Marketing in rare diseases – getting closer to your customers
pharmafile | January 20, 2010 | Feature | Sales and Marketing | Orphan, marketing, marketing ideas, niche
The 21st century healthcare industry is reorientating its strategic focus on the increasingly successful areas of orphan and rare diseases.
There are several reasons behind this shift away from the primary care blockbuster that treats millions (in hypertension or asthma, for example), toward the more targeted, usually secondary-care based medicines.
The first is the development in pharmaceutical science, with drugs now getting much closer to targetting specific mechanisms in diseases. This in turn means a much smaller group of patients can benefit. Secondly, legislation in the US and Europe to encourage the development of drugs to treat orphan diseases is now well established, and providing real commercial advantages for companies who can meet unmet need in these niche areas. Rare and orphan disease promise the potential of less intense competition, faster and less expensive development, and lower marketing spend on a small, highly motivated audience. Last but by no means least, they can also offer a chance to demand premium prices, which combine to make a very attractive and less risky investment.
Although many of us are likely to find ourselves in unfamiliar territory when marketing within this specialist area, more and more pharma companies are looking to orphan and rare diseases to replace rapidly disappearing revenues from key brands as their patents expire.
Therefore, to ensure a successful future and accelerate our career progression, we marketers will need to be aware of the difference in approach, and in the skills needed, to successfully market drugs within this ever more popular area.
What makes an orphan drug?
The EMEA define orphan disease treatments as those for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU, or those which, for economic reasons, would be unlikely to be developed without incentives.
Should a therapy fall within this criteria, companies are supported by EU legislative incentives including 10 years market exclusivity after marketing authorisation and greater support, guidance and reduced fees during the market authorisation process.
But what about the increasing number of diseases which do not fall under this defined threshold? What if the prevalence of your disease was between 6 to 20 in 10,000 (0.06 – 0.2%), such as oropharangeal cancer, or angle closure glaucoma. Also included in this bracket of non designated rare diseases would be cystic fibrosis, multiple sclerosis (MS), renal cell carcinoma, and acute myeloid leukaemia, diseases we have all heard of.
In fact, the EMEA have estimated that there are between 5,000-8,000 different rare diseases registered in the EU (both within the orphan threshold, and just outside the prevalence criteria). This represents a total population of between 27-36 million patients, which continues to grow with up to five new diseases being described every week.
Regardless of the threshold set by the EMEA, a marketer will face similar challenges when directing a brand for non-designated rare diseases as they would for an orphan designated therapy and given the focus of this article is on the alternative skills required to market in this specialist environment, let us consider both orphan and non designated rare diseases.
What is it about these diseases which make the commercial and marketing challenges different to working on more mainstream brands, like that treat diabetes or cardiovascular conditions?
1. There is often a high unmet medical need and diseases usually have a significantly traumatic impact on all those affected
2. Disease management tends to be inconsistent and fragmented
3. Healthcare professionals (HCPs) are highly specialist and usually very passionate about their specific area of interest
4. Patients and patient associations are very involved, with close relationships to key opinion leaders (KOLs)
5. Treatment options are usually limited and can be complex, very high cost or used off-label
Big pharma interest in orphan & rare diseases
In a recent interview, GSK’s chief of R&D Moncef Slaoui clearly stated that the company would soon be unveiling plans to develop more drugs for rare diseases, in particular outlining the benefit of less pricing pressure when compared to drugs that treat common illnesses with multiple treatment options.
In doing so he would be building on a series of new alliances the company has formed that focus on specialist disease areas:
• October 2009: GSK and Prosensa form alliance to fight Duchenne Muscular Dystrophy a severely debilitating childhood neuromuscular disease affecting 1 in 3,500 newborn boys
• Also in October, GSK received FDA approval for Votrient (pazopanib) for patients with advanced renal cell carcinoma designated as Orphan by the EMEA
• August 2009: GSK and Genmab announce results for Arzerra (ofatumumab) in rituximab refractory follicular non-Hodgkin’s lymphoma. Follicular Lymphoma is orphan designated by the EMEA
And it is not just GSK focusing on orphan and rare diseases. Looking at the development pipeline for MS which, with a prevalence in the EU of 6-15 per 10,000 is not a designated orphan disease, we see that there are currently 19 therapies in phase III trials, with a further 43 in phase II – and not from small dedicated biotechs, but from big pharma companies like Pfizer, Sanofi-Aventis, Novartis, AstraZeneca and Roche.
The rationale behind the new focus
IMS forecast global pharmaceutical sales increasing by 4-6% in 2010, however, specialist and biologic drugs are expected to grow at up to 10-12.5%.
