Orphan drugs are different than mass-market therapies – and your commercial strategy should be, too

, , , , , ,

By Dan Schulman
Associate Partner
Beghou Consulting

When patients with a rare disease fail to receive a proper diagnosis, treatment plan, and therapy, they can suffer for years. Failed treatments for these prolonged conditions strain patients, caregivers, and the health ecosystem. In addition to the significant physical and emotional struggles these patients endure, delayed diagnoses for rare diseases can also result in many years of lost revenue for manufacturers.

By definition, a rare disease is a condition that affects fewer than 200,000 people in the United States – and the majority of these are considered serious or life-threatening. Rare diseases are not only rare, but also inherently complex with complicated underlying causes, unusual symptoms, alternative progressions – and not a lot of patient data. That’s why these therapies take so long to develop; in fact, orphan drugs take 18 percent longer, on average, from patent filing to product launch compared to other new therapies. Further, life sciences companies must overcome vastly different challenges to bring orphan drugs to market than mass-market therapies that treat diabetes or heart disease. Despite significant progress and innovation in recent years, 95 percent of rare diseases have no FDA-approved treatment.

The biggest challenge for these orphan drug manufacturers is finding and tracking patients. That’s why life sciences companies must work overtime to 1) develop a holistic perspective of the patient journey; 2) engage patients throughout research, development and commercialization of the product; and 3) hire specialized sales forces to introduce their product to the most relevant physicians and ensure patients receive proper support while on the therapy.

Find and embrace ‘needles in the haystack’

Without specific diagnosis codes to define these conditions and provide relevant medical information – including clinical care, research and disease patterns – orphan drug manufacturers can face an uphill battle as they bring their therapy to market. This lack of data and information is further exacerbated if a company seeks to launch a first-in-class therapy. To form an effective go-to-market strategy, commercial teams should use advanced ensemble modeling and marketing research to identify patients who would benefit most from the therapy and the physicians who treat these patients.

To find these “needles in the haystack,” orphan drug manufacturers can combine relevant quantitative information – such as claims data and electronic health records – with previously conducted research, such as literature reviews of disease areas, patient blogs, comments and product reviews. Fortunately, recent developments in machine learning enable manufacturers to more quickly sift through and analyze terabytes of data and information to identify relevant patients and their physicians. Beyond patient identification, this translates into more accurate forecasts, better defined territories and more realistic goals for the sales force.

The patient journey often begins with symptoms of the disease, which prompts patients to seek medical attention from various health care providers. These appointments typically lead to a diagnosis, an initial round of treatments and then additional therapies to better treat remaining symptoms and underlying disease. The commercial team can use marketing research to gather more information about these patients and how they receive their medication. Marketing research can include interviews with the patient, as well as others involved in the patient journey, including caregivers, nurses and physicians. The commercial team can also gather information from other stakeholders, such as payers for information on therapy access, pharmacists on the disbursement channel (specialty pharmacy or retail pharmacy) and their impact on patients’ ability to access the prescribed medicine.

The commercial team can use these findings to map a patient’s journey to diagnosis and effective treatment, identify opportunities and barriers in better serving the patients’ needs, as well as train predictive models to identify additional patients who could benefit from treatment and their prescribing physicians.

Maximize customer engagement

Orphan drug sales forces are more like Army Rangers than infantry. They are few in number – between 10 and 100 – and must specialize in selling the therapy. Most companies will need to recruit from known networks of reps who work in related therapies. Because these sales forces are small, commercial teams must be sure to design their sales forces to maximize productivity. These teams should focus on territories with recognized medical centers that treat the rare disease, maximize sales force engagement with customers most likely to prescribe the therapy and design territories to optimize travel time across states – that is, prioritize medical centers near airports and other mass-transit hubs. Commercial teams should identify these high-potential customers and deliver tailored messages and marketing collateral based on real-world evidence to support the therapy’s necessity and justify payer reimbursement. In turn, payers must thoroughly understand the benefits and value of each drug.

When possible, sales reps should introduce their additional support colleagues to physicians. These nurse educators and patient support coordinators can help physicians and patients gain access to the therapy, better understand side effects, and ensure compliance. These educators can also answer any questions that patients may have about the therapy or reimbursement, as well as navigate necessary approvals to dispense it.

Put patients first
To help physicians diagnose and prescribe more quickly, orphan drug sales forces should educate physicians and specialists about common shortcomings and misdiagnoses related to the disease. Disease awareness programs hosted by manufacturers and specialists can help physicians proactively identify and treat these rare diseases earlier in the patient journey.

Of course, given their complexity and small patient population, orphan drugs can come with higher costs. The overall cost of treatment, however, is often lower than what these patients would require without these therapies. Without regular dosage, some patients may require extended hospitalization and more frequent emergency care, including transplants, transfusions and surgeries. Taken together, all of these have the potential to further strain health care resources and contribute to excess costs. For example, an effective oral medication administered during outpatient care enables these patients to spend more time at home and avoid mounting costs of extended hospital stays. In this way, orphan drugs can help patients feel more in control of their treatment program and serve as a bridge to a potentially curative treatment.

As life sciences companies continue to develop orphan drugs, commercial teams should be sure to study the patient journey, involve patients in product development and help their sales forces, nurse educators and patient support teams serve as resources to their customers. Looking ahead, orphan drug manufacturers will invest in even more targeted therapies to treat even smaller subsets of patients based on genetic testing, therapy resistance and additional characteristics. Life sciences companies must continue to challenge the status quo, share their innovations and research, and brainstorm creative ways to identify patients who need these therapies most. With a thoughtful commercial strategy, they’ll succeed in helping these life-saving treatments reach patients as quickly as possible and play a critical role in the journey toward a cure.


About the Author

Dan Schulman is an associate partner at Beghou Consulting. An industry veteran with more than 15 years of experience, he has extensive experience working with clients on incentive compensation plan design and management, sales force size and structure, business intelligence reporting and data management. Dan holds bachelor’s degrees in both sociology and mathematical methods in the social sciences from Northwestern University. He leads the firm’s San Francisco office and can be reached at [email protected].