By Deborah Lotterman, Chief Creative Officer at precisioneffect, and Jamie Timmons, MD, Group Account Director at ETHOS Health Communications

 

The first rule of rare: There are no rules.

There are over 7000 different rare diseases—each with its own symptomology, history, and clinical and political landscape. Most are virgin territory for marketers; only 5% have an approved treatment.

The opportunities are extraordinary – to dramatically impact patients’ lives, to rewrite the rules of medicine, to bring together patients and build communities.

The pitfalls may be just as great – to raise false hope, to be perceived as an interloper and opportunist, to create discord and fragmentation.

What we’ve learned in our years as marketers (and Jamie’s tenure as an internal medicine physician) is there are, in fact, a number of guardrails to grasp as you bring a novel treatment for rare to market. We think of them as the ABCDs.

 

A is for Access

Access is foundational for every drug—patients don’t benefit if they can’t get the drug—so a thoughtful approach to access is essential. Rare diseases are often chronic, requiring regular, even daily, treatment over a lifetime. When payers have concerns about the price or the expected use of a new treatment, they will often develop strict utilization management criteria that can slow and even deter patients from receiving necessary care.

An antidote is to start conversations long before launch. By definition, many payers are not familiar with the disease. Supplying context and evidence helps lay the groundwork for equitable coverage and mutually beneficial contracting.

Begin by helping them understand the size of the population. If you are truly only going after less than 5% of the population, demonstrate that, and help them understand the budgetary impact. Be willing to engage in discussions regarding what constitutes the correct patient population. Many companies avoid this as they don’t want to “write their own PA.” Suggesting appropriate parameters of a prior authorization generally works out more favorably than living with one the payers create.

Through it all, our colleague, Todd Edgar, Senior Vice President of the Access Experience Team at Precision for Value, suggests transparency: “Have a well thought-out and justifiable price, and be prepared to explain how you arrived at that number. Be sure to call out any direct savings the payer may realize by using your therapy.”

While pricing is a negotiation with many parties, helping ensure patients have consistent access is an experience that manufacturers can control. For patients out in the real world, positive life moves—changing jobs, relocating, traveling, experiencing milestones like heading to college or getting married—can become serious health issues if access is interrupted. Companies have the opportunity to be a vital means of assistance in ensuring smooth transitions. It’s a level of support that patients expect from a great brand.

 

B is for Brand

When it comes to truly impacting the trajectory of a rare disease, it’s imperative to reach every patient who requires treatment. Those who live with rare diseases have often had bruising experiences with the healthcare system. It can take years to get a diagnosis; they often bounce from specialist to specialist in search of answers. Those who experience pain may have been perceived as drug- or attention-seeking. They are looking for individuals and institutions they can trust. A strong brand is a pathfinder, helping to attract patients and usher them through the treatment experience.

Developing a strong corporate brand can create an essential bridge to patient families, advocacy groups, and the treaters. The company should state a clear mission and commitment to the space, offer transparency about the organization’s goals and initiatives, and set up communications platforms – web, social, and CRM that keep all audiences in the loop on progress. The steps are seemingly simple—part of any drug launch—but getting the tone and cadence correct can be nuanced. Success is highly dependent on establishing strong relationships and gathering insights from the community from the start (see C for Community).

Defining the product brand is no less critical. “First and only” is too often “short and sweet.” Rare is no longer rare in pipelines, from companies large and small. Long before a rare drug therapy comes onto market, highly educated patients and key opinion leaders will be developing a point of view, comparing with existing therapies and those in the pipeline. Distinctly characterizing your product brand sets your control over the narrative in the marketplace. Rare disease communities are often quite noisy. Your brand needs to have a sharp, relevant message to be heard.

Broadcasting that message in rare is a misnomer. Think of it as narrowcasting.

SEM will certainly catch those who search for drug or disease by name. But to reach every patient, to find those who may have stopped looking for treatment or fallen out of the system altogether, search isn’t sufficient. It can become far more powerful to turn search around and look for telltale signs and markers suggestive of the patients. Do they cluster in geographic locations? Do they rely on assistive technologies or apps? With these populations pinpointed, you can query other words they might have searched. This helps paint a media profile of likely patients, and now you can deliver messages (in display or programmatic) to a narrower group with higher efficiency.

Leading up to and at launch, most advertising and nonpersonal promotion should be driving patients and physicians to the website(s). There they should encounter compelling content that is not available elsewhere (see C for Community). On disease awareness sites, this can translate to delivering basic disease information in formats and language that is far more engaging and accessible than what audiences encounter on traditional information sites. On brand sites, this might be explanations of the mechanism of action, dosing and administration, and patient stories that go beyond the pharma standard, with surprising storytelling, character development, and production design. Having a full (and consistently updated) view of what competitors, advocacy groups, and YouTube is offering will clarify the white space. All these efforts should be designed to drive engagement, to encourage users to take a definitive action—downloading talking points, signing up for updates—that grant permission to continue the conversation.

When it comes to conversations, a chatbot on the site delivers multiple benefits. For patients or physicians, it enables them to circumvent navigation, searching specifically for the information that interests them. On a patient support site, chatbots can triage requests, if appropriate, directing patients (or healthcare professionals) to information without involving human intervention. Behind the scenes, seeing how users are phrasing questions and exactly what they are requesting can help optimize existing content and shape future development.

 

C is for Community

Every pharmaceutical company is patient-centric but those looking to bring a treatment for rare must be community-centric.

Patients, families, advocacy groups, online influencers, front-line treaters, specialists, multidisciplinary team members—it’s a long list. This is your greatest source of insights, your most powerful set of allies, and most ardent cheerleaders.

In many rare disease states, the patients (and/or patient families) are highly engaged, highly informed experts with knowledge hard-won from fighting for a diagnosis, to obtain services, to bring more attention and funding to the disease.

It’s actually rare that brands need to build communities; they are already out there as official advocacy groups and informal circles on social media. What a company can do is bring these groups together towards a common goal of ushering the therapy to market. Establishing relationships early, understanding their needs and aspirations, can ensure that the services, information, and leadership offered are additive rather than duplicative.

Deep relationships with KOLs are foundational. But it’s also important to speak with front-line treaters and those clinicians who have the opportunity to identify patients. Early on, you want to gather insights on their frustrations and aspirations.

 

D is for Diagnosis

This is really where it all begins. To ensure the greatest impact on the disease, patients must get diagnosed accurately and quickly. Yet so much stands in the way.

When confronted with an unusual set of symptoms, frontline physicians are often pulling from med school learning, years or decades past. Those definitions become ossified, and patients that don’t fit the classic presentation can be missed.

Early education efforts to physicians should support a screening mindset.

Prevalence is a given. There’s no way to increase the number of needles in the proverbial haystack, but the odds of success will increase if educational efforts help highlight more likely needles rather than hunting for perfect ones. A sponsored testing program can remove barriers to diagnosis and may mean seeing more negative results. But it also increases the likelihood of finding those patients who are less severe or not textbook, perhaps some who need the therapeutic now and some who will become future patients.

 

E is for Early

We didn’t include E but it shows up in every section above. An exceptional rare disease treatment launch is dependent on involving all key audiences early.

Today, it’s rare. Tomorrow, we expect these principles to apply to the increasingly personalized medicines that will dominate the market. And in fact, rare, personalized, or for greater populations, there is one rule: Know thy customer. Early.