When cancers are well understood, it’s often because they affect a large-scale, globally diverse population or because they are featured in prominent awareness campaigns that inspire further bumps in research and development activities. However, there are many more types of cancer that fall under the “rare disease” umbrella and require a far savvier approach to marketing.

This topic is significant because of a huge unmet need in the so-called “orphan disease” arena. Pharmaceutical brands operating in this market have unique challenges for which standard research, development, and marketing approaches do not usually apply. Strategies that best fit the rare disease sector should focus on category leadership and value proposition, meeting unmet needs, and decision-making support.

What Is a Rare Disease?

What qualifies a disease as rare? Rare diseases are defined as affecting fewer than 200,000 patients and often have variations that demand different, individualized therapeutic approaches. As of 2018, there are  between 6000 and 8000 rare diseases, including different cancer types, and 95% of these have no available treatment options.

In Europe and the United States, 1 in 10 people has a rare disease. However, the patient population actually benefiting from a so-called orphan drug is extremely narrow by definition. These medications are designed to help a very limited number of beneficiaries and are usually high-risk and expensive to develop. That said, there is a clear need for orphan drugs, and their marketing relies on alternate strategies for establishing value.

Challenges of Developing Rare Diseases Therapies

A major challenge in the rare disease sector is that there is no access to a large-enough patient sample, which is traditionally a requirement when bringing a medication to market. This limits the ability of researchers not only to generate enough data—with enough power—but also to replicate results through further studies or in a real-world population.

Additionally, the mechanisms behind the disease are often poorly understood or have so many variations that it is difficult to pinpoint an appropriate therapeutic target. And even when this is possible, controlled studies (in which a portion of participants receive a placebo instead of the therapy) are generally avoided, out of ethical concerns.

Without the mountain of clinically significant data needed to validate the efficacy and safety of a given medication, marketers of orphan drugs need to be much more innovative in how they frame their narrative. Strategies include establishing category leadership, scientific storytelling, or providing a platform for informed decision-making.

Category Leadership as a Marketing Strategy

Category leaders are not always the ones to be first to market or touch on more popular corners of cancer research. You can position yourself as a category leader by being the first to identify a new target, novel indication, or unique combination therapy. In the absence of quantitative evidence, category leadership establishes trust, credibility, and authority.

As a category leader, exercise your voice of authority by presenting yourself as able to bridge the gap between patients and providers. Your marketing narrative should simultaneously recognize the unmet need of patients while reminding providers of your organization’s relevance and experience in oncology marketing.

Typically, oncologists positioned as leaders in their field have more room to experiment. When it comes to developing a treatment plan, they can piece together different sequences and therapeutic combinations that are unique and effective for each individual patient. Research and marketing for rare diseases can also benefit from this approach.

Scientific Storytelling as a Strategy

In the absence of large amounts of quantitative evidence, the best strategy in marketing orphan drugs is to establish the unmet needs of a certain population—be it patients, physicians, or stakeholders—and position your drug as able to meet those needs. To demonstrate value in a way that is not 100% grounded in data, a good device is scientific storytelling.

Scientific storytelling already lends a much bigger strategic impact in the oncology sector, especially in rare cancers. Not only does it express the effectiveness of a certain therapy, but this approach also motivates patients to continue their treatment. In this way, you create value by illustrating the reasons your particular drug is meaningful to patients and providers alike.

Tim Mitchell, in his article “A Prescription for Reinvention: The New Realities of Oncology Marketing,” shares an example of this in which he describes a tumor-shrinking agent as “starving the tumor.” The language of storytelling combined with scientific knowledge and experience can be positive and generate awareness and, importantly, keep your audience engaged.

Informed Decision-Making as Strategy

Patients are more empowered today to engage and manage their disease through therapy; they are even more likely to share their experiences with other patients through disease-specific online communities. These are especially beneficial in rare diseases because the affected individuals are potentially few and far between, and a shared experience surrounding a rare disease is extremely powerful.

Therefore, engaging with patients as decision-makers is a relevant and appropriate strategy. Marketing orphan drugs without backing evidence from giant trials and prospective analyses is a big challenge; however, decision-making–based strategies are all about actively engaging patients and care providers in the context of developing a treatment plan. In this regard, supporting patients through decision-making activities helps them get the therapy they need.