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Small diseases need big picture communications

An early commitment to communications planning in rare diseases is worth the risk

Orphans 4

European directives provide welcome incentives for companies to focus R&D resources on the development of new solutions for rare diseases.

However, despite such governmental and regulatory-level advances, numerous local level post-approval hurdles remain to be navigated before orphan drugs can reach the patients they were designed to benefit. Issues of evidence needs and expectations, physician understanding and prioritisation, healthcare infrastructure and patient access represent significant barriers to orphan drug success. 

Strategic communications that are specifically tailored to the unique orphan drug environment can bridge the gap between European-level aspirations and local-level adoption. In the face of this unique set of challenges, a streamlined and joined-up communications approach is required.

A long-term investment 
For an orphan drug, communications planning that is focused solely on launch is not enough. Drug uptake will, by definition, be slow, although financial pressures mean that growing the market within an acceptable timescale is essential. 

Developing a strategic communications plan for an orphan drug requires stepping back and seeing the bigger picture; identifying knowledge gaps that need to be filled at the earliest possible opportunity, finding ways of making a limited per patient market spend reach across geographies and healthcare stakeholders, and identifying the restrictions that will be faced post-launch. However, such an approach requires early investment in communications, and this can carry financial risk. 

Small pharma or biotech may struggle with funding for communications early on in the development process, while larger pharma may be held back by slow processes that are embedded in a rigid stage-gate decision-making process. It is necessary to strike a balance between waiting for confidence to grow in a candidate, but not leaving it too late to have proper communications support in place at launch. When reviewing the options, it is important to remember that, in general, orphan drug designations are associated with a heightened chance of success; figures show that approximately 82 per cent of orphan products receive European approval, compared with a success rate of 35 per cent for traditional drugs.

Four steps to success

1. Start early 
Whereas a traditional communications strategy might start to gain momentum as phase III trials begin, the accelerated development and approval processes of orphan drugs mean that the phase III milestone is already too late. 

In one analysis, the average time from phase II to launch for orphan drugs was found to be 3.9 years, compared with 5.4 years for non-orphan drugs, thereby shortening timescales in an area where more preparatory work than usual needs to be carried out. As a result, companies often find themselves months behind in their planning when a product has gained regulatory approval and the exclusivity clock is ticking.

Under-developed and poorly-researched disease areas need more time to lay the groundwork for success. This includes time spent on understanding epidemiology, completing disease characterisation, and measuring the economic impact and burden of disease. As rare diseases are often a low research priority, literature searches may not answer all questions, and time should be built in to commission original research. 

Taking time at this early stage to understand the data gaps that may be left by the clinical programme is also essential, particularly with regard to real-world data requirements around market access and reimbursement. Registries are an important tool in developing an understanding of the natural history of a rare condition, and 90 per cent of experts testify to their value in rare disease research. As well as fulfilling post-approval requirements for an orphan drug, multinational registries will form an essential source of additional data to help further disease understanding.

2. Adopt an end-to-end vision 
Orphan drug uptake can be thought of as an endurance event, with launch being only the first of many hurdles to be faced. It is essential to think beyond launch and take a collaborative approach to identifying all elements needed to support physician and payer uptake. These should become an integral part of the product communications plan right from the start. 

One of the key challenges is to optimise the effectiveness of what is likely to be a small field force with large territories to cover. The difficulties are compounded by a high need for disease education and awareness owing to low levels of clinical and product experience. Most physicians receive little, if any, training in rare conditions; in the UK, rare diseases are still not included as a part of the general medical curriculum.

To address these needs, classical field force teams will need to adopt multiple roles over time; for example, key account managers can offer advice and support for physicians and payers who are involved in obtaining funding for individual patients. Medical science liaisons (MSLs) can play a strong educational function, arranging scientific discussions to encourage understanding, as well as implementing effective tailored analyses of where the new drug may be of value. Technology should also play a role, and the wise use of multichannel communications, such as e-detailing, can make a real difference over a widely-spread customer base.

3. Enable real-time data connectivity 
Faced with the difficulties in running clinical trials in rare diseases, it is necessary to take an iterative view of data generation, building the evidence base as a series of ripples rather than as one large splash. 

