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.

Patient registries

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.

The future

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.

The following article was written in 2013, but the contents and thrust remain relevant today, and we hope that anyone involved in the marketing of a brand for a rare disease will benefit from reading it. 

Anyone involved in the marketing of orphan drugs for rare diseases must, at moments of weakness, surely have wished for a magic wand. One quick wave, say the magic word and ‘WHOOSH’, all of a sudden patients who have been slipping through the net are finally recognised as having a rare condition. A clinician, somewhere in the patient journey, finally joins the dots and realizes that this unusual pattern of symptoms is typical of ‘Mystery’ Syndrome and, at long last, the patient is prescribed your orphan-designated drug.

Sadly, outside of Harry Potter’s world, there are no such easy fixes, and marketers have to fall back on more mundane ways of unearthing patients who would benefit from their product. At this point it probably helps to relate my own personal story regarding Fabry Disease, as it will highlight some of the commonest ways in which patients are missed.

A personal tale

Back in the mid-90s, family life became more difficult because a mysterious malaise started to affect my wife. The symptoms were a peculiar mixture of constant exhaustion, GI problems and ever-present, low-level pain in the extremities. Even though we knew that her father had died young of a mysterious illness called Fabry Disease, we also ‘knew’ that it only affected males. This was confirmed in our minds because her much older sister, who must have inherited the same defective (but recessive) gene on one of her X chromosomes, had never suffered any ill-effects.

Support from the GP was reasonable, but unsurprisingly he was as puzzled as we were. There then followed referrals to a neurologist (fruitless) and a gastro-enterologist (equally fruitless, but physically invasive and unpleasant). Then we had a stroke of luck: the agency I was running was approached by Genzyme to help them launch a product for Gaucher Disease and in my background research, there were many mentions of Fabry and in particular, symptom manifestation in female carriers. Immediately the penny dropped and when we listed out my wife’s symptoms, it turned out to be an almost perfect checklist for Fabry Disease.

Again, through my industry connections I learnt that one of the few ‘go to’ specialist clinicians was Tim Cox at Cambridge, and we persuaded our GP to refer us. My clearest memory from that consultation was his surprise that until this moment, nobody had bothered to take a full case history. He maintained that if they had, it would have been clear what the cause was very early on in the process.

The final part of the story worth mentioning is that, although we subsequently became loosely involved with the Fabry patient support group, it was very unlikely to have played any role in our lives before diagnosis.

Lessons learnt

My personal experience must have been repeated many times over the years across different rare diseases and there are clear lessons to be learnt from it.

Firstly, even well-informed patients and their families can, for whatever reason, be ‘in denial’ about their family history, or at least be reluctant to push the agenda with their doctor.

Secondly, GPs have too broad a focus and Hospital Specialists too narrow a focus to be alert to rare disease symptom spectra. It’s as if the medical profession as a whole is equipped with a diagnostic microscope but with some lenses missing.

Thirdly, the value of taking thorough case histories has somehow been diminished in the modern era. We have the most wonderful diagnostic tools at our disposal, but they only highlight detail and often blind clinicians to the bigger picture.

Finally, patient support groups are not the gatekeepers to patient identification. They have an important role to play, but only when confirmatory diagnosis ‘pings’ an individual patient on to their radar screen.

Options open to the orphan drug marketer

If money were no object, then perhaps the first three points from the lessons learnt above could be tackled, but that will never be an option. There has to be a sensible decision taken regarding ROI, and to help us with this it’s worth considering the broad categories of patient in a rare disease population:

  • Diagnosed patients
  • Affected individuals who are under medical care but not correctly/definitively diagnosed
  • Affected individuals with symptoms, possible presented to their GP and a hospital specialist but not yet seriously engaging the healthcare system
  • Affected individuals, currently not seriously affected by symptoms of the disease (or in denial) and have not presented to a HCP

Of these, the second group probably represents the best target and tactically, there are a number of initiatives that the orphan drug marketer can employ to identify them. 

