Each healthcare sector demands different market research strategies to respond to different challenges and market landscapes. Unlike many other sectors, however, rare disease asset development poses some truly unique challenges for researchers and pharmaceutical groups looking to move an asset through the pipeline or disseminate a rare disease treatment. Many of these challenges revolve around establishing both clinical and commercial value for stakeholders, which requires creativity and exceptional scientific support to overcome.

What makes rare disease different?

When trying to bring an asset to market, there are a number of considerations virtually unique to rare disease asset development. These considerations include:

  • Smaller patient pools

One of the most important components of early asset development is establishing strong evidence-based science to generate clinical and commercial projections for an asset. With very small patient pools, however, establishing this kind of evidence can be a challenge.

  • Smaller number of treating providers

With low rates of prevalence and incidence, it logically follows that there will be relatively few treating providers in the rare disease’s field. Prescribers are among key stakeholders necessary to establish value of an early asset, which presents yet another challenge to pharmaceutical and R&D groups developing rare disease assets.

  • Lack of precedent for asset development

Aside from patients and prescribers, other key stakeholders critical to the establishment of asset value include regulatory bodies and payers. Often, rare disease assets become orphan drugs because of reluctance from these stakeholders to invest.

Despite the challenges facing rare disease asset development, it is still possible to succeed in bringing an asset to market with effective relationships and strategies.

Traditional marketing vs. the rare disease landscape

Marketing for rare disease asset development is different from traditional marketing and traditional market research. Whereas typical marketing may focus on promotional strategies, rare disease marketing faces certain regulatory differences as well as variances in the way a drug or asset is introduced (and why it is introduced). In the rare disease landscape, asset development hinges primarily upon the identification of an unmet need. With high quality scientific evidence that the asset or drug can help that unmet need, payers and providers are then engaged in disseminating these findings to as many people as possible (which can be very limited in some cases). Not only does this method support benefitting as many patients as possible, but it also supports the overall economic future of the asset. Without sustainable returns, it would never be realistic to bring the asset to market.

Where do medical organizations fall short?

Because rare disease asset development is an often misunderstood sector within the healthcare industry, many groups succumb to common pitfalls. A few of these common missteps include:

  • Not having a thorough understanding of the patient journey

Particularly for rare disease, but true of all asset development, understanding the patient journey is critical. Target audience research should yield information about prevalence and incidence, diagnosis patterns, treatment schedules, and the entire life cycle of the patient from before diagnosis to completion of treatment. While this can be challenging with small patient pools and low rates of prevalence, it is nonetheless critical information and cannot be overlooked.

  • Poor market entrance

Pharmaceutical groups that experience poor market entrances for their assets have often mispriced their assets or failed to choose an effective launch strategy. Further, particularly start-up pharmaceutical companies move a clinically acceptable asset through the pipeline to discover there is no real commercial value, which can quickly cripple years of work. For groups that do demonstrate both clinical and commercial value at launch, these organizations must still have an intimate understanding of how the market has changed and shifted in the years leading up to launch so that the asset will be in the best position possible when executed.

  • Failing to adequately educate stakeholders

If stakeholders do not understand the value of an asset, this will ultimately jeopardize its value. Stakeholders must be onboard with any strategic changes made throughout asset development, for example, adjusting commercial endpoints as a result of direct competition. Stakeholders must also understand the revenue timeline of your asset, particularly with regard to return schedules, many of which are delayed until post-launch. Stakeholders expecting an ROI sooner may be disappointed, putting their investment at risk.

Rare disease asset development is, in many ways, a field unique unto itself. Different methods of establishing commercial and clinical value are necessary to move an asset through the pipeline than with more traditional assets. Leveraging resources and relationships such as expert healthcare consultancies and medical communications agencies can be invaluable to pharmaceutical and R&D groups navigating this tricky landscape. Cello Health has a demonstrated record of moving rare disease assets successfully through the pipeline with a combination of industry expertise, research and insight, marketing, and strategic experience.