In this series, David Lapidus talks with members of the Rare Collective to debunk common myths surrounding rare diseases. In this post, Patti Engel talks about the importance of having experience in the rare disease space.

David:

Patti, your market research firm was one of the first to develop rare disease expertise, which you built from your earlier roles in industry where you originated orphan-specific commercial strategies such as REMS programs.  Based on your long, and almost unparalleled, experience in the orphan drug world, can you break down a myth that affects market research for rare disease?

Patti:

It’s all about that word, “experience.”  Too many orphan drug companies undertake research with a vendor who says “sure, we can do that.”  But what you really want to hear from your vendor is, “yes we have actually done that.”  I have seen the rare disease space grow from a place where there were no vendors—we literally had to beg advertising firms to work with us!—to a situation where firms portray their experience in conventional pharma as being relevant to orphan drugs.

David:

Can you give me an example of an area where you’ve seen those skills in conventional pharma fail to translate to orphan drugs?

Patti:

It seems to happen a lot in clinical trial recruitment. I recently got a call from a client who had asked a conventional CRO to find patients with an ultra-rare disease.  Even though the CRO was working with the country’s best treatment center for this disease, it had failed to recruit a single patient after 18 months.  This CRO was top-notch at conventional trial recruitment:  newspaper and radio ads, targeting a single physician specialty, and so on.  But rare disease patients often have a complicated odyssey of diagnosis and treatment.  Many patients may not even know they have the disease.  And even when they do, there may be no standard paradigm for clinical care, so they may be treated by an apparently-random specialty.  The drug developer wasted a lot of time and money because their vendor figured that their proven expertise in conventional trial recruitment would be relevant to an ultra-rare disease.

It’s not just about finding the patients, either.  Some treatments, like gene therapy, might require a far deeper involvement in patients’ lives than conventional CROs can provide.  If it’s a long-term trial, they might have to help patients find a new place to live near the trial site, a new church, you name it!  Participation in a trial can upend patients’ lives, so a CRO must be able to predict burdens and prevent them before they impact recruitment.

David:

You mentioned patients’ odysseys, which is a great way to describe the “patient journey” that our clients are so keen to understand.  Compared to conventional pharma, can you tell me more about how this kind of research differs for rare diseases?

Patti:

There are two differences:  fragmentation and evolution.  Let’s start with asthma as a counter-example to rare diseases.  A company developing an asthma drug doesn’t have to invest much, if anything, to understand those patients’ journey.  It’s relatively straightforward, and the clinical care paradigm is well established.  We know which specialties treat patients, which drugs ought to be used as first line, second line, and so on.

For many rare diseases, the opposite is true.  Several specialties could be playing a similar role to each other.  And for even more complexity, ultra-rare diseases often depend on a handful of specific key opinion leaders.  While it may seem like a boon if knowledge and influence is concentrated in a few KOLs, it also has significant downsides for understanding your market.  Chances are that patients can get only a fraction of their care from these recognized KOLs—maybe a KOL makes the diagnosis or designs the treatment plan.  But that KOLs may not be able to inform you about the odyssey those patients went through before they showed up at his or her clinic.  And they might not be able to address the daily challenges the patients face when they return to the community.  In rare diseases, a single physician may not have the whole picture—and even worse, they may not be aware of their blind spots.  A market researcher needs to have the depth of experience to realize when this situation occurs, and to understand how to assemble all the pieces of the puzzle.

So that’s the fragmentation issue.  The question of evolution is even more interesting, because it shows how we can have a positive influence on the disease state.  Going back to the example of asthma: it’s a well-defined disease.  Every textbook agrees on what asthma is, so there’s little controversy around who can be treated with a particular drug.  By contrast, think about how Fabry disease has evolved:  we used to think that it only affected males, and females were merely carriers.  Now we know that females do manifest the disease, but in a very different way from males.  So females’ patient journey also differs from males’.  This changes our assessment of the market size, of course, but it also means that we need to use different techniques to reach these patients and their physicians.  A market researcher must consider how the disease state might evolve, because our clients need to plan activities years ahead of time.

David:
Fabry disease is a great example of how market size estimates can radically evolve in rare diseases.  Can you tell us about other pitfalls of market sizing that you’ve run into?

Patti:

Well, as your work in rare disease epidemiology has shown, we need to watch out for founder effects with genetic diseases.  We can use those to our advantage for trial recruitment, but we also need to understand that patient numbers can be “spotty” at a local level.  The definition of a “market” for your market research needs to be flexible, depending on the specifics of the disease.  Rare diseases don’t follow a template, so it helps to have seen lots of different examples.

David:

Market size estimates can certainly go awry for rare diseases.  You and I recently discussed a case where a client had obtained a market size estimate from a well-regarded firm.  On deeper examination, it turned out to be a huge overestimate, because they used conventional-pharma techniques that are often inaccurate for rare diseases.

Chris:

That was a great—but unfortunate—example of something that’s occurring in our field.  Large market research firms might hire people with tremendous experience in rare diseases, but that doesn’t mean that everyone else at the firm has suddenly become an expert, too.  I know an orphan drug company whose trusted vendor was acquired by a large firm, but the drug company won’t work with that firm unless their original vendor team is on the project.  It’s a lesson they learned the hard way.

David:

What’s your advice to pharma companies that are trying to vet a market research firm?  How can they prevent themselves from becoming a vendor’s guinea-pig or training ground in the rare disease field?

Chris:

The basic concept isn’t too hard: you need to check for depth and breadth.  It’s great start if the vendor has already worked in a relevant disease, but dig deep and find out what kind of work they did.  Was it logistical support for a patient event, or did they do strategic work with the people who influence the field?  Also learn whether the vendor has participated in the evolution of the disease state; it helps if they’ve already worked with one of the drugs from pre-clinical to post-launch, in the US and globally.  Have they done the work to find patients?  That might start with a simple estimate of disease prevalence, but it has to evolve to include “counting noses,” which means reaching out to individual physicians and confirming that they really have patients.  This takes you from your early-stage market size estimate to your sales & marketing strategy.

There’s a similar kind of evolution for patient groups, too: did the vendor start with the parents who set up a foundation at their kitchen table, and then help those parents find medical advisors and grow into something bigger, and maybe even start funding or performing their own drug development work?

It’s important to dig deep when you vet your vendors, because rare disease market research is typically harder to validate than conventional pharma work.  Going back to the asthma counterpoint: if you’re unsure about the asthma market research report you acquired, you can probably just go out and buy another one.  But rare disease market research is nearly all custom, one-off work.  And because patient numbers are typically small, rare disease market research is especially sensitive to errors.  There’s a huge difference between a market of 2,000 patients and 200 patients: that difference could break your business model if you can’t find enough patients for a trial or obtain the price you need.  Compared to conventional pharma market research, there is more likelihood of error, yet less room for it.  So take the time to understand your vendors’ experience with rare diseases, and make sure that they’ll be educating you instead of vice versa.

David Lapidus is the Founder of LapidusData, and his proprietary models and data collections systems serve as the backbone of commercial infrastructure throughout the product lifecycle.

David Lapidus
Patti Engel

Patti Engel, President and CEO of Engage Health, has helped define the orphan drug market through her work in market development, research and strategy

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