Paul Mannu | Article

Demystifying ‘value’ in rare diseases

The case for early patient and payer engagement

rare diseases

Pharma must work with patients, payers and HCPs from the outset of the drug development process to ensure deep understanding of each stakeholder’s needs and, in turn, successfully demonstrate the true value a new treatment can bring. Early engagement with all stakeholders produces the greatest likelihood of attaining market access through understanding what ‘value’ means to each. Payers must be taken along on the journey to ensure cost models are relevant. In-depth research with patients themselves can show actual needs and hence where value can be added for real-world improvements in quality of life.Success for each of these stakeholder groups relies on demonstrating the same thing: value. But value often means different things to different interest groups and, when it comes to rare/ultra-rare diseases,

Early engagement with all stakeholders produces the greatest likelihood of attaining market access through understanding what ‘value’ means to each. Payers must be taken along on the journey to ensure cost models are relevant. In-depth research with patients themselves can show actual needs and hence where value can be added for real-world improvements in quality of life.

Success for each of these stakeholder groups relies on demonstrating the same thing: value. But value often means different things to different interest groups and, when it comes to rare/ultra-rare diseases, value can be extremely challenging to define, measure and communicate in order to achieve positive reimbursement decisions.

Shared vocabulary of value
Taking the path of proactive, early engagement can put pharma in a unique place to drive and deliver a positive understanding of value. Early involvement with the patient and his/her world provides insight into the true burden of the disease. Similarly, early consultation with payers facilitates a true understanding of their budgetary and cost considerations.

This process enables the development of a shared vocabulary of value that each stakeholder understands and can use to communicate effectively with others in the value chain.

Understanding the world of rare diseases
Demonstrating the genuine value of anything is best achieved from a standpoint of deep understanding, yet the world of rare/ultra-rare diseases is often misunderstood. The reality is that, while the numbers with any particular rare disease vary globally between 1 and 8 per 10,000 population, collectively an estimated 400 million people worldwide are currently thought to have one.

This equates to 3.5 million people in the UK (1 in 17) alone and helps explain pharma’s growing commercial interest in rare/ultra-rare diseases and the potential tsunami of disease treatments expected to emerge over the next few years.

Within the UK however, the above exists in a localised healthcare context where the NHS faces a major funding gap with a need to find an extra £22 billion in savings over the next five years. Delivering on its commitment to deliver the most effective, fair and sustainable use of its finite resources will ultimately mean payers having to increasingly say ‘No’ to some areas of patient care. With the present government’s stated position over this five-year period being to focus more on primary care services, the seemingly inevitable consequence is that market access for hospital-based specialised drugs will become harder.

Understanding the world of the payer
A helpful starting point is to remember that payers are, first and foremost, people – people following processes directed by local-, regional- and national-level policy, within an external regulatory framework. Policy guidelines can often be fluid and, in the case of the UK, even the very structure of the payer framework itself is in a process of transition. As individuals, payers also have varying assumptions about rare/ultra-rare diseases as a whole and may not have even heard of a specific condition or treatment. Furthermore, given the low patient population with any rare/ultra-rare disease, the conventional methods of clinical evaluation in determining patient outcome, budget impact and therefore value, simply don’t work.

See value in clinical and economic terms
Understanding the world of the payer means recognising that value is seen in both clinical and economic terms. To determine this, payers typically need information to help answer fundamental questions including ‘which patients need this and why?’, ‘how much is this product and why is it worth the cost?’, ‘can I afford it?’, and ‘what are the consequences of NOT funding it?’ These, and many others, will be asked of many different products, at different stages in the product/disease lifecycle, and at different stages in the funding cycle. They will also be asked where patients, physicians and payers may be facing healthcare challenges relating to a specific geographical area and/or time of year. This shows the highly changeable set of interacting parameters that payers must negotiate in order to determine the value of a product. Early payer engagement is therefore essential to understand the context and unique challenges facing them; equally importantly, it helps show each payer that you understand his/her own personal drivers. Consequently, they will, individually and collectively, be more likely to consider the potential value that your product could bring to their patients.

Ensure you understand the payer’s perspective
“You’re not taking the payers on the journey at the moment. You launch a molecule; you thrash around with KOLs, professors of disease. You have lots of launch meetings that have nothing to do with the payer strategy and at the end somebody, somewhere, who’s probably not qualified or appropriate, will come and see me with a cost model that bears very little relevance to either my national or strategic health economy. And so, really, the payers have not been taken on the journey at all.” – NHS Trust, payer

In rare/ultra-rare diseases, expert knowledge is often small and fragmented and the preserve of a few thought leaders. Moreover, existing information may be geographically spread, in a number of languages, and contain markedly different medical, social and cultural assumptions and judgements. Greater emphasis needs to be placed on the role of the individual patient, not just as a potential expert in the condition, but also as an influencer in the development of any treatment. In addition, the disease burden stretches beyond the patient to friends and community, all of whom can be impacted positively by effective treatment, expanding the real value of a drug.