  1. Focused dialogue – with specialists that encounter the individual symptoms from within a rare disease spectrum. For example in the case of Fabry disease, this would include nephrologists, neurologists, gastroenterologists, ophthalmologists and even cardiologists
  2. Case study dissemination – individual case studies can be very revealing about typical patient journeys. Every ‘touch-point’ on that journey should be seen as a target for receiving the case study
  3. Literature reviews – can be used to identify the authors of case studies who have the potential to be ‘champions’ for a particular rare disease
  4. Education/awareness campaigns – if properly conducted and realistic objectives set, these can be highly effective and the modern, social-media, tech-friendly world gives us tools to use that even 5 years ago were unavailable

It can seem like a daunting task to identify patients who can benefit from the incredible scientific and human resources committed to bringing an orphan drug to market, but the rewards for everyone involved – the patient, their carers, doctors, and of course the manufacturer – are well worth the effort. 

A needle in a haystack can be found – you just need the right approach, the tools for the job, and belief. 


Since this article was first written, Dice has been fortunate to work on six rare disease brands, for clients such as Alexion, Amicus, Biogen, Recordati and Santhera. Our experiences echo the sentiments of the article.

On the whole, working in rare diseases can be very rewarding, and it’s very satisfying to know that you can make a real difference to people’s lives. For example, our client Santhera had a letter from an ophthalmologist who said that our disease awareness campaign for Leber’s Hereditary Optic Neuropathy (LHON) had alerted him to a patient he had recently seen with a mysterious loss of vision. The symptom checker made him realise that he was faced with a patient with this rare condition and he was able to intervene to save his sight.

That really does make our job feel very worthwhile.

Rare Disease Day is taking place on February 29th, 2020. To find out more about Rare Diseases, you can visit the Rare Disease Day website here

Have you ever wondered what the statistics actually mean in the data that support the core claims made on behalf of your brand? Or how percentage differences between the effects of an active drug and a control, such as a placebo, are calculated?

As medical copywriters, we are asked to explain these calculations often enough to suggest that there may be widespread misunderstanding out there. That’s understandable and nothing to be embarrassed about: after all, we are marketers not mathematicians.

Statistics can be a tough subject to get your head around: the double negatives of disproving a null hypothesis is just the start! That’s why the team at Dice have prepared an informational resource for medical or pharma product and marketing managers, that provides an overview of understanding and managing the basics of statistical data.

You can download this resource by subscribing to our email newsletter below, you’ll then be presented with a download link immediately.

I had to have a partial knee replacement during the year and my experiences were a distillation of modern inventions made real.

Firstly, 3D printing. When this first came into the public domain, I couldn’t really grasp its utility. It seemed a clever idea but it was hard to imagine any practical applications. How wrong was I?

A CAT scan of my knee was sent to a US company who would produce a 3D model of my knee joint from which a made-to-measure prosthesis could be manufactured.

Secondly, robotic arm surgery. Of course the surgeon still holds the scalpel and the saw, but the robot dictates EXACTLY how much bone to excise for the perfect fitting of the new joint – down to fractions of a millimetre. 

All of which means, nine weeks post-op, I’m close to being back to normal. But of course huge thanks must also be extended to my surgeon Mr Nick Morgan, who was great throughout.

It’s another example, as if we needed one, of how lucky humans of today are in terms of the amazing healthcare at our disposal. I shudder to think about what it must have been like to have lived in the era before anaesthesia, antiseptics and vaccines.

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The pharmaceutical industry has entered a period which will test the most fundamental aspects of its relationship with patients and payors around the world. 

I wonder if it has prepared itself for the battle ahead?

By ‘battle’ I mean the constant need to justify the prices of cutting edge medicines that it brings to market. Gone are the days when companies could launch a primary care drug with literally millions of potential patients and charge, say, £250 for a year’s treatment. That was easy. But what if you have a life-saving drug for an ultra-rare disease? You don’t need to be a mathematician to work out that any commercial return on the R&D investment will require a price not in the hundreds of pounds, but in the hundreds of thousands of pounds – and even more.

Indeed there are now many examples of such therapies and earlier this year, the FDA approved a single-injection gene therapy for a rare childhood disorder at a price of over $2 million. This inevitably pushes the debate into philosophical territory and my fear is that this is a debate that the industry can’t win.

Why? Because hard science combined with the need for shareholder reward does not sit comfortably in a philosophical framework. The question of ‘can you put a price on life?’ is centuries old and these recent developments only put a sharper focus on the question, but they don’t provide an answer. 

