June 2016 – by Sarah Garner

As the keynote speaker in the EMIF Conferences, E-managing the Future of Health Data, Dr Sarah Garner spoke on a number of initiatives making use of real world evidence—specifically highlighting the significance in RWE in furthering the conversation on MAPPs. Here, she reflects on how the opportunities changes in technology allow us today and how initiatives like ADAPT SMART are paving the way for MAPPs.

I first visited Hungary as a pharmacy student in summer 1991 shortly after the last soviet troops had departed. Two friends and I arrived on an overnight train from Yugoslavia with our lonely planet guide heading for the tourist office. There was no Internet, no mobile phone, and we didn’t have a credit card or a word of Magyar between us. How very different things are now. Technology has enabled so many things making our lives easier in some cases and harder in others! We book hotel rooms and transport online, purchase goods, conduct our work and social lives and so many other things with a keystroke. And now we are at the next frontier: improving our health.

I’ve worked for NICE now for 16 years. In my experience, most of the issues we see with HTA decision-making are due to the unintended consequences of choices that were made during the development of new pharmaceuticals in a system that was driven by regulatory goals, particular in the US. There is nothing worse than being involved with an appraisal where the evidence standards have not been met. The case has just not been proven. This led to our efforts to offer scientific advice, which provides insights from our unique vantage point between the regulators and the healthcare systems with a clear view both upstream and downstream. We know intimately the difficulties that companies face with development. Also led to our new offering – the office for Market Access to work with companies on strategies to access the UK National Health Service.

One aspect of my role as head of the science policy and research team is to work with partners to discovering new, efficient means of harnessing existing health data for better decision making and assessment of new therapies. We want to help pull those game-changing innovations through the system.

“Advances in science and technology are there—It’s up to us to keep up with them”: Sarah Garner, PhD, MPharm, Associate Director – Science Policy and Research, National Institute for Health and Care Excellence (NICE)

NICE is participating at the EU level with a number of projects from the Innovative Medicines Initiative dealing with MAPPs, which aims to provide timely access to specific medicines, with a potential to address unmet need in specific, well-defined, patient populations, in a sustainable way. ADAPT SMART is one example of note. This unique partnership with 32 EU stakeholder organisations (between patients, regulators, payers, HTAs, industry and academics) aims to find consensus on how the levers in the existing system can be aligned to make the development process more efficient.

ADAPT SMART aims to harness adaptive pathways to foster access to beneficial treatments for the right patient at the earliest appropriate time in the product life span in a sustainable fashion. In doing so, it needs to tackle the vital question: “How can health care decision-makers best achieve balance between early access and safety?” MAPPs is not about rushing to expose patients to unused treatments. It’s essential to gather sufficient data that shows the benefits of a new treatment will very likely outweigh the risks in a patient group. The regulatory seal of approval can only be given to medicines that have been thoroughly assessed and found to have positive benefit-risk for a defined patient group. With targeted development of treatments, we can see smaller, faster clinical trials and earlier results. When combined with closer interaction and faster assessments by decision-makers, we can realise the ambition of getting new needed medicines to patients sooner. However we must be able to negotiate sustainable market access agreements and ensure appropriate use and safety monitoring.

An important feature of MAPPs is the acknowledged need for additional learning following an initial market launch. Putting in place a plan for post-licensing knowledge generation will require the support of a “life span” approach to learning, with the license and reimbursement conditions of a product being reassessed and revised continuously, with additional knowledge. Such an approach requires the participation of many players—which is why it’s essential that initiatives like ADAPT SMART bring many diverse players to the table up-front.

At this point, MAPPs is a learning, living project. I look forward to giving my input from the HTA point of view into initiatives like ADAPT SMART and to help these collaborative initiatives can accomplish their goals. Further, I look forward to seeing the science and technology that allow us to even have such discussions will bring. The advances in technology and science are happening—it’s up to us to harness them.