Back in the early 90s I remember pitching for a new product launch. It was for a top ten pharma company, and the budget that was allocated for this launch was very significant. It was a nice piece of business to win.
But that pitch would never happen now. Why? Because it was to launch the fifth – or sixth – proton pump inhibitor.
It’s hard to imagine that situation in the pharmaceutical industry of 2012, where the commercial future for branded drugs in ‘everyday’ diseases is pretty limited. Another striking feature of those times was that there seemed to be a set formula for launch: pay lip-service to generating some minor KOL endorsement (after all, which KOL would be remotely interested in ‘me-too’ number 6?); develop an impactful campaign; then send the salesforce out to do their bit. Easy.
Contrast that with the task facing the modern marketer faced with the job of launching an orphan-designated drug in a condition affecting fewer than a thousand patients in the country (‘ultra-orphan’). Clearly, the job would be unrecognisable to the product manager of two decades ago. Demonstrating value in a credible way is at the heart of any successful ‘campaign’ for the marketing of these agents, so it’s worthwhile to remind ourselves how we have reached the stage we are at, before examining the ways forward for marketers.
NICE – the knock-on effects for orphan drugs
The emergence of NICE profoundly affected the marketing of pharmaceuticals in the UK and its influence has spread far beyond, becoming a point of reference for many healthcare systems. In particular, the pressure on manufacturers to demonstrate a QALY below £30k is familiar to everyone and how the pursuit of this objective has distorted outcomes research can only be guessed at. But when it comes to orphan drugs, meeting this criterion is impossible.
This forced NICE into the adoption of an ‘orpan get-out’ clause whereby when considering the funding of these drugs, the priniciple of equity held sway. In other words, patients should not be discriminated against because of the rarity of their disease.
There are, however, an increasing number of ‘ultra-orphan’ drugs (where the condition has a prevalence of under 1:50,000) for which NICE acknowledged there existed ‘special difficulties’. This lead to the formation of AGNSS (Advisory Group for National Specialised Services), a move welcomed universally as a sensible forum for the resolution of funding issues for ultra-orphan drugs. Yet only this month AGNSS has been given notice of its termination with the role moving to NICE. The uncertainty created by this move must be a concern to the pharmaceutical companies involved in this area.
Orphan drugs – no longer rare
It must be acknowledged that the age of the orphan designated drug has been with us for some time – over 20 years. So perhaps we should now be considering them as almost mainstream pharmaceuticals? They have given new emphasis to marketing disciplines involving the development of sophisticated health economics and its application to Health Technology Appraisals; this is now as much a part of life as product registration, manufacture and distribution.
But what does it mean for the day-to-day working life of the orphan drug Product Manager?
Defined audiences, closer relationships
First and foremost, anyone in charge of an orphan drug must be adept at building bridges on a one to one level and between different, sometimes competing factions. By definition, clinicians specialising in an orphan disease are few and far between and any one individual can be the gatekeeper to a sizeable percentage of your potential patient population. So it goes without saying that it is a relationship that must be nurtured.
And the same is true of patient support groups: they tend to play a much more central role in clinical trial development and market access than in other therapy areas.
Promotional campaigns or pure ‘education’?
Every marketer in any sector enjoys the excitement of creative campaign development, but it has to be recognized that the logic of doing so for an orphan drug does not really stack up. After all, if only five or so centres in the country can prescribe your drug, is there much point in promoting it to the rest of the medical community? That inevitably leads marketers down the road of high-level education and this makes absolute sense because in orphan diseases there is a genuine knowledge gap. In my own family case, I remember having to almost teach every clinician we encountered about Fabry disease before we made it to the very top of the medical tree.
However, focusing on medical education does not mean that creativity goes out the window. On the contrary, a good creative agency can really prove their worth in this environment by lifting dull but worthy communication to captivating and inspiring support programmes.
When there are so few patients actually on treatment, it seems crazy not to collect as much information about their experiences of being on treatment as possible. This is obviously in the interests of the pharmaceutical company, but it could also be argued that there is an ethical imperative to do so. With so few patients, data is at a premium, yet it is of huge potential value to future generations, allowing us to identify sufferers more easily, clarify meaningful treatment goals and in time maximise the efficiency of the resources spent on a particular patient group.
Anyone wanting evidence for the value of patient registries need look no further than the KIGS/KIMS programme in growth hormone therapy. Launched in the early 90s by the then Kabi Vitrum, this plan raised eyebrows internally because of the long commitment to such heavy investment, yet it has proved to be of immense clinical value and commercially, an invisible bulwark against the competition for first Pharmacia and subsequently Pfizer.
However, in the modern environment for orphan drugs, it must be said that any such registries should ideally sit outside the commercial sphere, yet that doesn’t meant that individual companies couldn’t benefit from the kudos of being the prime mover in facilitating such a movement.
Disease management – going the extra mile
At Dice Medical we call this ‘Product Plus’. It’s about saying to your customers “We are not simply selling you pills in a box, but partnering with you to generate better health outcomes for a needy group of patients”. One highly effective way of demonstrating this is to launch an orphan drug supported by a targeted adherence intervention programme aimed at ensuring patients get the most out of what is an inevitably high value therapy, whilst minimizing wastage.
Our sister company, Spoonful of Sugar, is at the vanguard of this movement and it is taking a familiar concept to a new level in agency terms. It demonstrates that well designed programmes can prove to be powerful glue that binds together healthcare professionals, patients and carers in a virtuous circle with the common goal of getting the most out of medication.
Where will the orphan drug sector be in the next 20 years? We have made some predictions here at Dice Medical in order to equip ourselves to become valued partners in what will become a far more collaborative approach to developing ‘disease solutions’.
Some clear needs that we see emerging and are likely to become prominent include:
- Enhanced ongoing patient support beyond the clinic
- Evolution of personalised / genetically-informed diagnosis into the orphan mainstream
- Changes in disease definition based on disease pathways
The strategic objective to demonstrate value will not change, but the methods and hence the skills required may become unrecognisable.