• Sustainability
  • DE&I
  • Pandemic
  • Finance
  • Legal
  • Technology
  • Regulatory
  • Global
  • Pricing
  • Strategy
  • R&D/Clinical Trials
  • Opinion
  • Executive Roundtable
  • Sales & Marketing
  • Executive Profiles
  • Leadership
  • Market Access
  • Patient Engagement
  • Supply Chain
  • Industry Trends

How to Build a Marketing Plan for Ultra-Rare Disease Products


Pharmaceutical Executive

Molly Harper, vice president of commercial development at Akcea Therapeutics, outlines best practices for this unique group of therapies

Molly Harper, vice president of commercial development at Akcea Therapeutics, has made a career out of creating commercial markets for drugs to treat very rare diseases.

She recently spoke with Pharm Exec about her strategy, what the challenges are, and advice for others who are in the rare disease space. She also provided leadership advice for current leaders and those aspiring to reach management-level positions.

PE: Tell me about Akcea Therapeutics.

Harper: Akcea Therapeutics is a drug development and commercialization company working to advance transformative treatments for serious cardiometabolic diseases. Our lead compound is volanesorsen, which has been filed and accepted for regulatory review and potential approval in the U.S., E.U. and Canada for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic lipid disorder. The Akcea pipeline also includes three other compounds that are or will be in Phase 2 clinical trials by the end of 2017 for treatment of other lipid disorders with significant areas of unmet need.

PR: Describe your role at the company.

Harper: As vice president of commercial development at Akcea, I am charged with building the company’s global commercial organization to support new product development and the strategy development and execution for commercialization of our lead investigational therapy, volanesorsen, which is pending regulatory review. This has included building out our global commercial leadership teams, which includes global functions of Advocacy, Marketing, Market Access, Operations and Training, and also the country-level teams for our priority countries of the U.S., Canada, U.K., France and Germany.

What I find especially exciting is bringing together our talented, experienced, and energized team to help improve patient care, with our goal of transforming the lives of those affected by FCS. Beyond volanesorsen commercialization, our team also provides the commercial perspective into our pipeline programs as they advance.

PE: You have made a career out of creating commercial markets through strategic market development and execution. Currently this is focused on rare diseases. What are some of the challenges when building a commercial plan for a rare disease drug?


Harper: The rare disease space is often described as unique, with certain overriding patterns in product commercialization. But it is not monolithic. Every disease and development program is different – and this will often have an impact on commercialization strategies. One important distinction is the way some rare diseases are diagnosed.

For example, diseases like phenylketonuria can be identified through newborn screening. The process for diagnosis of diseases like cystic fibrosis is typically later, but well established so that a large percentage of patients are diagnosed. In both cases, many or even most patients are known. In our case targeting FCS, when we started the disease state had very little awareness among both HCPs and affected patients. There was a challenge of terminology in both the medical literature and clinical practice, and as a result, very few diagnosed patients. There was also no established advocacy community. We had to start with a focus on building awareness about not just the disease but the importance of diagnosis for these patients, which is the foundation of our commercialization strategy.

PE: How do you overcome these challenges?

Harper: We start by – and continue to prioritize - listening. Again, using FCS as an example, we initially worked to identify and connect with patients and their caregivers to better understand the experience of living with FCS. We also connected with many doctors and broader healthcare teams to learn about the areas of unmet need. You then need to bring that understanding back to every member of the organization, because it will shape not just your commercialization strategy, but clinical trial recruitment, medical affairs activities, and how we position and think of ourselves as a company. You continually have to confirm that your commercialization strategy is in sync with your goal to make a positive difference for patients.

In this case we saw that there was tremendous value in helping patients learn about their condition and connect with other patients. We focused on disease awareness and education to ensure patients are able to learn about their diagnosis, access educational resources and support, and learn about the existence of patient organizations that can provide community. We also recognized critical gaps in existing literature on this disease, and my colleagues at Akcea conducted a first-ever survey of patients to assess the impact on FCS on their quality of life .

PE: Give me an example of a successful rare-disease commercialization campaign you’ve run in the past, and how you measured its success.

Harper: To help patients understand and live with FCS, we launched FCS FOCUS - a comprehensive online educational and support hub. For the first time, people can review educational content, order resources watch videos to both understand the disease but also, more easily manage the very difficult and specific diet this disease requires. All of these were co-developed with patients, physicians and registered dietitians. 

We have all been humbled by the response to date and the appreciation we’ve heard from people living with FCS and their caregivers. We have heard of patients who recognized their own symptoms because of FCS FOCUS who are now getting appropriate guidance and care by lipid experts and dieticians.

PE: Akcea Therapeutics is working to develop a first-ever treatment for a rare condition called familial chylomicronemia syndrome (FCS), a potentially fatal lipid disorder, with a regulatory decision in the U.S. and E.U. anticipated in 2018. Tell me about the commercialization planning strategies that went into this project.