Think of it from another perspective by turning the drug development process on its head. Imagine if you set out on an R&D project, not with the intent of finding a way of repairing a specific genetic fault (almost pure science), but by being asked the following question: “how much are you prepared to commit to your R&D budget to save this child’s live?”

The answer from a purely commercial perspective would actually be unpalatable. No amount of genuine sympathy for the child’s predicament amongst shareholders would overcome the realisation that the budget would have to be hundreds of millions of dollars at least and with no guaranteed return on that investment.

Eventually, if this dilemma isn’t resolved, drug development for rare diseases will dwindle, which would be a tragedy on several levels. Firstly, and most importantly, patients will continue to suffer and die from potentially treatable disorders. Secondly, science will suffer because some truly ground-breaking discoveries have been made in the search for these drugs. Thirdly, the reputation of the pharma industry will once again be tarnished because it will be portrayed as only being interested in a return on investment and not patient outcomes. 

Interestingly, this scenario is already being played out in the sphere of antibiotic development. Despite everyone, including governments around the world, recognising the need for new antibiotics, there are currently only around 40 candidates in development. That compares with over 3,000 immuno-oncology drug candidates. It’s not hard to see why when a recent (and rare) new antibiotic entrant to the market is priced at around $5,000 dollars for a course of treatment compared with over $200,000 for a new immune-oncology drug. Yet the former SAVES lives, and the latter merely extends for a few months.

So there are some very profound questions that need well-thought-out answers if the pharmaceutical industry is going to safeguard its future. My worry is that nobody is even trying to address the issue and time is running out. 

This is a truly great industry that we can all be proud of. We don’t always get it right, but good news is everywhere even if you take just a cursory look. People are living longer, surviving cancer, thriving with rare diseases and so much more. At the very least, we should make sure these stories are out there in the public domain because, while governments and payors around the world may struggle to understand the true value of our discoveries, patients always will.

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Every drug has its core clinical papers, and usually a set of ‘less important but maybe useful/interesting’ ones that need to be assessed. For a copywriter, this often means that, at least at the outset of a project, you are faced with a ring-binder full of papers to work your way through. So it goes without saying, that a good scanning technique is a vital tool to be able to get a quick grasp of the strength and breadth of data supporting a particular drug.

This is how I go about it, but there are no doubt other, equally valid approaches.

As an example, for the purposes of writing this article, I have used a study that was published in 2014 here. The study looked at real-world clinical outcomes in multiple sclerosis patients in order to ascertain the ‘value’ of treating with disease-modifying-drugs (DMDs) – either immunomodulatory (IM) or immunosuppressant (IS).

Step 1 – establish the broad credibility and veracity of the study

1.1 Go right to the end and check to see if there were any commercial sponsors: that may nuance slightly the veracity of the data – and the word ‘nuance’ is important here as I am not suggesting that company-sponsored studies are any less rigorous than non-sponsored trials! But it can sometimes be thrown back at you as an objection, so it’s important to be aware of it.

1.2 Where was it published? Is it an obscure journal or more mainstream? This is important because most authors would love their studies to be published in high profile, respected medical journals, but of course that depends on the ‘strength’ of the study. In this case, the Multiple Sclerosis Journal is a credible, disease-specific publication that is endorsed by leading MS research groups such as ECTRIMS and ACTRIMS. 

You can also search for the Impact Factor of the journal which will provide you with a measure of its importance or rank based on the frequency its articles are cited. In the case of the MSJ, its impact factor of 5.649 places it in the top 7% of all journals – so very respectable, although admittedly not at the dizzy heights of the NEJM or The Lancet. 

To find out more about the subject of Impact Factors, just go on-line, where there is a plethora of useful information. It’s an interesting and informative read. Suggestion: search ‘impact factor of medical journals’

Step 2 – what are the headline findings?

2.1 Go straight to the conclusions in the summary on the first page!

That’s right. Don’t delve into any details until you’ve absorbed the main outcome of the study. 