The very nature of rare/ultra-rare diseases means that early patient engagement can have profound impacts on cost savings, as well as improved patient outcomes in terms of Quality of Life (QoL). Consider the following:

  • The challenge in obtaining a correct diagnosis – this can evolve and often takes years, if not decades
  • Initial misdiagnosis occurs in approximately 40% of cases, which can lead to detrimental health consequences
  • If incorrect diagnosis is overly prolonged, the effective treatment window for a given treatment may be missed
  • So the impact of the disease burden beyond the patient is obvious. If left untreated, patients will invariably need ongoing support. Additionally, the search for a diagnosis, let alone a treatment, can involve patients, their families and carers, having to frequently travel long distances or even physically relocate. Household income may be significantly decreased when patients, parents or close relatives are forced to reduce, or give up, work in order to meet increased patient care demands.

For example, according to a recent, high-profile international survey on pulmonary arterial hypertension (PAH), over 85% of patients said their employment/work was affected, with nearly three quarters of patients saying their household income was affected, leaving most families below the average level of income. If this in turn impacts pension contributions, the financial consequences can last decades.

The reality is that the more you engage with the patient, and the earlier you do, the more clearly you understand the potential burden in terms of cost, QoL and patient outcomes – as well as the potential cost savings and improvement in QoL if a correct treatment can be provided in a timely manner.

A model for early engagement
To this end, we have developed a four-step, patient-centric process that collaboratively and pro-actively engages with payers, patients and HCPs in order to demonstrate a meaningful, shared sense of product value.

The purpose of Phase 1, ‘Align’, is to understand how the impact of a disease burden is currently measured and identify any gaps in understanding.

Phase 2, ‘Deep Dive’, looks to identify and understand the key factors of the disease and their financial impact. Both of these are achieved by a tailored, multi-method approach involving ‘in-the-moment’, real-time feedback via our bespoke mobile app, as well as ethnographic or semi-ethnographic elements, plus utilisation of an online community of doctors, patients, payers and KOLs who provide longitudinal feedback to capture the ongoing impact of the disease.

Phase 3 uses Co-Creation workshops to explore and understand meaningful outcomes from the perspective of the payer and the physician on the disease impact and co-create potential endpoints.

The process ends with Phase 4, which quantifies the patient endpoints identified during the collaborative process.

While the patient remains at the centre of the approach, ideally each stage is led by a steering committee to provide guidance, credibility and gravitas to any outcomes, as shown in the PAH study already referenced. The result of the process is that everyone is on a shared journey to understand ‘value’ in the world of rare/ultra-rare diseases.

Further examples
Case Study 1: Duchenne Muscular Dystrophy (DMD)

The aim here was to better understand the significance of the disease burden from the perspective of all stakeholders, especially the patients (DMD affects young boys). What emerged was that parents and patients did not find the ‘wheelchair moment’ – when the disease has progressed to the point where the boys can no longer walk – to be as negative as expected. Instead the child felt he gained freedom and independence.

In fact, loss of arm and hand strength was much more adversely impactful on QoL (for caregivers as well as patients), yet this had not been taken into account previously by other stakeholders, including payers. This is a view shared by a recent clinical study that considered the importance of measures to look at improvements in upper ambulatory function.

Case Study 2: Cachexia

The key aim was to identify which of the available QoL measurement tools most effectively captured the key metrics of cachexia, a muscle wasting disease – specifically the full extent of any suffering. The study involved patients in creative card-sorting exercises where existing QoL metrics were used. Engaging directly with patients showed that they themselves could help create measurement tools that more accurately reflected their own disease burden. In this case, it also demonstrated how little the existing tools took into account the financial strain on the patient and caregivers.

Ongoing benefits of early engagement

Early engagement with patients and payers to define value clearly helps facilitate the initial market access of products for treating rare/ultra-rare diseases. However, it has the capacity to go much further than this, in demonstrating ongoing value, which will become increasingly important as payers look to have ever greater assurances over the longer term. Ongoing value could take the form of fewer hospital interventions or prevention of future generations developing a given condition. It may be that more follow the example of shared risk pioneered by Sweden, which involves ongoing real-world evidence of the benefit of treatment. What is certain, however, is that understanding how to demonstrate value to payers in terms which resonate with them will be the key to unlocking access to rare/ultra-rare disease treatments for future generations of patients. For further information, please visit: Optimizing market research in rare diseases.