The Value Manifesto: 20 Actions to Improve Access for Patients (#20V4P)

In recognition of GalbraithWight’s 20 year anniversary in 2017, having spent 20 years dedicated to global strategic market access, we have set out our Value Manifesto; 20 key points under five major domains to help guide & improve access to innovative healthcare interventions & deliver better health outcomes for patients.

Why focus on Market Access? For three specific reasons;

1.    Market Access is the single most important determinant of commercial success for life science businesses – success which delivers value to patients, employees, shareholders and investors.

2.    Market Access is the process by which patients gain access to improved health outcomes – the most important reason for the existence of the Pharma, Vaccines, Biotech & MedTech businesses. By helping healthcare professionals to deliver better health outcomes, country healthcare systems create a more healthy population which drives economic activity & reduces overall healthcare & societal costs.

3.    If Pharma companies, Governments & Payers continue conducting market access processes in the same old adversarial way, healthcare systems will grind to a halt, patients will not get the treatments they need and deserve, and the innovative Pharma & Biotech industry will, ultimately, fail.

Market Access delivers real benefits to patients, healthcare professionals, healthcare systems, country economies, life science business employees, shareholders & investors.

A. Define & Communicate Value

1.     Define Value: One of the biggest challenges we have around ‘value’ is that we’ve not really defined value properly, so it remains elusive. What really constitutes value? In oncology, is three months extra survival ‘value’? Perhaps not so much now in Her2+ breast cancer – but definitely yes in pancreatic cancer. So perhaps it’s different for different circumstances? How much do patients really value the outcomes improvements we deliver? How can we build a better case for more focus on the societal value of healthcare? If we can help someone get better, and they return to work, or we prevent a child from being sick and so avoid their parents taking time off work, there is a direct positive economic impact – yet most Payers around the world say that this should not be counted as value, which is clearly ridiculous. We need to do a better job in defining what value really means to patients, caregivers & society, & how to measure & calculate that value – not just the ‘easy’ value metrics.

2.     Communicate Value: Having established what ‘value’ really means, we need to communicate it well, so that all stakeholders understand exactly what we mean by value. Curing hepatitis C in 12 weeks in a limited population (there is a finite number of people who get hep C each year) therefore limiting & containing the greater impact of the disease, is something that should have been communicated much better about the value of Sovaldi rather than the sole focus of attention on the price.  It is not enough to define and communicate value – we need to go even further and make sure all our stakeholders; inside & outside our organisations, really understand, and most importantly, believe in the ways we calculate value, and as a consequence, take action to adopt innovative healthcare interventions which deliver value.

3.     Be Clear About Value Strategy & Create Compelling Value Propositions: Many Value Propositions & Value Stories we see in companies really are not fit for purpose. The front end of the Value Proposition is the most important step; be crystal clear about the specific healthcare problem, from the Payers’ perspective, your brand is going to fix, with clear, evidence-based metrics about the patient population, the current Standard of Care & the associated direct & indirect healthcare & societal costs. If there is no real problem to be fixed from the Payer’s perspective, your brand is irrelevant.

B. Improve Value Measurement

4.     Improve HTA Methodology: The methodology around HTA is constantly evolving as we learn more about how to conduct HTA. However there are still many improvements needed in the methods used among the national & regional HTA agencies around the world, and among the Value Frameworks which have been developed in the US. Many of the existing methodological shortcomings are driven by expediency – as, to do a better job would require more effort. For example the use of cost per QALY measures may be perfect for some situations such as long term therapies for younger patients with chronic conditions such as asthma or diabetes, but the same measure inherently discriminates against those therapies for patients with a short time to live, such as for late stage disease. In addition it has been shown that the ~￡20k threshold set (and the new proposed ￡100k threshold for rare diseases) by NICE in England is entirely arbitrary, has never been independently validated by the public (who pay for NICE and the NHS through their taxes), and has not increased for 15 years, thus effectively reducing the threshold in real terms. Using the same tool for every situation in every disease is unlikely to be an intelligent decision; a rigid adherence to one solution is pure dogma. While industry (individual companies and the industry associations) has spent plenty of time moaning about the methodology and the conclusions drawn from it, they have done little, if anything, to suggest better alternatives other than to try and turn back the clock. It’s time to stop whingeing about it or wishing for the past, and come up with better solutions.