In this case, the conclusion signposts that there could be some very useful findings in this study, from the top-line (to paraphrase: “These drugs work!”) to the more subtle (“They still work, but to a lesser extent, if you delay treatment”). That is a very enticing conclusion for a copywriter, because you can be confident that there will be some useful nuggets of data on display further on in the paper. 

Step 3 – look at the top line results

3.1 You might have noticed that we still haven’t gone past page 1 yet, but that’s fine. The paragraph relating to Results in the Abstract gives you some strong clues as to how excited you should be about the data within. 

In this case, the very first result that is given jumps out at me. The risks of progression to a major disability milestone are reduced by 94% in patients treated with IM drugs compared with untreated patients. That really whets my appetite – I want to know more!

3.2 Look at the statistics. It can’t be avoided – as dry as they are, such a big result that we just mentioned needs to be statistically credible if we are to use it in any promotional sense. So we head to the main results section of the paper – page 3.

Here, at the foot of the right hand column you can immediately see that this seems to be very solid data from a statistical view point. 

The CI 95% figures mean that this reduction might ‘only’ have been 91%, but could also have been as high as 96%, and a p value of <0.001 means that there is less than a one in a thousand chance that this result is purely down to chance and not a treatment effect.

See our article ‘Basic Statistics for Marketers’ if you want to delve deeper into the subject – INSERT LINK HERE]

Step 4 – is the patient cohort credible?

Now that we are excited about the data, we need to check that the patient cohort wasn’t skewed in any way, such that a good outcome was inevitable. This is the point where you have to do a little more in-depth reading. 

I usually begin by scanning the Patients and Methods section (page 2), but in this case, I found more interesting information at the beginning of the results section. There is one stand-out paragraph:

What I deduce from this is that the study not only contained a good number of patients, but also that, very unusually, they were all living under similar conditions as Sardinians on the same island. To understand the power of this, imagine if it was a cohort of 316 patients spread across the whole of Italy: that would immediately raise suspicions that patients had been specially selected in some way and such a low number would also undermine the credibility of the findings. 

Then delving deeper, table 2 on page 5 reveals that the patient numbers for the group my client is interested in (those taking immunomodulatory drugs and EDSS 3 as the milestone) is still very credible: 1306 treated patients vs. 781 untreated.

Note that this particular consideration is sometimes not an issue. In some of the rare diseases we work with, drugs are often trialled in only a handful of patients by necessity

Step 5 – are there any worrying safety signals?

The efficacy data mentioned above are exciting, but that would be easily undermined if in that same cohort of patients there happened to be a high morbidity or mortality impact. In this case, the study was not designed to reveal any safety signals, so there is simply no mention of safety throughout the paper. 

You might think this is unusual, and it is, but the immunomodulatory drugs that were included in the study have been on the market a long time, and their safety profiles have been very well defined in many other studies and treatment databases.

Step 6 – Are there any other major caveats?

Sometimes, when reading a clinical paper, it becomes clear that the authors are themselves slightly ‘uneasy’ about reading too much into the findings because of some inherent flaw in the methodology, or data collection etc., and if this is stated strongly, then you have to take notice of it. 

In this paper, the authors quite fairly point out that observational studies per se have their drawbacks, but then they do contextualise this in a positive way. 


That quick scan of an important clinical study took about 15 minutes, but if done correctly, it tells the copywriter that this paper is worth more detailed investigation, or if it can be dismissed. It’s a useful skill to master for the busy marketer or medic. 

To share your views on this article please contact me directly on [email protected]

My career in the pharmaceutical industry began in research and then took a swerve to the commercial side in the early 1980s. So my working life has involved nearly 40 years of studying, selling, marketing and writing about drugs. 

Needless to say, pharmaceutical R&D focus and direction has changed dramatically in that time, and hence so too have the drugs that are developed and launched. It’s remarkable to think that in my early stint as a GP medical representative, I was selling a very basic pain killer (a variant of ibuprofen) and a first generation cephalosporin antibiotic – hardly cutting edge medicine!

Over the years there have been some standout ‘super’ brands – Losec and Lipitor instantly come to mind – and usually the reason for them achieving that blockbuster status was the fact that they were very effective (and ‘safe’) at fixing common problems that every GP encountered. But around the turn of the century, it became clear that all the low-hanging fruit had been harvested. Common conditions such as hypertension, asthma, hypercholesterolaemia and others had largely been ‘fixed’ and we see the evidence of that now in the rapid growth of the elderly population. So it was necessary for the industry to focus on more difficult challenges such as cancers, rare diseases, autoimmune disorders and so on. 

