The global sales of orphan drugs are expected to rise 10.5% annually to approximately $176 billion in 2020, according to EvaluatePharma. That is also one reason why orphan drugs have become such high-valued targets in M&A deals. Of the 18 deals announced since last August, half involved an orphan drug, according to the Wall Street Journal.
All of this means Big Pharma companies will need to learn the best way to market these products, which have some inherent challenges not present with treatments that serve a larger patient population. Starting with: How exactly do you even find physicians and/or patients to target? PM360 asked 10 experts that very question and more, including: When is the best time to involve your target audience? How important is your relationship with patients and advocacy groups? What would you recommend as the best promotional approach to take?
In the orphan/rare disease space, patients, advocates and healthcare professionals are a driving force behind research, education and policies that advance care—they push for answers and seek to address unmet medical needs. Because awareness is often low and clinical advances are not typically discussed in mainstream media, patients must be their own advocates.
As partners in seeking new treatments, pharmaceutical sponsors and marketers have the opportunity to engage, educate and empower at each step from discovery to reimbursement. This means providing updates on clinical development; translating the meaning to patients; providing resources to help key stakeholders learn more about the disease, navigate questions and understand options. Careful thought should also be given to the use of communication channels—ensuring that audiences are engaged in the most accessible way, such as online, live engagements or print. Of course, messaging should be mindful of FDA rules regarding pre-launch and post-launch communications.
Getting Advocacy Involved
Patients can be discouraged by the lack of information or new data, particularly in the early stages of research. Engaging advocacy at the outset means advocates feel like partners. Pharma should encourage their involvement in educating peers and supporting clinical trial design and recruitment—a process that can often stall the entire development cycle.
In the post-market world, patient and physician allies can help to create awareness, provide disease education, and direct others to new treatment. Advocacy organizations may even help to quell misconceptions by lending their credible, third-party voice.
Work closely with patients to identify needs and enable them to play an active role in furthering awareness. It is essential to bring patients and physicians together so their voices and concerns are heard. They have a stake in bringing the disease to the forefront of conversation—and, ultimately, to help bring a potential treatment to patients in need.
Finding physicians to target is like NCIS. It takes a lot of legwork, but when you find the right doctor, you get so excited! To build your initial list of targets consider the following:
• Rare Disease Clinical Network (RDCN): Researchers who study rare disease.
• NORD/Disease Advocacy Group: From NORD, find the disease advocacy group and then look up their medical advisory board and even use their physician locator service.
• Centers of Excellence: Ask clinical investigators who they believe has a CoE, or even ask the patient advocacy group.
• NIH/ORDR: Identify doctors through their GARD info center.
• Clinicaltrials.gov: Check out the researchers on this site.
• PubMed.com: Search for who is publishing materials on rare diseases or submitting case reports. Also check out Medscape for guest editors.
• Claims Data: Identify doctors who are treating this condition by the medical claims they submit. If the rare disease does not have a code, then use the associated symptoms.
• Rx Data: Look into prescription records to see who is prescribing the common drugs that patients take for treatment.
• Genetic Counselors: They are familiar with those who treat rare diseases as they may make referrals. Check with the National Center of Genetic Counselors or the Genetic Testing Registry.
Building Relationships With Patients
One of the most important things you can do is connect with patients and build a community in which they can talk. Rare disease is a very emotional area and patients often feel isolated. “Rare disease moms” will stop at nothing to find the best treatment for their child. Patients turn to advocacy groups for support—and pharma needs to work closely with them for disease education and access to medicines. Pharma can partner with advocacy groups to create educational platforms, such as unbranded websites or social media engagement to expand the group’s reach to patients.
It takes about five years after their first symptom appears for patients living with rare diseases to get diagnosed. This creates an unmatched urgency to recognize and understand rare diseases and to prepare the market for the orphan drugs developed to treat them. A lot of education will be needed. And to do it right, it takes more than just time.
Raising awareness and educating physicians about the impact and mechanisms of rare diseases is a necessity—just consider that nearly two-thirds of them are likely to refer a patient to a specialist to make a diagnosis, and that almost half of the specialists say they would likely consult the literature before making it. For this reason, it’s never too soon to develop your strategy and initiate innovation—both vital for any orphan brand. This is your brand’s double helix.
Promoting Clinical Trials
For a long time, orphan therapies have been novel, first-in-class efforts. Now that we’re seeing competition in orphan drug markets, there’s a greater need to promote information about novel therapeutic platforms and experimental end points earlier. Promoting clinical trials is a great place to start.
During your clinical trials is when you want to find your patient advocates. They are the key to diagnosing future patients. If you’re not finding patients, you’re faltering.
Companies should pioneer strategies to identify patients as a part of their prelaunch initiatives. The more you connect with these rare communities, the faster you will foster awareness. Without a strategy to identify patients, your brand won’t survive; but with one, it can thrive.