5.     Improve Economic Modelling: Many of the economic models we see today are ‘standard’ out-the-box solutions which often fail to really capture the true value of an asset. Reading through a selection of NICE ERG reports on Pharma company models yields a fairly scathing analysis of the methodological integrity of many of them. Researchers including Bengt J?nnson1 have shown that if we examine the contribution delivered by innovative medicines over their true period of utilisation (often decades past loss of exclusivity) then society is invariably the beneficiary. In addition researchers at the OHE have demonstrated how this aspect can be integrated into model development2. If we look at R&D pipelines, we see many major innovations coming through from gene therapies, CAR-T, TCR which will deliver potentially ground-breaking improvements from one-time interventions, delivering potentially decades of benefit. Some of these innovative therapies generate a long tail in their KM curves. Therefore our statistical skills need to be at a much higher level to ensure these benefits are being extrapolated appropriately to express the real value being delivered.

C. Improve Governance of & Capabilities within HTA Agencies

6.     Define & improve competencies within HTA Agencies: Having met many people working in HTA agencies around the world, I know they are staffed by dedicated, hard-working people who do their best to ensure that the work they do has the best intentions for patients at the core of what they do. I also know that these individuals are under increasing pressure as the workload & scope increases. As HTA agencies grow in size & scope, what capabilities should they have? Who decides that? And who measures whether they have these capabilities? The time has come for a more transparent assessment of the capability requirements within HTA agencies, whether at national (such as G-BA) or regional (such as Agència d'Avaluació de Tecnologia i Recerca Mèdiques de Catalunya) level. Therefore we need an international initiative focussed on first defining, and then assuring those capabilities throughout HTA agencies. This, of course, also needs to extend to those private companies who wish to fulfil a similar role – such as ICER in Boston, MA. While the current EUnetHTA JA3 Workpackage 6 covers quality standards, it focusses on SOPs rather than competencies.

7.     Increase governance of HTA agencies: We know that the workload within HTA agencies is increasing, as more of them are started up around the world, and as they expand their remit. Given the importance of the decisions made by HTA agencies, it is vital that those people within these agencies use appropriate evidence-based scientific methodology, and have the appropriate level of skills, knowledge & experience to conduct these technical appraisals. Right now, there appears to be little, if any, oversight. It is vital to ensure that there is transparency & public oversight of the methods & key capabilities & competencies of those making these potentially life-or-death decisions. A key initiative may be to call for increased governance and standards across HTA agencies & organisations.

8.     Define the scope & remit of HTA agencies: HTA agencies have been evolving since 1986 with the role of PBS in Australia, & in 1987, SBU in Sweden. So the idea has been around at least 30 years. What we have seen over this time is that HTA agencies are very good at expanding their remit. For example, NICE began in 2000 by looking at some new medicines, and now has a much wider scope in healthcare interventions, including medical devices & vaccines. They also developed NICE International; a service which helps the development of HTA agencies around the world, particularly in developing countries. Let’s be clear, nobody anywhere in the world, in any country, ever voted for HTA. So while there is clearly a desire among country healthcare systems for HTA, there also needs to be a public debate about what is actually desirable for the public who pay for it through taxes & health insurance premiums. HTA needs to be seen as a useful & meaningful exercise which brings value to patients by improving health outcomes.

9.     Teach HTA Agency staff about clinical development: An important area of market access is the development of appropriate evidence to demonstrate value; whether in clinical, economic or societal terms. The way much of this evidence is developed is through clinical trials, during the clinical development process. One of the key aspects is that the data from clinical trials evolves, from early response rates, into more mature outcomes such as, for example, progression free survival & then overall survival in cancer trials. From many appraisals conducted and opinions stated publicly by HTA agency managers, it is clear that while some individuals have a great understanding of clinical development, this tends to be the exception, and there is sometimes a lack of understanding about the evolution of data from clinical trials, or the clinical value of some of the early outcomes, and the relationship between some of these early outcomes with more mature patient outcomes. For example, there is a public disagreement between G-BA & IQWIG in Germany over the value (or not) of progression free survival. Therefore it would seem a sensible course of action to ensure those in HTA agencies involved with the technical appraisal process are more familiar with how clinical trials are constructed, how patient outcomes are measured, and how the data evolves, so they understand the evolving nature of data sets, and the value of these outcomes. An educated evaluation is more likely to provide better outcomes, especially for the patient.

