Navigating the Maze of Change in Rare Diseases

Haemophilia-Adelphi-image-1-rgb-coverOur rare diseases’ experts often tackle research in hard to reach populations and complex therapy areas – and Haemophilia definitely fits these boxes.

With several advances on the horizon, brand teams are increasingly in need of impactful customer insight to support their pre-launch planning. Our experience has taught us that market research in Haemophilia, and other rare diseases, must take into account the following:

1: Addressing Multiple Viewpoints
Haemophilia is a lifelong condition whereby patients and their families need to work together with healthcare professionals to manage it effectively over time. In addition, Haemophilia, like most other complex conditions, is managed by a multi-disciplinary team. Many research questions require a multi-faceted approach to cover all viewpoints, as all stakeholders have an impact on treatment choices and treatment success.

2: Uncovering the Patient Experience
The level of burden and risk faced by people with Haemophilia can be hard to comprehend, and many remain uncontrolled. Despite this, on the surface many patients lead a seemingly ‘normal’ life and may not realise that their disease is inadequately controlled. It is important that patient research gets to the bottom of the real day-to-day experiences, challenges and disease understanding.

3: Accessing the Right Respondents
Haemophilia is an extremely rare condition with between 1 in 10,000 to 1 in 50,000 males affected depending on the type. From the physician’s point of view – experts are hard to come by. With treatment led by a relatively small number of specialist centres such as Haemophilia Comprehensive Care Centres (HCCCs) and Haemophilia treatment centres (HTCs)) in the UK, recruitment has to be tailored and engaging to be effective, and even then small samples are to be expected.                                                                                                                         

4: Guiding Customers Through Change
Until recently, Haemophilia treatment has been relatively stagnant. Treatment has typically focused on clotting factor replacement to prevent bleeds – options vary by Haemophilia type and the derivation of clotting factor (recombinant vs. plasma derived), with little differentiation in terms of product benefits.

However, the market is about to undergo a series of changes with new brands on the horizon, and longer-term advances from the likes of gene therapy peaking around the corner. Some of these therapies could change the landscape entirely with their aims to reduce the overall severity of patients’ Haemophilia. It is likely that physicians will need guidance on how to negotiate these changes and incorporate new treatments into their practice. Experiences of analogous situations in other therapy areas that have undergone a series of changes, such as MS, have served us well in recommending the best approach.

For now, it remains to be seen how the new Haemophilia market will pan out. Questions that will need to be answered include:

  • Will treatment frequency and/or perceived convenience become a driver of choice?
  • Will cost become even more of a driver (due to tender process and/or the desire to reduce cost of long term bypassing treatment?)
  • Will the patient’s voice grow?

Adelphi’s rare diseases’ team have experience of negotiating these types of challenges/considerations in Haemophilia, and in many other complex conditions. Get in touch if you’d like to know more about what this means for your brand.