Harper: The overall commercialization plan for volanesorsen has many active components, all moving forward concurrently. As I mentioned, we started by listening to and learning from both patients and medical experts. From there, our strategies include, working with medical experts to identify opportunities to use clearer and more consistent terminology related to FCS – especially in defining the clinical profile of the disease and how patients may present in clinical practice. This plays a role in helping reduce the risk of misdiagnosis.

Initiatives that capture the impact of living with FCS, such as testimonials, case studies, and IN-FOCUS, a research study supported by Akcea to understand the impact of FCS from the perspective of the patient, address gaps in educational resources and programs for both healthcare providers (HCPs) and families affected by FCS, facilitate HCP education that makes the diagnosis straightforward and also emphasizes the importance of a diagnosis. To do this, we partner with experts who treat pancreatitis (including pancreatologists, gastroenterologists that focus on the pancreas) as well as those who see lipid disorders, who are typically endocrinologists, cardiologists, or internists, as well as dieticians.

It also connects more people living with FCS with the resources and tools they need to manage their health and access appropriate care and empower patients to take control of their disease and ask questions of their doctors.

PE: When creating a global marketing plan, how do you create an inspiring overall campaign while also tailoring the message for each market and adhering to different agencies’ regulations?

Harper: Again, this begins with listening and observing to what is truly the patient experience and the challenge that we need to address on a global basis. What is true that isn’t affected by borders, policies or healthcare systems? In our case, it is the need for diagnosis, education and connection for people living with FCS, and raising the visibility of the burden of this illness.

Both the generation of these insights, and the development and execution of the campaign work, has to be done in close partnership with our colleagues as the country-teams have been building out. You also need experienced teams who can adapt both strategy and materials across channels – PR, traditional advertising, digital advertising, social media – to comply with local regulatory guidelines and their unique patient and HCP populations.

PE: What role does a strong patient community play in making a rare disease drug successful?

Harper: In many rare diseases, there is a paucity of data. In any disease state, people who live with the disease and their caregivers are the most important resource for insights. In rare diseases, because of the lack of research and data, the patient community is crucial to advance science and learnings. Partnering with the patient community is essential from the earliest stages of drug development for effective clinical trial design and recruitment, regulatory strategy and ultimately commercialization. As with any business, these important decisions need to be market-driven. In rare diseases, patient communities can often provide essential insights.

PE: What are some best practices when working with patient communities?

Harper: One important best practice is to have a dedicated team member who is the lead for patient advocacy. At Akcea, our Executive Director for Patient Advocacy was our sixth hire, and this individual is the primary point of contact for the patient community and the company by partnering across all internal functions from clinical operations to corporate communications. Advocacy organizations should not be dependent on one or just a few industry partners for success.

Having a broad base of support is crucial for long-term survival and independence. In general, industry should emphasize listening, and only partner in areas where there is truly shared mutual interest. Groups need to feel free to communicate with industry partners honestly. In collaborative partnerships, advocates must retain full editorial control.

PE: Decreased access to doctors is something that comes up a lot when we speak with pharmaceutical executives. How has that change shifted the way you market your drug to doctors?

Harper: The onus is on us to have compelling programs and educational resources that work for them. It is our responsibility to generate work that clearly helps improve the quality of care for patients. With FCS, we have done this by producing the first FCS educational materials including a 36-page book co-developed with patients, caregivers and physicians, and endorsed by multiple societies and disease foundations.

Right now, we are in the midst of launching a number of materials to help this community live with the very restrictive diet their disease requires – handouts, website content, fat trackers and multiple videos with patients and registered dieticians including a cooking demonstration. This is in response to what we heard physicians tell us they need: basic, credible and clear materials that make it easier for patients to understand and live with their disease.

PE: In your opinion, what’s the overall number one challenge the pharma industry is facing when it comes to successful marketing of their drugs?

Harper: Market access, reimbursement, and patient support for complex drugs for serious disease have been and will continue to be crucial success factors for commercialization. For marketing, we actually have more channels and data and ways of reaching practitioners and patients than ever before. The challenge I don’t think is new but is more important than every - t takes a lot of time and listening and testing to get to a simple, clear message that resonates with the people we need to reach to improve care and that meets regulatory guidelines. Complicated is easy. Clear is hard, but it is what works.

PE: What’s the best business advice you have ever been given?

Harper: In my first job at a consulting firm, the founder used to say, “It’s another day in business.” The context was that organizational dynamics can often be distracting, but we need to stay focused because we have important work to do. There are few places where that is as true as it is in healthcare.

PE: What do you tell recent grads about how to be successful when it comes to pharma marketing?

Harper: As you pursue roles, seek out opportunities to build strengths in analytics and strategic thinking, and separately, those that emphasize execution. It is not common to find people who can both be strategic and execute well, and it is a differentiator. Recognize that every role, no matter how junior or tactical, is an opportunity to lead and demonstrate impact.

Lastly, it should go without saying that in healthcare it is also essential to keep insights about the patient experience front and center. Take the time to meet them, talk to them and really understand the impact that disease has on their lives – and what needs to change in order for their situation to improve. That understanding will drive you to new levels of innovation and dedication.


Related Videos
Related Content