Of course you have to evolve with the constraints—nothing about the orphan space is color by numbers—but the earlier you start advancing what your audience knows, the greater your chance of influencing and enhancing your market success.
Pharma has been talking about the need to work with advocacy groups for ages, but for the most part they have yet to effectively or consistently engage with the stakeholder community. Their reluctance is understandable and primarily due to their interpretation of the regulatory/legal restrictions perhaps compounded by government-enforced Corporate Integrity Agreements (CIAs). But given the emergence by many top pharma companies from CIAs and resulting communication modifications that respect the new FDA, pharma now has a real opportunity to build trust with these groups by providing valuable services to rare disease community members.
Good information on a rare disease is hard to find and rare disease communities tend to be incredibly fractured. Pharma has the unique ability and resources to make things happen—in an urgent manner. This is a skillset they can bring to the table to become that valued partner the community is seeking—and they can do it by providing disease awareness and education beginning as early as Phase II. The key is to engage early, earn respect and become a valued member of the community.
Commercializing An Orphan Drug
In rare diseases, the traditional “feet on the street” Big Pharma approach to commercialization won’t work. You can also forget about huge exhibits at medical conferences. Orphan drug commercialization is more intimate. When done well, it is characterized by understanding and serving the emotional needs of your stakeholders (patients, caregivers, advocates, physicians, etc.) as well as satisfying their insatiable quest for information and providing it to them on their terms, in a way they like to receive information and share experiences. For the pharma company, it means becoming a valued partner in the rare disease community through effective stakeholder engagement—this may mean going places and using a new mix of media that pharma has been very hesitant to embrace up to now.
Marketing orphan drugs requires a unique strategic approach to effectively reach targeted professional and patient audiences. Some key considerations:
Condition the market in prelaunch: Before launch, marketing efforts should include unbranded disease education to raise awareness among patients, family members and healthcare professionals.
Include payer marketing: Payer programs should be educational, emphasizing the efficacy and benefits of the drugs and the importance of early diagnosis and adherence to treatment.
Identify physician targets:
- Traditional sales force efforts are ineffective for orphan brands, so new ways of targeting doctors are critical, including digital initiatives as part of multichannel efforts to deliver impactful brand messages.
- Patients may begin their journey at the office of their primary care physician, but visit numerous specialists before receiving a correct diagnosis. Pharma must understand all key stakeholders and their roles in diagnosis and treatment.
- Marketers should work with varying data sets, such as a medical claims analysis, to identify which doctors are treating rare disease patients.
Provide a sense of community for patients. Why? Because patient journeys can be long and frightening, patients may not know anyone suffering from the same condition, and it’s difficult for people with rare diseases to find support and make connections. Creating a sense of community through advocacy, foundations or associations is mission critical. And, because of the scarcity of support, patients tend to welcome information they can trust via a direct relationship with pharma companies.
Marketing orphan drugs is an important and valuable undertaking, requiring understanding and sensitivity to patients and caregivers. Pharma companies that develop impactful and relevant marketing initiatives should include all key stakeholders to maximize success of their brands and the best outcomes possible.
When entering a rare disease space, manufacturers must focus on building strong partnerships with the advocacy community. This is true even very early in the lifecycle, when you’re designing Phase II protocol. Patient and advocacy involvement in drug development starts much earlier and is much more active than in larger disease categories. Ultimately, the patient advocacy community often has the most knowledge and the strongest voice in that category.
It’s critical for any industry partner to have a well thought out, consistent approach and communication platform for these stakeholders—one that demonstrates value and purpose to the community.
Building Patient/Advocacy Partnerships
First, listen and learn—get to know the influencers and spend the time to develop a clear view on the landscape and needs/desires of that particular community.
Then establish a shared agenda with the community—you need to elevate your brand purpose beyond just product. Identify common goals and don’t be afraid to put a stake in the ground regarding your brand and company’s commitment.
Be sure to engage with authenticity—it is important to be human, accessible and maintain open, consistent communication channels while getting senior leadership to demonstrate commitment.
Finally, be sure to deliver on that larger purpose by bringing value to the lives of the patients, families and treaters. Have an impact beyond just your product.
If you do this well, and do it early, it will transform your mindset; how you prioritize your activities and investments; how you organize and structure your staff and resources; and the nature by which you engage the community. This is what we call “start and stay with purpose.”
A patient-centric marketing approach is essential for all medicines, but this is particularly true for orphan drugs. Effective marketers need to gather a deep and thorough understanding of the patient’s journey, including where and how patients are presenting, patient’s beliefs and behaviors, and where within the patient journey a new modality would provide the best outcome.
An additional approach to achieve patient centricity is through a clear and thorough medical affairs strategy. Researching existing treatment guidelines and/or anecdotal case reports of on- or off-label drug use in these patients can help to identify Key Opinion Leaders (KOLs) in rare disease areas. These opinion leaders can, in turn, help biopharma companies understand physician networks, referral patterns and educational strategies.
