning for five types of cancer.”

Team Lead – HTA & Outcomes Research, Associate Director

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If you would like to find out how Lumanity can help you achieve HTA success, please contact us.