To deliver treatments for rare diseases more quickly, companies share how they are adapting their launch strategies and engaging more deeply with physicians and experts.
“The only thing that was impossible was to do nothing.” These are the words of Terry Pirovolakis, CEO of Elpida Therapeutics, and father to Michael who was diagnosed as an infant in 2019 with an ultra-rare neurological condition, SPG50. Despite being told there was no cure, Terry moved mountains over four years to find a breakthrough gene therapy that his son and other affected children all over the world could benefit from.
Michael, Terry, and their family are not alone in facing a long, daunting journey to access life-enhancing therapy for a disease with no cure. In the European Union, less than five percent of rare diseases have at least one approved treatment.[1] It can take up to five years for adults to receive a diagnosis, and half of patients will receive a misdiagnosis first.
By 2030, the goal is to support the development of 1,000 new therapies in Europe.[2] However, rare diseases challenge traditional ways of doing business. Once a medicine is approved, organizations must launch and scale quickly, often across multiple markets. Field medical teams must keep track of complex science at a time when the volume of medical knowledge is doubling every 73 days.[3] They need to identify, engage, and provide education to relevant healthcare professionals (HCPs) and experts even though they’re harder to reach via traditional methods (Veeva Pulse data shows that in-person meetings with physicians have declined in Europe in the last 12 months).
Despite these difficulties, life sciences organizations are improving how their teams organize market access and educate prescribing doctors on new medicines. Companies with comprehensive, accurate healthcare ecosystem data and insights relating to medical experts can prepare the market pre-launch and hit the ground running to accelerate execution after launch. This helps all customer-facing teams to deliver a consistently high-value scientific exchange in which every interaction builds off the last.
Greater agility during launch
Emerging biopharmas play a key role in delivering treatments for affected patients. Of the 192 orphan medicinal products (OMPs) authorized by the European Medicines Agency (EMA) between 2010 and 2022, one in ten successful applicants was designated an ‘SME’.[4] Facing more resource constraints than their competitors (which may have sales organizations multiple times their size), early-stage companies are increasingly seeking the best customer reference data and key opinion leader (KOL) insights to become more nimble when launching new treatments.
Rich information on experts, available in CRM, supported Sweden-headquartered Sobi to launch the first and only medicine for hemophagocytic lymphohistiocytosis (HLH) — a rare, hyperinflammatory condition affecting one in 50,000 resulting in a two-month life expectancy. In the U.S., MSLs at the company now routinely use real-time intelligence to find the right KOLs, understand their interests, priorities, and activities, and consolidate scientific information and updates. “90% of MSLs found new insights for their next engagements, which is critical for a rare disease company with lean teams,” says Rich Palizzolo, executive director of CX and advanced commercial capabilities at Sobi.
An added complexity is that rare diseases often involve several specialties. MSL teams have to get up to speed quickly on multiple areas before meeting experts — while also being responsible for other (more mainstream) therapeutic areas. ADVANZ PHARMA, which focuses on specialty, hospital, and rare disease medicine, found having scientific resources and activity data in one place helped MSLs use their time efficiently. Head of CRM and Digital Solutions for Global Commercial Excellence, Andy Eeckhout, notes: “Our customer-facing teams need to be agile communicators and effectively switch to a more patient-oriented, in-depth scientific discussion than with generics. Pre-call planning is crucial for MSLs before and after launch. The more data they can find, including on past interactions, the better.”
Other companies use customer reference and patient data to improve operational agility as they launch and scale their first products across Europe. For example, one late-stage biotech leveraged its data on the healthcare ecosystem to get a head start on launch by identifying market access roles in Spain, Benelux, and the Nordic countries.
One voice to the physician
After identifying the right experts, teams can engage them more effectively by ensuring that each HCP interaction builds off the previous one. However, sales, marketing, and medical teams often use disconnected technology. As a result, 65% of HCP engagements are not synchronized.
When these teams are connected in the same system handovers are smoother, and HCPs can find answers quickly or connect with MSLs if needed. ADVANZ PHARMA introduced a pre-launch module in its CRM to help market access, medical, and commercial teams share information compliantly. Eeckhout explains: “Physicians need a direct line to the industry, so they know who to contact when they have questions. Medical and commercial teams need to talk to each other and remain agile across customer conversations.”
ANI Pharmaceuticals, which delivers treatments for certain rare autoimmune and inflammatory conditions, only had 75 days to commercialize following swifter-than-expected regulatory approval. By using an industry-specific CRM with master data management, it consolidated its view of each HCP to include interactions with medical and sales reps. “Having this information accessible within the CRM system facilitates more thoughtful and helpful conversations with providers, as well as sales teams’ success and high click-through email rates,” explains Bob Acropolis, executive director of operations and analytics at ANI.
The foundation of successful interactions is accurate reference data — on physicians, healthcare organizations (HCOs), or affiliations. If applied across functions as part of a complete life sciences-specific CRM, it helps companies speak with one voice. In most cases, data change requests (DCRs) can now be made (and resolved) in hours, so reps and field medical don’t duplicate attempts to modify account information and instead work from the same database. With greater trust and confidence in reference data, teams save time so they can focus on high-value scientific exchange.
Sharing medical content that engages
Europe’s fragmented regulatory landscape and evolving local requirements intensify the pressure on marketing teams: they must provide highly personalized, compliant medical content that field teams can share at scale with scientific experts and physicians. With a single view across the entire content lifecycle, biopharmas can streamline and speed up medical, legal, and regulatory (MLR) reviews.
To deliver highly personalized content across a large rare disease portfolio, marketing teams need clarity on which content to recommend, and when. One global biopharma uses data analytics on its global repository of promotional and medical engagement tools to support content use across 17 areas of focus (and a growing global footprint). As its head of marketing and customer engagement noted, “How we engage with HCPs is critical. We need to know what percentage of our content is being developed and relevant to support different HCPs, whose patients rely on them for their rare disease diagnosis and management.”
As a new generation of digitally-savvy HCPs comes through, companies are considering the most effective tactics to engage them. More scientifically active than their peers and four times more likely to adopt a new treatment, younger HCPs require a different mix of scientific channels and content. They seek medical insights to inform their clinical and commercial decisions, which requires close coordination between medical affairs and field medical. “Gone are the days when medical could just focus on the top-tier scientific thought leaders. The range of stakeholders has broadened, and it’s imperative to expand our engagement strategies beyond traditional experts,” says Angela Smart, director of global medical excellence and operations at ADVANZ PHARMA.
Defying the odds, then beating them
Scientific discovery continues to bring hope to patients affected by rare diseases and their families. Life-changing conditions will eventually become chronic illnesses, thanks to the efforts of organizations willing to launch in a high-risk commercial environment. Companies ranging from emerging biotechs to global biopharma are using high-quality customer reference data, deep data on scientific experts, and connected technology to identify, engage, and provide medical education to the most relevant HCPs and KOLs.
Every rare disease patient faces a daunting journey. When Terry Pirovolakis’ son, Michael, was diagnosed, his family was told he was the only child in Canada with SPG50. Life sciences will do its part to help patients defy overwhelming odds — and eventually beat them.
By Chris Moore, President, Veeva Systems Europe
References
[1] “The building blocks to make rare disease treatments more common,” European Commission, February 2022
[2] “What is Rare Disease,” EURODIS, 2024
[3] “Challenges and Opportunities Facing Medical Education,” American Clinical and Climatological Association, 2011
[4] “Trends in orphan medicinal products approvals in the European Union between 2010–2022,” Orphanet Journal of Rare Diseases, 2024
