The tricky second album. From RCTs to RWE, does industry have the right mix for clinicians tastes?

Transcription

1 The tricky second album From RCTs to RWE, does industry have the right mix for clinicians tastes?

2 FOREWORD INTRODUCTION Real World Evidence (RWE) is becoming a significant new currency in healthcare but is industry in danger of aiming this tool at the wrong problem? Each year billions of dollars are spent on randomised controlled trials (RCTs), testing that new medicines work (at least as well as current options) and that they are safe. Researchers are largely in the driving seat, focused on ensuring that products can stand up to intense scrutiny from regulators before their discoveries are licensed for sale. Elsewhere pharma colleagues examine the same data, suggesting additional elements and analyses to be conducted within the RCTs, aiming to build a case demonstrating the economic value of the new medicine, in search of the holy grail proposition for a new therapy that it not only has superior efficacy, but will reduce system costs if licensed. RWE takes this exploration of efficacy, safety and value one step further, potentially proving that, over time, a medicine does indeed deliver the benefits both in terms of patient outcomes and economic value that had been promised at the time of launch. In this context, the industry can demonstrate that a medicine offers further clinical effectiveness in real-world patient groups, confirming its performance and value in patient populations that have more complex health needs and comorbidities than RCTs allow, in the hands of prescribers working without the close scrutiny of RCTs. The fundamental role of the modern healthcare payer, to drive efficient healthcare decision-making based on value-for-money, demands that new medicines demonstrate richer, more clinically relevant evidence. RWE can be part of meeting this need as well as confirming the findings of RCTs. However, RWE can also go much further than informing a debate about whether, in principle, a treatment should be made available by a healthcare provider. RWE offers the depth and range of data required to inform decisions around medicines broader usage in the clinic, based on patient preference, for example, and around its performance in practice. But does it currently provide clinicians with the information they need? And are they willing to use it to inform their practice? New research from MHP based on a survey of General Practitioners (GPs) in the UK shows that communications can have a vital role in ensuring the full potential of RWE is realised. By maintaining trust in its robustness, and ensuring it is accessible, understood and aimed at highlighting use in practice (not just confirming efficacy and value), RWE can be transformed from the tricky second album to the cornerstone of pioneering clinical care.

3 UNCOVERING THE VALUE OF REAL WORLD EVIDENCE UNCOVERING THE VALUE OF REAL WORLD EVIDENCE In theory, theory and practice are the same. In practice they are not Albert Einstein In the eternal quest to get from bench to bedside, faster, the challenge that lies between licensing and widespread use has become as significant as optimising the process of trials and regulatory review. National initiatives such as the Accelerated Access Review in the United Kingdom have been established to explore this dilemma at length 1. RWE is not yet embraced as an integral part of informing or shaping clinicians prescribing decision making. The question is - does it have the potential to be? A new survey of 1,003 general practitioners (UK primary care physicians) commissioned by MHP looked to explore two potential barriers: Do clinicians value RWE enough to actually use it? Are we asking the right questions of RWE to generate the data clinicians and commissioners really need to inform their practice? Do clinicians value RWE enough to actually use it to change their practice? Are we asking the right questions of RWE? 3

4 RWE CAN CHANGE PRESCRIBING PRACTICE RWE CAN CHANGE PRESCRIBING PRACTICE GPs say that RWE has an important place in validating phase III trial data (27 per cent identify this as the key use of RWE). However, other uses are important to them with validation of phase III trial data coming second out of the five most valuable factors overall. Primary care clinicians want to use RWE for an array of purposes, ultimately helping to inform their decisions about prescribing something that RCTs aren t designed to provide. Despite the recent shift in focus, putting more priority on payers than has historically been the case, changing prescribing habits remains a tough nut to crack. So industry investment in rich data, and ensuring that this is scoped to inform these decisions, will be money well spent. Almost a third of GPs (30 per cent) say that RWE is most useful to them when it identifies how best to fit a treatment into the current protocol, including when to switch patients to a new treatment. This is significant as data available on new treatments doesn t necessarily guide decisions on medicine switches. Clinicians are required to take a trial and error approach with their patients, and most will wait for early adopters to share their experiences. Having a record of which medicines worked well, how, and in which patients, is valuable data which allows for informed decisions about efficient treatment choice, and can speed up the sharing of experiences meaning that more patients can appropriately access treatment earlier. One in four general practitioners (25 per cent) say that RWE is most useful when it shows how medicines are being used by their peers in a clinical setting again pointing to the role of RWE in independent validation and peer communication. Only one in ten GPs identify the patient s treatment preference as the most useful type of real world evidence (nine per cent) and only one in twenty cite adherence rates in a clinical setting as most important (six per cent). However, while these aspects aren t highly regarded as most useful, general practitioners interest in them is substantial, with around half (46 per cent and 47 per cent respectively) ranking these aspects in the top three of the five types of real world evidence tested. These findings stress the real interest in information that can support personalised prescribing and patient centred care. With a very high proportion of general practitioners acknowledging the usefulness of RWE (only three per cent say it is not useful to them), its value is evident. The spread of factors that these clinicians feel RWE supports are a key message to those investing in developing databases, and communicating datasets. RWE can have the power to help inform prescribing, as well as decisions around switching medicines, and offers independent validation of medicine usage in the clinic. 25 per cent of clinicians find RWE that shows how a treatment is being used by their peers in a clinical setting to be most useful to them, of the types of RWE tested. A quarter of GPs say that RWE that validates phase III trial data is the most useful to them 27% One in three clinicians say that RWE is most useful to them when it identifies how best to fit a treatment into the current protocol, including when to switch 4

