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Rare diseases, a sustainable opportunity?

Once a neglected area by the pharmaceutical industry, rare diseases are now very much on the radar with drugs in the category worth $50 billion worldwide.


29 April 2016

By: Mike Young

Once a neglected area by the pharmaceutical industry rare diseases are now very much on the radar with drugs in the category worth $50 billion worldwide. A third of these achieve annual sales of $1billion with the market rising at 20% per annum over recent years. Unsurprisingly, the interest and growth has been driven by the decline of the traditional blockbuster as well as more positive factors including premium pricing, reduced marketing costs, increased chance of reimbursement for chronic unmet need and longer exclusivity. So what are rare diseases and do they represent a sustainable answer to the industry’s problems?

According to the European Commission on Public Health rare diseases are life threatening or chronically debilitating diseases of such low prevalence that special combined efforts are needed to address them. Low prevalence in this context is defined as less than 1 in 2000 people in the population. The European Organisation of Rare Diseases estimates that there are between 5 and 7,000 distinct rare diseases affecting 6-8% of people in the EU with 80% of these thought to be of genetic origin.

Whilst one of the attractions to this space for the industry is the opportunity to charge premium prices, it also creates controversy. Critics ask whether such prices can be justified. The companies respond by pointing out that the drugs are true innovations which address unmet needs and are expensive to develop. The prices look high because they are spread over so few patients. However, from the payers’ perspective things may look different. When trying to contain or reduce health care costs affording these medicines can be a major issue. It follows that unless rare disease drugs can be shown to be cost effective gaining the support of payers in the future is likely to be a key marketing challenge.

It is not the only marketing challenge. Others include, early identification and treatment of patients, working with the rare disease expert community, building the right educational and product brands and ensuring internal alignment and support. In many ways, marketing of rare disease drugs is different to conventional drug marketing. This difference isn’t simply about finding and reaching so few patients. It concerns the whole personality of the company/product brands and the style and tone of the communication that flows from them. The rare disease community is made up of patient advocate groups, their expert clinicians and academic researchers. The patient groups are highly active on social media and are important to achieving increased awareness, successful clinical trial recruitment and positive brand perceptions. Marketers need to be highly attuned to their sensitivities and treat them with the appropriate respect.

In conclusion, sustainable success in meeting the unmet needs of rare diseases requires more than great science and innovation. It also implies a high level of appreciation of the needs of payers, clinicians and patients in an emotive and informed environment. The stories that marketers tell will therefore need to focus on brands that embody empathy, integrity and genuine value. Nothing less will do.

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