Communications will evolve over time, as necessary. Although the product scientific story may be aspirational at the start, especially where long-term and patient-reported outcomes are needed to complete the clinical picture, a gradual approach has its advantages. In particular, it enables an assessment of how messages are being received, as well as a re-evaluation of understanding and communications priorities as clinical experience with the new product increases. 

A sophisticated data tracking process that allows continual landscape analysis and rapid assimilation of new longitudinal data into the overall scientific and value story is key. Ultimately, in a situation where data is limited, each new dataset should have optimum impact. By connecting data to planning in real time, it is possible to achieve a new immediacy in communications outreach.

4. Involve all stakeholders
Involving key stakeholders in communication planning as early as possible provides a clear indication of on-the-ground educational, management support and patient access needs. Identification of thought leaders in a small specialist field may seem to be easy at first glance, but concentrating on publication history alone risks missing many valuable contributors. Examination of rare disease networks, patient associations and Centres of Excellence can be good pointers to which clinicans work in the field.

An exploration of the patient experience should also be an essential part of any orphan drug communications strategy. For rare diseases, more than most, patients and patient associations are the acknowledged true experts in their condition, and can provide hugely valuable insights into the reality of care. Across Europe, over 30 million people are affected by rare conditions, comprising approximately 6 - 8 per cent of the whole European Union population and creating a strong voice waiting to be heard.

Rare disease management is often based around a Centres of Excellence approach. The Council of the European Union places a strong emphasis on Centres of Excellence; however, in practice, there are either not enough of these centres, or patients are unable to navigate their way to the required specialist centre, relying instead on local care. A recent UK survey showed that just 27 per cent of people with a diagnosed rare condition currently receive their care through a Centre of Excellence. 

In consequence, a focus on specialists alone will miss a significant proportion of the target patient population. This situation necessitates a two-pronged approach to stakeholder engagement, combining intensive education for specialist physicians within Centres of Excellence with a widespread awareness programme, which is available locally, to enable patients to access specialist management pathways.

Rare diseases raise a number of additional challenges

Patient pool small, diverse and widely spaced; this means that data is:

  • Slow to gather
  • Limited in amount

Reduced data quality:

  • Double-blind, placebo-controlled trials often not possible
  • Lack of statistical power
  • Heterogeneous populations
  • Difficult to match controls
  • Harder to gain acceptance in clinical guidelines

Compressed lead-in times

  • Accelerated development and approval timelines
  • Faster to market means less time to prepare
  • Reduced clinical experience, the majority of which occurs post-approval
  • Reduced time for publishing activities meaning fewer citable sources to support launch 

Combating attitudes and perceptions

  • Low awareness – many doctors may never have seen a case
  • Poor understanding – disease not fully characterised
  • Little evidence of disease impact – limited research published
  • Low medical priority – deal with the many, not the few 

Under-resourced infrastructure

  • Lack of established diagnostic and referral routes
  • Expertise clustered, not uniformly spread
  • Lack of resources, especially around diagnosis and monitoring
  • Potential for low skill base in some slow-moving fields
  • May be lack of specialist professional societies/guideline bodies

High barriers to access

  • Generally high price to low-cost comparators
  • Underdeveloped patient and reimbursement pathways
  • Difficulties in demonstrating health technology assessment (HTA) value
  • Lack of validated patient-reported outcomes measures 

Lack of patient support

  • Slow diagnosis
  • Poor referral pathways
  • Lack of local expertise
  • May need to travel long distances for care
  • Difficulties in obtaining funding 

A new communications approach 
Communication strategies for rare diseases should be tailored around the unique challenges of this space, which often means taking a more holistic and proactive approach early on in the product development cycle. Second chances that make a positive impact will be rare, so each communications opportunity should be anticipated and carefully prepared with a truly long-term vision in mind.

This article was originally published in the PME supplement Orphan Drugs and Rare Diseases: Strategic communications for orphan drugs

Article by
Ruth Slater and Charlotte Carey

Slater is senior director, medical communications, Carey is account director, medical communications, at Quintiles. For further details contact: Charlotte

27th January 2014

Article by
Ruth Slater and Charlotte Carey

Slater is senior director, medical communications, Carey is account director, medical communications, at Quintiles. For further details contact: Charlotte

27th January 2014

From: Marketing



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