5 OPPORTUNITIES FOR RWE IN NON-SPECIALISED CARE OPPORTUNITIES FOR RWE IN NON-SPECIALISED CARE It is clear that RWE will be welcomed to support clinical decisions in real care settings but there is a risk that a payer engagement-led approach will aim RWE at a narrower set of questions than it is able to answer. Health systems, industry and the policy community need to reflect on where and how RWE should have impact. Around the world health systems are struggling with rising demand and increasing treatment options that need to be evaluated and prioritised for reimbursement. Increasingly RWE is looked to in the area of specialised treatments with the aim of identifying the value delivered from a new treatment, with companies required to collect data while products are used under access schemes. Rather than focus on these specialised treatments our research asked clinicians to consider asthma and other long term conditions when completing the survey. Reflecting on the findings we suggest that the industry should be cautious of RWE becoming dominated by specialised care thinking, and more open to the opportunity they hold for more common conditions. Case Study Asthma is a condition treated mostly in primary care. Outcomes have stagnated over recent years, with relatively few medicine advancements in the past few decades. While some new medicines are being developed for rare, severe asthma offered in highly specialised tertiary settings this is not the case for people with mild to moderate asthma. Meanwhile as many as 70 per cent of patients are not adhering to their medication 2 and, as a result, three people die from asthma in countries such as the UK every day 3. RWE has much to offer here. Beyond unlocking more information on value of products, key challenges of long term conditions can be better understood, and different approaches to tackling them evaluated and shared. RWE can turn anecdote into evidence, and evidence can inform action. One in twenty GPs say that they find RWE that shows what the adherence rates are in a clinical setting to be the most useful type of RWE to them 5

6 COMMUNICATING TO REALISE THE FULL POTENTIAL OF RWE COMMUNICATING TO REALISE THE FULL POTENTIAL OF RWE This research challenges some assumptions about RWE. It is an essential piece of the medicine puzzle, not just offering ratification of trial data or support for a price point in the market. Attention is needed on how we collect, share and promote understanding of the data to realise its full potential. RWE uncovers more about the performance and usage of a medicine than any RCT could, provides rich insight into how individual patients respond, and shows how health systems fare when treatment pathways are overlaid. Importantly, this research suggests that there may be a real appetite from clinicians to genuinely apply its learnings in the clinic. So what is stopping GPs from accessing the data and using it to transform their practice? First it is important that the data is robust. This research suggests a high level of trust in RWE. While only 30 GPs from the entire sample of 1,000 said RWE isn t useful to them (three per cent), 26 of those say they would find it useful if it were peer reviewed. As RWE develops, trust must be maintained and it is essential that wider communication of the data, including increasing numbers of analyses performed on datasets, does not lead to a reduction in perceptions of robustness. Second, and we believe crucial, are the issues of message and messenger. RWE moves discussion about new medicines on from the does it work? of RCTs, through does it work in practice? and how much better is it in practice? to what our survey suggests is the key question of how do I use it?. Communication from any stakeholder looking to inform prescribing decisions needs to embrace this and talk more about practice rather than theory. For advocacy organisations, and anyone working in partnership with them, this requires a significant shift to ensure that RWE is accessible, understood, and turned into clear information and relevant, feasible asks. As questions about new medicines evolve in the light of RWE, patient organisations need to continue their evolution from we want to we know. A shared commitment to building the data and skills to understand and use RWE offers a real opportunity to change behaviours to the benefit of patients. Primary care clinicians are more than ready to listen; those who would speak need to step up. 6

7 REFERENCES REFERENCES 1 UK Government, Accelerated Access Review, Available from here: Accessed October R Horne, Compliance, Adherence and Concordance: Implications for asthma treatment, Asthma UK, Asthma Facts and FAQs, Available from here: Accessed October 2015 About the survey The survey was commissioned by MHP and conducted by ComRes in August The aim was to uncover clinician insight into how useful general practitioners find RWE to be. ComRes interviewed 1,003 UK-based general practitioners online between 12 and 21 August Quotas were set to ensure a representative spread of general practitioners across the former SHA regions of England, Scotland, Wales and Northern Ireland. ComRes is a member of the British Polling Council and abides by its rules. MHP Communications is a trading division of Engine Partners UK LLP, a limited liability partnership. Registered in England & Wales No. OC Registered office 60 Great Portland Street, London W1W 7RT, United Kingdom. List of members names open for inspection at registered office. 7

8 6 Agar Street London WC2N 4HN