Meet the Authors

Vince Piccioni

Vice President, Business Development

Identify Rare Disease Launch Archetypes to Determine Your Optimal Launch Approach

Do you know your asset’s rare disease launch archetype?

A rare disease is designated by having less than 200,000 patients in the US. However, when we consider rare diseases as a category, we start to form a different understanding of the scale; they affect 30 million Americans. While there are 7,000 different diseases identified, many patients have common experiences; 41% are initially misdiagnosed, sometimes taking between 15 months to 28 years to determine an accurate diagnosis.

When planning to launch a rare disease product, there are also some common challenges:

  • Smaller and more dispersed patient populations also mean there are more specialized KOLs that see fewer of these patients in a given geographic area or over a year.
  • Rare disease products are often smaller in scale to manufacture, as well as novel MOAS that are extremely expensive, which puts a strong emphasis on pricing and access for these products.
  • First-to-market treatments have additional investment needed to reach, engage, and support the entire rare disease ecosystem

We analyzed rare disease product efficacy and market maturity to develop launch archetypes that determine the optimal launch approach. Rare disease products have unique patient populations, market conditions, and complex challenges. However, archetypes can help us to prioritize focus areas for a unique, rare disease product. These archetypes are also essential to determine internal and external capabilities required to maximize brand performance.

The two most impactful parameters for a successful rare disease product launch are the product profile differentiation and the market maturity within that identified disease state:

  1. Is the product efficacious? Does it only offer symptomatic relief, and will an individual continue to take the product for the remainder of their life, or is it curative? And how do we feel that product compares to current standards? Does the product have increased complexities to navigate?
  1. How well understood is the disease? Are there several patients already diagnosed and might be on alternative therapies, or is this a completely naïve disease state yet to be defined?

We have identified four distinct rare disease launch archetypes:

  • Traditional or Classic Therapy
  • Ultra-rare/Orphan Therapy
  • Established Rare Disease Therapy
  • Next-Gen Therapy.

Considering that the rare disease category has over 7000 diseases, some assets may fall within multiple archetypes. Each one of these archetypes requires differing capabilities and expertise to successfully launch a product. Click below to learn how to identify an archetype based on these brand examples. 

If you want to learn more about how to identify your launch archetype and design support programs to improve patient experiences and access, Let’s Talk. 

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Are you launching a treatment for a rare disease? We are always happy to share our learnings from the 125+ pharma and biotech launches. Let's talk about your archetype.

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Herspiegel is a leading professional services firm helping biotech start-ups and the world’s largest pharmaceutical companies navigate the path from science to brand performance and change patients’ lives. We are committed to using strategic insights from medical to commercial to market access to solve critical problems.