10.  Align Payer & Regulator Positions on access to innovative medicines: FDA (with Breakthrough status), EMA (through PRIME) and PMDA (through Sakigake) have initiated mechanisms to bring therapies which appear to offer real benefit in areas of significant unmet need to patients more quickly. The reality (user experience) of joint early scientific advice & the new adaptive pathways process is generally positive for the quality of responses from EMA but generally a very poor experience for the quality of HTA feedback. This again raises the question on whether this array of small HTA agencies really has the competency for this kind of work. There is an urgent need for Payer organisations to adopt a much more open mind to finding solutions when regulators are trying to get innovative medicines with real promise to patients quickly rather than stand in the way.

D. Improve Industry Capabilities for Market Access

11.  Don’t Silo Market Access!: The Pharma industry has an unparalleled successful track record in its ability to silo functions. I have witnessed arguments in major companies between Commercial & Medical & between HEOR & Market Access about who is ‘in charge’ of market access. These are all completely futile. Market Access is not a department, it’s a mind-set; a way of building the value case for a healthcare intervention based on the evidence of the value it delivers for patients, for healthcare systems and for society. Yes, of course we need highly qualified technical experts in health economics, outcomes research, value strategy, value communication, as well as functional expertise in Clinical Development, Medical Affairs, Regulatory & Marketing. However trying to value one over another has no place in gaining access to medicines for patients. What is much more important is close collaboration between each group of technical experts with a joined-up approach. Market Access has maximum value when it is fully integrated into the core business functions of R&D, Launch Excellence, Strategic & Tactical Brand Planning, Business Development & Licensing. So don't even think about siloing it.

12.  Build Cross-Functional Expertise: Market access is not a department; it’s a set of cross-functional capabilities which everybody needs; from R&D to Commercial. Therefore we need to ensure there are clear Market Access competencies identified based on the specific role all individuals may play in their contribution to Market Access, and then effective programmes developed & delivered to build those competencies through a combination of formal, social & experiential learning.

13.  Integrate Market Access into Core Business Processes: We witness many instances in companies where business processes such as clinical development, brand planning, strategic planning, annual financial budgeting & market access, are run as completely separate exercises, with the consequence that these are not aligned. The way to get the best solution is to integrate Market Access fully into cross-functional processes; clinical development, brand planning, strategic planning & annual financial budgeting.

14.  Start Earlier (much earlier): The single most common problem we see across the vast majority of companies is that market access planning & implementation takes place far too late in the development process; so not soon enough to have a real impact on value. This means there is often a lack of preparation and lack of appropriate evidence at the time of launch. We hear feedback from companies saying they don’t want to invest too much before they know the Phase III results in case they waste money. Value is created throughout the clinical development process. Brands with FDA Breakthrough status may file from Phase II trials, and so will come to market much more quickly. This means market access work has to start at the very beginning of clinical development, with market access requirements built into clinical trials from the very beginning, plus a clear plan for how real world evidence will be built to demonstrate value in more heterogeneous patient populations. Start now, no excuses.

15.  Develop Key Performance Indicators (KPIs) for Market Access: Missing from almost every market access plan are a clear set of KPIs. The view we hear from many is that you can’t measure market access until after the event (reimbursement yes or no). This is just not true. While a set of lag (rear view mirror) indicators is relatively easy to construct (did we get the price? the level of reimbursement? for the right population? for the right place in the treatment algorithm?), lead indicators (satnav/GPS) are more difficult, but all the more important to do. Lead indicators are the steps leading up to a decision, and more often than not require the measurement of beliefs & attitudes – activity missing from the majority of market access plans we see. Building a set of lead indicators can help you determine how likely you are to succeed – so if you see a lead indicator being missed, you have time to address the issue before it is too late. Lead indicators include the level of agreement with your Payer stakeholders on Standard of Care & comparators, the design of the clinical trial, the power of the statistical plan, the type of economic model to be built, the clinical outcome measures to be studied, the validity of patient outcome measures (PROs) to be studied, the measurement of utilities, and so on. These need to be measured and reported, with action taken where any are adrift from the plan in terms of addressing Payer beliefs & behaviours.

