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Rare diseases and individualised approaches

Orphan drugs require a more innovative, insightful take on branding strategy
Rare diseases and individualised approaches

Fast track

  • Small patient numbers and target customers mean traditional marketing techniques are often not viable for orphan drugs
  • Innovation, creativity and authenticity are key in communications surrounding orphan conditions
  • First-hand insight and feedback are necessary to understand the experience, needs and wants of patients, carers and healthcare professionals 

A paradigm shift is occurring within the pharmaceutical industry: there are few genuinely novel products being approved, the market is increasingly more generic and, due to patent expiry, companies can no longer rely on their mainstay blockbuster drugs to sustain them. Many companies are now diversifying, seeking new therapeutic opportunities, and this, in combination with changes in marketing authorisation requirements for orphan drugs, has resulted in rare diseases becoming an increasing priority for the industry. 

A drug can apply for orphan status if it is intended to treat, prevent or diagnose a disease that is life-threatening or chronically debilitating and considered rare – with a prevalence of fewer than 5 in 10,000 in the EU. Within Europe, there are currently an estimated 5,000 to 8,000 distinct rare diseases affecting 6 to 8 per cent of the population, equivalent to between 27 to 36 million people. This represents a significant opportunity overall, but one which is fragmented in terms of individual conditions and their discrete populations. 

Orphan drugs present unique treatment and communication challenges. The small patient populations for any specific rare disease can be diverse and geographically widespread. The path to diagnosis can be long and convoluted due to a poor understanding of the condition. And the healthcare professional's experience with orphan conditions can be limited to non-existent with few leading experts. 

Patients with rare diseases can often end up on treatment pathways for more common conditions, which are not often suited to their unique needs. Within primary care, physicians may only encounter one patient with a specific rare disease in their entire career and yet are still relied upon to identify, refer, support and manage the condition appropriately. For patients this can mean years of frustration, lack of support, little to no understanding and feelings of helplessness and hopelessness. 

The small patient numbers do not command the same media attention or support within the healthcare system as more common conditions and, as a consequence, interest in rare diseases can be low. Also, depending on the type of drug and its expected benefit, patient expectations have to be carefully managed. Even if treatments are, or become, available, there are often barriers to access due to their high cost and even physical distance to travel to receive treatment can be an obstacle to overcome. 

Therefore, working with orphan drugs and rare diseases requires different approaches to developing brand strategies. Due to the small numbers of patients and target customers and their wide geographical diversity, traditional marketing techniques are often not viable. Orphan drugs may not have a large salesforce, or a salesforce at all, so representative independent communications can become critical and, thus, digital can play a massive role. 

In addition, competition for share of voice will be high due to the large number of pharmaceutical companies targeting the same customer. Internally it is essential that all key stakeholders be aligned, with all communications reinforcing clear messages and helping to promote the desired call to action. Identifying key patient touch points, precise customer targeting and education provision are vital, while at the same time balancing resources appropriately. From a marketing perspective this means communications have to be truly innovative in order to cut through, and communication channels and media plans need to be carefully thought through during development. 

To address the challenges of marketing orphan drugs, it is critical to gain first-hand insights and feedback about the condition, about existing care pathways and about current management from patients, carers (if relevant) and healthcare professionals (HCPs). It is also important to understand the patient numbers in terms of HCP workload, in order to contextualise where the condition sits in their daily priorities.  

Only by gaining true insight into the issues, current experiences, wants, needs and desires can a successful communication plan be developed for a particular orphan drug. In this way concerns and gaps within referral and treatment pathways can be identified and tools created to overcome them.

Rare disease facts

  • A rare disease affects fewer than 5 in 10,000 people in the EU
  • 27 to 36 million people in the EU have one of up to 8,000 distinct rare diseases
  • The global orphan drug market is expected to be worth $127bn by 2018
  • 65 orphan drugs were launched in the EU between 2002 and 2013

Historically a medicine intended for a small population would probably lie below the value-radar, however, in an increasingly cost-conscious environment, any patient investment could be scrutinised. This presents unique issues for rare diseases. The low populations can mean limited available outcomes data and this is often poor in quality due to the impossibilities and impracticalities of running large randomised clinical trials, which normally drives the value-story for any therapy. All this means gaining acceptance in clinical guidelines is incredibly difficult and can result in treatment access issues. 

The reality of rare diseases is that many HCPs who are key to patient management do not get the routine experience of seeing these patients. Therefore to compensate for the lack of day-to-day exposure it is important from a communications perspective to help paint this critical step in the buying cycle. Patient interviews and film can be particularly effective as can virtual therapeutic administration training. 

In these often-overlooked areas, patient organisations are highly influential and can play a large, personal role in these patients' lives. Because of this, companies need to be aware of coping strategies that patients have already established for themselves, ensuring they do not duplicate the efforts of existing patient support groups. Engagement with these groups is therefore vital to shape brand strategy and form solutions. With their knowledge and endorsement, more can be done to support patients and raise the disease profile, ultimately raising standards and driving better care. 

Innovation, creativity and authenticity are key in communications surrounding orphan conditions. The approach needs to stimulate curiosity in what is often a low interest territory, drive media interest, communicate a well rounded value story, while helping to mobilise key stakeholders to do more for patients, driving therapeutic uptake and improving care.

Article by
Whitney Andrews and Elizabeth Chambers

Andrews is senior account director and Chambers is group account director, Langland. Email Andrews or email Chambers

24th July 2014


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