A recent Deloitte Insights white paper found that three product characteristics are strongly associated with strong launch performance: priority review and specialty or orphan drug status – all of which are typically associated with drugs targeting rare diseases. Of course, these are not the only characteristics that define a strong performance, so what else is it that differentiates these launches and sets them up for success?

In the field of rare diseases, it’s common to come across statements that emphasise how launching a rare disease medicine demands a different launch model from that of traditional drugs. There’s no doubt that drug launches targeting rare diseases have distinct characteristics from those of more mainstream drugs, but in some ways their chance of success is more assured (since they target diseases that typically have limited treatment options). Furthermore, a priority review designation by the FDA or EMA signals to the market an expectation that the drug will provide an improvement over currently available treatment.

However, a rare disease product launch faces its own set of challenges – with increased competition and pricing pressures, regulators demanding more evidence of therapeutic value, and patients themselves driving treatment decisions and impacting success through globally connected communities. Despite these challenges, rare disease products are increasingly setting the gold standard for launch success – so are there any lessons we can learn (from these frequently smaller pharmaceutical companies) or any best practices that we can carry over to more mainstream drug launches?

While each rare disease launch is different, several similarities are shared – a key element being a truly patient-centric, cross-functional approach. One that genuinely puts patients at the heart of all communications by addressing their needs. As with all products, a holistic, long-term launch strategy is required, with life cycle management and scenario planning. However, during our work with pharmaceutical companies launching products in rare diseases, one key characteristic stands out. Collaboration – a cross-functional approach to launch execution that allows all stakeholders to input, not only through the sharing of insights and development of collaborative tools, but through a true alignment and consistency of messages across audiences and regions.

As with any drug launch, a comprehensive understanding of the patient and physician’s journey and ecosystem is required. Journey maps are essential to build understanding of the challenges and opportunities facing the patient or prescriber throughout a disease course. These maps not only help us understand the practical aspects, but they provide insights into the needs and emotions each stakeholder experiences – allowing us to identify treatment decision drivers and pain points. Furthermore, a thorough mapping of the ecosystem allows us to form a detailed picture of the different stakeholders involved (including the role of patient advisory groups [PAGs], caregivers, primary care physicians, specialist physicians, nurses, payers and policymakers), as well as the influencers of these stakeholders. A full understanding of all stakeholders and their individual journeys also ensures interactions can be tailored to meet their needs at each touchpoint.

Companies focusing on rare diseases must be patient-centric. Small and highly connected patient communities and limited general disease awareness mean that patient and PAG involvement is critical to success. In the rare disease setting, information exchange and best practice is therefore typically driven by the patient community and with an increasingly digital world of interconnected patients this is a trend we’re likely to see duplicated in other disease areas. Consequently, if we’re considering best practice, shouldn’t strong relationships with patients, PAGs and other stakeholders be the gold standard we aim for in all diseases?
When developing a drug targeting a rare disease, patients/PAGS are frequently involved in trial design, with companies seeking their input through advisory boards to ensure the trial is clinically relevant, patient-centric and fulfils regulatory requirements. This is clearly an important factor in a setting where endpoints may be disputed, or biomarkers not validated – but it’s important to ensure an understanding of patients’ needs for all clinical trials. With an increasing number of drug trials in crowded markets such as oncology (for example, in non-small cell lung cancer and multiple myeloma), understanding patient needs is not only critical to ensure a good trial participant experience but can also help drive recruitment.

Rare diseases are often genetic, disproportionately affect children, and carry a high burden to patients and their families/caregivers. In these situations, ‘beyond the pill’ services can provide significant support to alleviate some of this burden. To be considered truly patient-centric, pharmaceutical companies must go beyond producing and selling drugs by addressing additional aspects relevant to the patient experience. Holistic services can add value in multiple ways, including support with access and reimbursement, nursing services (eg, supporting drug administration and adverse event management), 24/7 helplines and through patient education. Many companies are already implementing such approaches, with digital health technology at the forefront (including development of wearable devices and apps), and a focus on working in partnership with patient representatives to make a real difference to patients’ lives (for example, Boehringer Ingelheim in the development of blister pack and medication bottle opening tools for patients with scleroderma) – but these initial efforts need to go further.

For many therapies (from chronic diseases to paediatric rare diseases and oncology), patient adherence and persistence with treatment can be challenging. Depending on the indication, up to half of patients may be non-adherent, with perceptual, psychological and practical barriers all contributing. Non-adherence is associated with poor clinical outcomes and higher healthcare costs, so it’s in our interest to support patients to properly manage their treatment. Several strategies exist to enhance adherence, including education and provision of personalised information, simplification of treatment regimens or patient reminders (such as patient diaries and apps) and psychosocial support (including family and peer support and mental health resources).

Pricing and market access are evolving, with several trends emerging, including earlier and broad dialogue with regulatory bodies, payers and healthcare policymakers. Establishing early partnerships with these stakeholders can increase the likelihood of product approval and provide a strong basis for later discussions regarding pricing and reimbursement. Furthermore, an increased awareness of regulatory demands from the start can improve product life cycle planning and ensure the relevant data is collected within clinical trials.

For novel therapies (including medicines aimed at rare diseases as well as gene or cell therapies) payers are increasingly requiring a strong evidence base over a longer time period. Real-world evidence collection is now standard practice, starting early and often continuing to product maturity. Ultimately, a value strategy needs to reflect benefits for the entire healthcare system, with evidence meeting the needs of patients, the pharmaceutical industry, and regulatory and health economics bodies. It is important to demonstrate not only why your product should access the healthcare budget, but to also consider the impact on the overall care pathway, enabling providers to manage the total cost of care, the timing of budgetary impacts and understand any potential budget savings.

Building trust, understanding and aligning with payers and the community (including PAGs) ensures that value is demonstrated, with a clear value proposition articulated across customer types and segments.

Proactive and transparent communication is essential for all stakeholders. Consistency of message is critical, as is personalisation of information. In an increasingly digital age, multichannel marketing is required, with a focus on education and patient-centricity, rather than promotion. An effective multichannel approach necessitates a deep understanding of the target stakeholders, but it allows for optimal engagement and can make the difference between failure and success.

So, can lessons be learned from the rare disease setting? Ultimately the key to success in both rare and non-rare diseases is a patient-centric, cross-functional approach. In practice this involves collaboration, not only internally, but with HCPs (including multidisciplinary teams), patients/caregivers, PAGs, regulatory authorities, healthcare policymakers and payers. By taking this route we should achieve a more focused and efficient launch that will benefit both companies and patients alike.

Amanda Henkel is Strategy Director at Nucleus Global Amanda.Henkel@nucleuscentral.com