16.  Senior Leadership Advocacy, Action & Investment: One of the aspects missing in many companies is true advocacy, action & investment in market access from the most senior leadership in the company. Because many of them built their careers during times & in countries where market access was less developed, there is often a misunderstanding of exactly what is required. Many still regard market access as post-marketing authorisation activity to achieve national pricing & reimbursement/coverage, or the provision of a health economic model to show (or not) cost-effectiveness. Because Payers will revisit decisions as market changes occur, Market Access is for life, not just for launch. There is much less understanding of when to start market access (from entry into the clinic) and even less about the investment necessary throughout the development process, with many reluctant to spend money on market access ‘in case the asset doesn’t make it’ – which is a bit like saying ‘we shouldn’t spend money on R&D in case we don’t find anything’. There is also misunderstanding about market access capabilities & responsibilities throughout the company; we have seen senior leaders say ‘market access is so important, I hired someone (i.e. one person) to take care of that’. Job done. We all have a duty to educate senior industry leaders about the true scope & impact of market access, which will in turn enable them to set the right tone among the different functions involved to work collaboratively, and ultimately to make the right decisions about strategic capabilities & investments, and so guide the company on the right path to success.

E. Get Pricing Right

17.  Stop Pricing per mg Chemical: Pricing per mg chemical has no basis in fact or logic. The reason why we continue to price by mg is because industry has been doing it the same way for over 100 years. It’s made even worse by today's HTA processes which aim to fix the lifetime price of innovative medicines across all indications at the time of launch of the first indication which makes no sense, & encourages Pharma to maximize launch price. What is needed is a much more dynamic approach to pricing, which can only be achieved by changing the mindset & process around price setting.

18.  Learn About Pricing From Other Industries: Thinking about how other industries have evolved their pricing models, we could learn from the software industry & the music business. We no longer own a ‘physical thing’ when we purchase software or music – we pay a monthly licence fee or a monthly subscription to have access to the product. We pay to have access to the service, not to own a 'physical thing'. In a similar way we could price innovative therapies based on the same idea; a monthly licence fee, for the duration benefit is delivered (for example per month of progression-free survival or overall survival in oncology) irrespective of mg of chemical required. This means we would no longer discriminate against high BMI patients who require bigger doses. It also means that affordability is addressed, as payment is linked to the duration of benefit, rather than all up front with high short term budget impact. It also means much greater predictability of budget impact (as the fee is fixed and known) – something Payers crave. By making the licence fee patient specific, it would mean that indication-based pricing, based on value delivered would be possible, as well as the collection of prospective outcomes data to ensure the value promised is actually delivered. The most important thing to get your head around is that nobody ‘buys’ a medicine anymore, they do not ‘buy’ a physical thing like a pack or a vial. This remains the property of the Pharma company. The license fee is for access to use the medicine for a specific patient – not to procure mg of chemical. It will be a brave move for the first company that does this, and it’s more likely to be an entrepreneurial biotech – now’s the time to be bold & challenging.

19.  Implement Innovative Anti-biotic Pricing to Stimulate Antibiotic R&D: - By adapting the licence fee model described in the last point, a license-to-use fee could be developed for antibiotics developed for severe life-threatening infections. This model works well for antibiotics, where volume use will be tiny (which is desirable - in line with antibiotic stewardship policies). By basing the price on a licence-to-have -access, rather than volume used, we no longer have the perverse negative incentives currently in the system which has shut down some antibiotic R&D. No one single company can take the responsibility for doing this alone – there needs to be a consolidated effort among companies & academics to develop this pricing method.

20.  Be Realistic: Finally, as one head of R&D once told me, “if you have lemons, make lemonade”. Don't pretend your asset is something it's not - make the most of the virtues it has, or what you could make it have, & price it accordingly. Trying to over-price a mediocre-performing asset reduces the headroom for the real breakthroughs, and reinforces all the negative views people have about the Pharma industry. Pricing an asset on it’s true value means it reaches patients, and that’s the only thing that really matters.

Colin Wight, President & CEO, GalbraithWight on LinkedIn

For copies of The Value Manifesto (#20V4P) contact GalbraithWight

Copyright GalbraithWight Ltd

References:

1.    Cost–effectiveness of statins revisited: lessons learned about the value of innovation. Peter Lindgren, Bengt J?nsson. Eur J Health Econ (2012) 13:445–450

2. Michele Pistollatoa, Office of Health Economics, August 2015, Research Paper 15/02. https://www.ohe.org/publications/incorporating-life-cycle-price-modelling-pharmaceutical-cost-effectiveness-evaluations