In addition, although you might not expect drugs designated as orphan or indicated for rare diseases to create high revenues given the small populations, there are more than 50 drugs in this area with annual revenues exceeding $200 million and of these 19 have attained the $1 billion blockbuster mark.
Admittedly, this is usually because they treat several rare diseases, for example Glivec, Novartis’ second largest brand, earned $3.67 billion in 2008 and grew a healthy 20%. Originally launched for the treatment of orphan designated chronic myeloid leukaemia, Glivec has since gained several additional orphan indications propelling it to blockbuster status and highlighting the key to success for these brands – indication extension, or in industry parlance “The Progressive Blockbuster”.
What this means for the marketer
The benefits for you as the marketer working in this area numerous. They include the fact that you are likely to be more customer-facing than you have been used to, not just with HCPs, but also senior budget holders and patient associations.
Secondly, a smaller team means you are likely to have a closer relationship with other internal teams and have more involvement in their direction and activities, in particular the medical and market access teams.
Lastly, in a growing naive market, there is likely to be a rapid increase in the amount of clinical data. This will be a fast moving, with messages and positioning for the brand developed on recent abstracts and presentations.
The challenges
1. Orphan and rare disease markets are small: The revenue opportunity for a single indication may be limited: internal cash expectations may require a longer term strategy, given the small opportunity within each indication. You need to ensure that your clinical, medical and commercial strategies are clearly aligned to maximise future potential.
A small customer audience means there is little room for second place, so developing strong KOL advocates early becomes even more critical. With a small number of highly dedicated KOLs, how much room is there in the customer’s world for your brand if it comes second?
To realise your ambitions you need to win this small audience early, and create strong brand advocates.
Disease management is likely to be poorly organised and disjointed requiring specific skills and support. The clinical management network, although motivated, is likely to be disorganised. So success isn’t just about providing a drug – it also requires organisational expertise and skills to jointly develop service provision and commissioning arrangements with NHS counterparts.
2. Clinical and market information is limited: Without a clear view of the market place, commercial decision making can be more of an art form. Without the usual market, customer and competitive information you might be used to, it can be difficult to always find robust data to support commercial decisions? You will need to consider innovative ways of gaining market insight and competitive advantage.
It can be hard to define the competition and as a result to know where your real battle is. Despite having clear clinical differences and agreed advantages, there is often a lack of consensus behind expert clinicians’ clinical approach. So you will need to gain deep customer insight, getting under the skin of what really motivates and drives decisions.
Limited and fragmented clinical data can lead to a lack of consensus over the best end points to evaluate different therapies: According to the EMEA “fewer than 1,000 diseases – essentially those that occur most frequently – benefit from a minimum of scientific knowledge”. With limited clinical clarity due to a lack of robust data, objective comparisons become difficult and result in KOLs choosing therapies on a more subjective basis. Success will require alignment of both communication and organisational skills to address the subjective needs of your customers.
3. Success requires free availability and provision of your drug, often more challenging than expected: High unit costs, reimbursement and access can all be a significant challenge. Orphan and rare diseases are invariably an emotive area, with a strong patient voice and genuine unmet need. The historical guarantee of ‘special status’ with unlimited access is rapidly changing, with a greater focus on budgetary impact and cost effectiveness. High unit costs and maverick specialists, mean that budget holders are keen to manage use and availability early on through strict guidance, clearly defined service provision and robust commissioning. Companies need to have the right expertise in place at the right time to ensure involvement in the development of this managed entry process.
4. Different organisational skills needed: The combination of a small specialised audience, an underdeveloped clinical system, and the likelihood of access issues means the ideal balance of organisational capabilities are different for orphan and rare diseases. A smaller team needs the skills to win KOLs quickly, expertise to ensure easy access and develop specialist services and the internal coordination to gain advantage through customer and strategy aligned medical programmes.
Summary
So orphan and rare diseases offer a different challenge when compared to marketing in the more familiar areas of primary care, cardiovascular, diabetes or respiratory. This means that as a marketer you need to consider a different balance of tools and skills to reach the brand goals.
Success will come from winning the hearts and minds of a smaller target audience, either by getting there first and defending strongly, or by aggressively replacing the current preference.
There is very little room for second place you need to get things right first time, so you are unlikely to get a second chance to displace those currently in favour. Two key considerations must therefore be:
• How confident are you that you have the right tools and approach to win those hearts and minds over the competition?
• How confident are you that you have the optimal organisational structure in place?
In part 2 of this feature, to be published in the New Year, we will look to identify skills and tools that an orphan and rare disease marketer can use to be most effective in their role – to displace the competition and create fast and early uptake for their brands. And we will consider the different organisational issues which inform the organisational capability required.
Michael Craig is a consultant at the MSI Consultancy and can be contacted at mcraig@msi.co.uk or via www.msi.co.uk.
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