Insight into the Unique World of Ultra Rare Diseases

Insight into Rare and ultra-rare disease state marketing
Insight into Rare and ultra-rare disease state marketing
rare disease

A Rare Disease is Different


In my career I’ve moved from what’s considered Big Pharma – Eli Lilly, to Biotechnology at  Amgen and Gilead, to a very small Biotech – BioMarin.  But they are all within the same industry of Biologics-Pharmaceuticals or “BioPharma”.  Therefore, you could assume that Sales & Marketing would be pretty similar in each of them.

But that’s simply untrue. It’s just not a move from small molecule to large, it’s also a move in patient population size and focus.  So when you move into the ultra disease state, you’ve stepped into a world of its own!

ultra rare disease

You can visually see there’s at least the difference in size and complexity of molecules. You move from combining the right organic ingredients to create a specific small molecule (like baking or cooking), all the way to a Biologic which a targeted process of a living cell replicating parts of rDNA. (Talk about crazy changes!). But, with that movement, you see less side effects and why biologics are always preferred.

This ends up affecting the “how” you take the drug.  Is it a pill, do you need to do it with a needle (injections) or even have to go into a hospital for an IV infusion? It’s all based on size and complexity.  And in general, you normally see the ‘small molecule’ drugs, over the counter at your local pharmacy.

You also see a major difference in patent types.  Small molecules like Aspirin – you patent the compound; but, with biologics you patent a process.  This is why it took so long to create generic “biologic” product.  It’s easy to figure out the ingredients for aspirin, but to do that and replicate the same process for a biologic adds an increase in complexity.  And with that increase, you guessed it – you see in an increase in cost.

It also creates a difference when it comes to the FDA drug approval process. Rare disease are primarily found in children. With some so devastating the mortality rate in children is increased.  So it’s why FDA will grant special “fast track” approvals.  Anything that can be done to save a child’s life is given special priority.  (At least there’s one thing the US government is doing right!)

Although, most may assume there isn’t a big difference between between drug types, the reality is that they are very different. And although understanding the science behind them may be difficult, it is essential to working with the company. You can’t expect to achieve the same results if you try to use the same methodologies for viagra versus hemophilia patients.  It just doesn’t make sense and it just won’t work.

Hopefully, with the quick overview of drug types, you can now see why it’s critical for suppliers need to know their business prior to pitching/contacting. Considering it’s  generally a good rule of thumb to understand the specific goals/strategies of a company to have them open the doors to you.

This is exactly why you can’t expect to walk into and a company producing ultra rare disease drug and assume the marketing and channel mix are the same as Big Pharma companies. J&J, Eli Lilly or even Amgen may have large media buys, but you might be shocked to realize we have none. Why would you need tv commercials when there are no other competitors in the market place and your goals are only to find and identify 4-5 new patients in a year? The differences are astounding.

With this in mind, you can now see how it becomes easy for suppliers to make mistakes. So here are some useful insights to know prior to approaching a rare / ultra rare disease state brand or company.


  1. Patients – in certain disease states there are less than 100 cases Globally! So they are very much are focus since we know them by name and patient events and support can be a high priority.
  2. Patient Location – Having the insight that certain diseases affect different populations (Asian, European, etc.) disproportionally is useful. If your patients have a genetic disease that may be focused in say the Latin American culture, then you’re going to need to have this geographic area of expertise (and how to market to them).
  3. Competitors – most likely, we don’t have any. You would assume that’s a huge plus, but it’s not. Very few companies can participate in a market space that tends to have a negative ROI / cause the business to remain unprofitable. So don’t assume we have big budgets like big Pharma and approach us with similar pricing (we really can’t afford it!).
  4. Physicians – if a disease is rare enough, physicians may have a very hard time identifying new patients, let alone treating them.  This drives an increase in percentage of spend for Medical Communication agencies vs. creative or media. Medical experience in an ultra rare disease state is KEY in marketing.
  5. Sales Force – Even though we are past the days of huge sales teams, ultra rare disease states may have less than 10 total Globally. Even if we use an outside sales, there is no reason for the team to be much larger than that. Re-thinking tactic types is not always obvious.
  6. Categories of Spend – You’re not going to see us spending millions in fleet or fleet management when the company has less than 100 Pharma Representatives. Total. And there are other categories of spend that you would see in the tens of millions for pharma, that are simply non-existent for Rare Diseases. Try to think through our use and at what scale before pitching a service we may not need.
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I won’t assume to say I am an expert on each disease state and marketing mix creation and execution.  But what I will say is that I’ve become an expert in understanding the spend and how it has been impacted in each Brand.

Personally, that’s what makes our jobs in Sourcing so interesting.  You have access to global spend with PO items and Purchasing Card/Travel Card details.  And I can see which suppliers are selling and to whom globally. Data analysis, becomes almost a source of fun – it’s just like a puzzle. And who doesn’t love a good puzzle you can get creative with?

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Ultra Rare Disease Marketing Additional Links:

Why We Should Care about Ultra Rare Diseases – LINK

National Organization for Rare Diseases – NORD LINK

Rare Disease Foundation: LINK

Rare Disease Day: LINK

Internal Category Management: LINK

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