In this endeavour, the pharmaceutical industry has been phenomenally successful and doesn’t receive the recognition it deserves amongst the general population. This is sad, but industry shareholders around the world probably don’t care about reputation, as long as the commercial returns from this intense R&D investment continue to be realised. So I could have written this article about the ‘best-selling’ drugs of the 21st century, but instead I wanted to focus on those that have made the greatest medicinal impact – the ones that have changed the way doctors think about disease management and have expanded the horizons of what we believe to be possible. This is purely a personal list and no doubt arguments could be made for lots of others. If you feel strongly that I’ve missed some obvious contenders then please get in touch – I would love to discuss them with you!

Number 5. Adcetris

The mode of action of this drug is a thing of beauty and that’s why it makes my list. It’s not the only modern drug that has an elegant MOA, but for me it just has the edge. The reason is that it can be described in layman’s terms in a way that is easily understandable, for example as follows:

It’s an antibody that carries the cancer drug directly to the cancer and squirts it in to individual cancer cells to kill them. Which means your healthy cells are not harmed.

Such simplicity belies the incredible science behind its development, so it’s important to remind ourselves that this isn’t about science, but people’s lives. In this regard, Adcetris doesn’t disappoint. I’ve heard on the grapevine that oncologists treating Hodgkin’s lymphoma were ‘blown away’ by its efficacy. Just a great story all round. 

Number 4. Gleevec

A diagnosis of chronic myeloid leukaemia in the last century was a fairly certain death sentence and the treatments that did exist were harsh and invasive. Enter Gleevec: a once or twice daily pill and bingo – disease controlled. (I know this is a gross simplification, but its availability really was transformational). As part of its mode of action description, I simply love the words ‘programmed cell death’ – a marketer’s dream. 

Number 3. Atripla

I’m old enough to remember reading in medical journals of the 1980s reports of a mysterious illness that was soon known as AIDS and fairly soon after, HIV was identified as the cause. It’s remarkable to consider the achievements of the pharmaceutical industry in transforming a deadly disease into a manageable chronic condition in only a few years. The early treatments were unpleasant oral therapies but the improvements and refinements came quickly. Once HAART (highly active anti-retroviral therapy) was identified as the way forward, the race was on to deliver this complex regimen as conveniently as possible. 

This was the genesis of Atripla – one pill, once a day, containing an effective trilogy of anti-retroviral drugs. For the HIV+ve person used to popping sometimes dozens of pills per day in complex regimens, Atripla really was a game changer. 

Number 2. Keytruda

After my early stint as a GP representative, I spent 18 months as an oncology specialist account manager selling a range of cytotoxins known as vinca alkaloids. In those days, for patients with advanced cancers (of virtually any description) oncologists were trying to eke out a few more weeks or months of life by using highly toxic regimens. But that’s all they had at their disposal. A particularly knotty problem was NSCLC (non-small cell lung cancer), which seemed impervious to any treatment. 

If, at the time, you were to present them with the kind of efficacy results delivered by a Keytruda-enhanced chemotherapy regimen, they just wouldn’t have believed it possible. 

That’s why it gets my vote, because my personal dealings with oncologists tells me that this was a very high mountain to climb. 

Number 1. Humira

Not only a great drug, but a great name. Humira was the first monoclonal antibody blockbuster and thanks to that status, it spawned innumerable MABs and other variations of immunomodulatory drugs. But most importantly, it transformed the lives of patients who hitherto had had little relief from chronic diseases such as RA, Crohn’s and psoriasis. It now has 14 indications, which is a testament to its incredible utility.

So that’s it. Whether you agree with my list or not, these five drugs are exceptional and there are millions of people around the world whose lives have been enhanced and saved by their development. There are so many that nearly made the list, such as Revlimid and Spinraza, but my arbitrary opinion made me reluctantly leave them outside the top five.

Please email me if you would like to discuss this article or propose an alternative ‘Top 5’.

To share your views on this article please contact me directly on [email protected]