The phrase ‘one and done’ has become routine vocabulary within the Tolley team over recent years and has become synonymous with Health Technology Assessment (HTA) success. In a UK HTA context, we use the phrase to refer to a National Institute for Health and Care Excellence (NICE) submission that achieves positive guidance and successful reimbursement after a single appraisal committee meeting (ACM). Beyond this, what it means to us internally is a much wider, more holistic view of HTA by which one ACM with subsequent positive guidance is the optimal outcome for both the company, NICE, and, more importantly, for patients and their families.
It would be easy to assume that technologies that receive positive NICE guidance after one ACM are simply those with the most favourable clinical efficacy data, and a submitting company with a generous pricing strategy. However, if it were that easy, we would surely see far fewer technologies requiring two, three and more NICE ACMs, but it is possible for companies to have good clinical data, and a sensible approach to pricing, and still not successfully engage with NICE at the first ACM. The reasons behind this can be numerous but we have taken our learnings from previous NICE appraisals (both those we have been directly involved in as well as those we have scrutinised as regular public observers at NICE ACMs) and devised a strategy that we believe increases the chances of ‘one and done’.
Beyond the usual requirements of evidence preparation for submission of a robust evidence package to NICE, met by following NICE-preferred methods, we encourage companies to think ahead, identify limitations and uncertainties in their own data (rather than waiting for the External Assessment Group [EAG] to do it!), plan advance mitigation, and build a positive discursive relationship with NICE to benefit all stakeholders. This usually results in a reduced back and forth with NICE and the EAG, quicker resolution of issues, and a shorter list of outstanding issues to discuss at the first NICE ACM and immediately afterwards.
Two of our most recent examples of ‘one and done’ were very different but nonetheless adopted these same broad themes:
- The first was a new technology for an ultra-rare inherited disease for which there were no prior condition-specific treatments. This appraisal was routed as a Highly Specialised Technology (HST) by NICE. Against a backdrop of multiple, significant evidence gaps due to the rarity of the condition, our strategic advice to the company was around pursuing as many different approaches to evidence identification and generation as possible, selecting the optimal but also, importantly, working collaboratively and transparently with NICE and the EAG, pre- and post-submission, to provide assurance on both sides that the most robust approach, given data limitations, was being considered. This meant that there was little, if any, room for other suggestions that had not already been considered. The company could confidently go into the first ACM knowing they had fully characterised gaps and uncertainties in their own evidence package, could discuss all the approaches they had taken to mitigation, and explain why they had chosen the approach they had. This nurtured an open and trusting relationship between the company, the EAG, and ultimately the NICE committee, with confidence in the system that the company were pursuing the most appropriate of the available approaches, rather than selecting whichever provided the most favourable result. This resulted in ‘one and done’ success and timely access to the first condition-specific therapy for a small but significant group of people living with a highly burdensome rare genetic disease.
- The second example was by contrast a new technology in a rapidly evolving oncology pathway with multiple therapies in different combinations over several successive lines of therapy, and many more in the pipeline. This was routed as a NICE Single Technology Appraisal (STA). While our advice to the submitting company was similar in terms of identifying gaps and uncertainty, mitigating risk, and reporting transparently, the key difference here regarding preparation, was taking learnings from all the previous HTAs in this therapeutic pathway, observing the issues from those appraisals, and learning from them. Many companies in this space had faced the same barriers so by predicting these and taking time in advance to prepare ways of mitigating them, this company went into the first ACM fully informed and prepared. This achieved a remarkable ‘one and done’ in this very challenging therapeutic area giving patients timely access to a new life-extending treatment as they reach a highly refractory disease status.
Some of these strategies may read as obvious, but time and time again we see patient access delayed as time elapses during multiple NICE ACMs (and even appeals) when oftentimes the issues arising could have been predicted in advance and mitigated sooner. Trying to game the system rarely, if ever, works out well for companies and so our advice will remain that with NICE, they should be aiming for a ‘one and done’ approach through advance planning, being flexible, identifying limitations and uncertainties, mitigating risk, and implementing transparent reporting of this in the company submission and other engagement with NICE.