Providing Services to Physicians and Patients
The nature of rare diseases means that the patient population is small, and the HCP audience is limited. Educational approaches for physicians should offer widely dispersed HCPs an appropriate combination of face-to-face and virtual interactions, with access to Medical Science Liaisons (MSLs) or other highly specialized resources as needed. Biopharma companies should also be prepared to provide digital content to HCPs when a rare disease patient presents, allowing for live virtual connections to address questions.
In addition, many orphan drugs are less accessible, with biopharma companies often using specialty pharmacies or distributing orphan drugs directly. As a result, critical success factors include rapidly connecting HCPs and patients to these access models and offering assistance in navigating reimbursement. These approaches may differ from those used for therapies distributed through the traditional supply chain.
Once the patient is on medication, various strategies may also be needed to support adherence and optimize the quality of care. Remote engagement may be helpful, with nurse educators and other touch points to help facilitate delivery of healthcare in partnership with HCPs.
The first challenge that orphan drug companies often face lies in finding patients. That’s why it’s best to begin building awareness of the condition as soon as possible. Providing disease education information for both patients and healthcare professionals on websites or social media and supporting the community in coming together—especially when patient organizations are lacking—will help more patients get diagnosed, raise awareness about the burden of the disease which your treatment will one day alleviate, and empower patients to advocate for their right to healthy lives.
Patients living with more common diseases often perceive pharmaceutical companies as large, faceless organizations that supply their treatment. But in rare diseases, patients and caregivers think of each company as part of their community. They’ll expect you to feel the same sense of urgency that they feel about getting treatments to market—they’ll also want and need to stay informed.
Good relationships are essential in this close-knit world. Having a company-wide commitment to empathy, transparency, respect and trust will help you earn reputation capital. And you really can’t afford to jeopardize it.
The need to grow awareness and build relationships continues even after you launch the product, but this is also a time when you have to focus on adherence. Rare disease patients have challenging lives and orphan drugs often involve complex treatment protocols and expensive co-pays. Offering personalized support will be key to your success. Anything you can do to make the lives of patients and caregivers easier, whether it’s by providing insurance support or creating an efficient and effective supply chain, will pay big dividends.
Historically, traditional pharma companies have built their strategies, processes and technologies primarily in stakeholder silos. As today’s world of focused therapeutics continues to emerge, orphan disease organizations are building around a more cohesive care continuum that addresses the wider variety of critical stakeholders in the ecosystem.
Commercialization and promotional approaches are also being designed in a more agile way to cater to each stakeholder’s needs. Pharma companies now require sales and marketing execution that operates at the intersection of each key stakeholder—instead of for a specific stakeholder mindset.
While the fundamentals are similar to those that exist to address a more retail-oriented, large population model, orphan drug promotion must truly put the patient at the center of the ecosystem. Patients and caregivers typically play a significant role in achieving a proper diagnosis and making therapeutic choices—even with limited options available.
Understand the Patient Journey
Development of the patient “journey” is imperative. Pharma companies must understand referral patterns, diagnostics and alternative therapies used during treatment, as well as the way patients and their caregivers live their everyday lives and the challenges their disease imposes. Companies must also understand each point in the journey to develop an appropriate model of patient support services.
This level of fundamental understanding will better drive marketers and sales teams to educate and promote at the right place and right time. This is a typical challenge for orphan sales teams who often cannot provide coverage as broadly as that of a more retail-focused sales team.
Success relies on the ability to identify known and unknown patients and orchestrate the variety of promotional, educational and patient service efforts at the pharma company’s disposal. Bringing the best overall strategy in a coordinated fashion to both patients and also the range of key stakeholders will drive brand success.
In general, a community is driven by a shared interest or location. Thriving communities can develop in neighborhoods around interests, whether it’s people who are passionate about their town, or people who retain a fascination with a particular brand and model of car.
Communities remain active because the individuals they are comprised of value the benefits they offer: Shared information, fitting in, and a sense of empowerment in common pursuits. Smaller communities tend to be more active than larger communities since it’s easier to make personal connections with other members.
Health communities are built around a specific condition and driven by people who have that shared experience. The condition experience is imposed on the members, and social media posts to the community are typically driven by a change in health or wellbeing—whether it’s a positive or negative one.
Becoming a Part of Rare Disease Communities
Rare disease communities fit the criteria for strong communities. The overall size of the group is relatively small and is passionate about their condition, hungry for information, and willing to share. Rare disease communities are supportive in nature and show a preference towards longer forms of communication.
As marketers we can recognize the desire for information as well as the community’s willingness to share. Information is shared most often in the form of describing a personal experience. We can recognize the importance of creating an appropriate, empathetic tone when reaching out to rare disease patients. Most importantly, we can offer value in our communications, which will create an opportunity for our communications to be adopted and become part of the information sharing structures rare disease communities have in place.