Rare Diseases. Challenges and Considerations for Geographic Expansion Using Distributor Partners

Recently, I have received enquiries on "Rare Diseases" from my followers and around one thousand subscribers to my book updates newsletters:

The enquiries have ranged from:

"What are my thoughts about distributor models for orphan drugs?"

"Are there any peculiarities for geographic expansion with a portfolio of medicines for 'Rare Diseases'?"

"Could I provide some guidance based on my experience with distributor models, which I have covered in my book, on how operating in 'rare disease' portfolios presents special challenges with distributor models, selection, and commercial considerations?"

"What might be critical success factors for rare diseases in geographic expansion?"

Given the list of enquiries (which I welcome), I am authoring this first article to gauge interest in a Newsletter focused exclusively on "Rare Diseases." If there is sufficient interest, I will create a new series of Newsletters on this topic.

This article describes the general scene before investing my time in a Newsletter that will follow if there is sufficient interest.

I am particularly keen to direct my Newsletter to CEOs and senior decision-makers in charge of a portfolio in rare diseases facing the following situations:

1. Geographic challenges in terms of assessing markets for investments.
2. After an initial expansion, those who have achieved registrations and revenue now want to raise their game for higher revenue to create a return on their investment over a five-year period.
3. Those wishing to enter new markets who want or need to better understand the key factors to their success in distributor identification, selection, and commercial models.

I am not looking for many CEOs and senior decision-makers, given that rare disease medications form a small number in an ocean of therapeutic agents. However, my key requirement is engaging with them and stimulating discussion on their issues and challenges in this sphere.

Given its high specificity, the newsletter is unlikely to interest students or those not operating in the rare diseases space. Unless anyone works in this space, they are unlikely to appreciate or contribute to discussions and comments on my posts and Newsletters on this topic.

I have set up a group to debate and discuss their specific concerns. I will publish the newsletter only within the group and not for the general readership of the broader LinkedIn community (link to group and details below).

This general article sets the scene before deciding if there is enough interest in group membership to write the Newsletters.

Are the Terms Rare Disease and Orphan Drug the Same?

The simple answer is "no". But they are closely related.

Rare diseases affect a small number of people. They are often considered "orphan diseases" because pharmaceutical companies may neglect to invest in developing them due to their limited market potential.?

Orphan drugs are medications specifically developed to treat rare diseases. The term "orphan" highlights that these drugs might not have a large enough market to incentivise traditional pharmaceutical research and development.?Some current therapeutics have applications as orphan drugs.

I can illustrate this with Ibuprofen. While ibuprofen is a common drug to treat pain, in the context of rare diseases, it's an "orphan drug" when used to treat patent ductus arteriosus (PDA) in premature neonates, a condition where the blood vessel connecting the aorta and pulmonary artery fails to close after birth.?

Rare diseases and orphan drugs are related in that orphan drugs solve the challenges of treating rare diseases, which often lack adequate funding and research due to their low prevalence.?

But of course, things are rarely as simple as this. The term "rare" is wide open to interpretation.

The US definition of a rare disease is one that affects less than 200,000 individuals.

While the corresponding number in Japan is 50,000 and in Australia is 2000.

These numbers translate to a prevalence between 1 and 8 per 10,000 population. The European Community definition is less than 5 in 10,000, and the World Health Organization has suggested less than 6.5-10 in 10,000!

Cystic Fibrosis is classed as a "Rare Disease". Over 11,000 people in the UK have cystic fibrosis; that's 1 in every 2,500 babies born. Cystic fibrosis affects around 100,000 people worldwide. (Source: Cystic Fibrosis Trust). Other estimates put it at around 160,000 cases worldwide.

To put such 'rare' prevalence into context:

Hypertension: It is estimated that 11.8 million adults in England had hypertension in 2017, equating to around 26.2% of the population or approximately one in four adults (Source: NICE). Globally, the World Health Organization (WHO) estimates that 1.28 billion adults have hypertension.

Breast Cancer: The most common cancer in women in the UK, accounting for 30% of all new cancer cases in women. (Cancer UK 2021). There are around 56,000 new cases of breast cancer in women every year; that’s over 150 cases every day (Cancer Research UK).

So in Cystic Fibrosis (CF), the data states around 11,000 people have the condition, and globally, the estimates range from 100,000 to 160,000.

Whereas globally, the WHO estimates that 1.28 Billion adults have hypertension. That's 160,000 in CF globally versus 1.28 billion for hypertension! The scale difference in commercial opportunity is enormous.

But before you all get excited and ask why anyone even contemplates operating in rare diseases, let me highlight that the difference is in competitiveness.

Hypertension has many players (hundreds of therapeutic agents and thousands of copies of the same). While CF has very few orphan agents competing for those 160,000 cases worldwide.

To square that circle, orphan drugs must be a much higher cost to pay back the development investment and risks.

The UK's National Institute for Health & Clinical Excellence (NICE) makes this distinction clear:

"The main criterion currently used by the National Institute for Health and Clinical Excellence (NICE) for approving drugs for use in the United Kingdom is that cost should be below about ï¿¡30,000 per quality-adjusted life year (QALY). This suggests a different method of defining a rare disease. If an orphan drug costs more than (say) ï¿¡30 000 per QALY, and if a disease is a rare disease, and if an orphan drug is used to treat it, then a rare disease could be defined as one whose treatment costs more than ï¿¡30,000 per QALY".

Maybe we should distinguish rare diseases as those defined by prevalence from orphan diseases that are defined by cost.

Using cystic fibrosis as an example, geographic expansion presents many challenges common to those outlined in my book. However, it also poses several challenges that may be peculiar to CF, and I will explore the rare disease opportunity in future articles and newsletters.

If senior decision-makers in the group I have set up for Rare Diseases show sufficient interest, I will publish a series of Newsletters specific to their interests and challenges.

Here is the Group I have set up. Please note the details about the group's rules and membership. I will invite selected connections and contacts to start the ball rolling.

https://www.dhirubhai.net/groups/13186328/

Do you like what you have read in this article? Are you working in Rare Diseases and Orphan Drugs? Are you a senior leader, CEO, or decision-maker accountable for the P&L and geographic expansion?

Then join my group! Click the link to the group and request membership.

About Samkoman Consulting Ltd:

I started SCL after a stellar career at AstraZeneca. I advise senior Boardroom executives on their commercial challenges, geographic expansion, revenue growth for B2B clients, and selling to complex, large, high-value accounts.

Growing the skills and capabilities of your organisation's staff on distributor and go-to-market models that can deliver the return-on-investment declared in business cases is likely the most critical investment you will make—an investment in your people.

Your people will become stronger and more focused. They will be more aware of models that can scale successfully and those that cannot and why. Use my experience and methodology to deliver higher-value revenues, growing year-on-year to expectations and beyond.

Explore with me how we can bring those skills to your organisation in the area of "Rare Diseases" geographical expansion and driving stronger business performance.

In my diary, use the link below to book a free, no-obligation Teams or Zoom call. You have everything to gain on this first step that could transform your business in 2025.

Book a free consultation with me: https://calendly.com/amitvaidya2021/