The Importance of Stakeholder Research in Rare Disease Drug Development

The approval of novel orphan drug designations continues to grow, while many existing rare disease therapies are receiving approval for expanded indications. With this increase Rare Disease Drug Developmentand broadening class of products, including some that target the same mutation or molecular defect, sponsors face new and significant market access challenges in securing reimbursement.

Leading manufacturers increasingly employ stakeholder research early in development to better identify the needs of patients and providers. This strategy can build in compelling asset value during development, help avoid pitfalls and better inform go/no-go decisions earlier to avoid costly development delays or even dead-ends.

Truly Meeting Unmet Needs?

Rare disease drug development already faces multiple hurdles: disease understanding, study design creation and perhaps most importantly, access to limited patient populations. Strategically incorporating stakeholder research at the earliest stages of development can help address one of the most basic questions from the start: does it make economic sense to continue to invest in the development of this orphan drug?

Answering this question requires an examination of the current and future treatment landscape to understand a number of key factors. These factors include the needs of the limited patient population, the efficacy of any existing or anticipated future treatments, as well as market reaction to introduction of a new product.

Stakeholder research enables sponsors to “fill in the blanks,” calculate the potential risks earlier in their development process and make more informed go/no-go decisions, before investing significant resources along the orphan drug pathway to market.

Uncovering the Perspectives of Payers and Prescribers

Even if a product is deemed safe and effective, sponsors must consider how it is positioned against competing products to avoid ending up on the “me-to” product shelf. As more orphan drug applications receive approval, preference for one drug at the expense of another becomes a real commercial risk for sponsors. An example of this is currently unfolding with competing immunotherapies targeting the same type of rare lung cancer.

Identifying a commercially viable market niche is more likely as a result of defining the comparator for a clinical trial through stakeholder research. This valuable exercise can help position a product with meaningful differentiators so that it is more likely to achieve adequate reimbursement and a stronger market position.

Even if an orphan drug makes it to market, payers may reject a treatment if they deem the cost-effectiveness unacceptable, or if there is limited clinical evidence or comparative efficacy for the treatment. Sponsors face increasing scrutiny and pressure to justify their pricing, underscoring the need to account for possible pricing scenarios in both their development and market access strategies. Stakeholder research can bring these considerations to the forefront earlier in development, giving sponsors time to prepare compelling communications to payers and prescribers to justify the product’s value proposition.

Including the Right Stakeholders

Depending on the indication, stakeholder research usually encompasses a mix of perspectives including those from key opinion leaders, clinicians and prescribers, health economists, hospital payers, third-party payers and even health technology assessors.

As the voice of the patient, Patient Advocacy Groups are also significant stakeholders in rare disease drug development. Inclusion of this stakeholder group’s perspectives helps to incorporate patient-centered health outcomes into clinical research and strengthens engagement between patients, families, caregivers and drug developers.

With the right mix and balance of stakeholders from multiple groups, valuable real-world data can be gathered to provide critical insights including:

  • Policies/guidelines that affect pricing justification and reimbursement
  • Needs for companion diagnostic tests, and whether diagnostic testing confirmation is likely to be required by payers
  • Expected efficacy, possible therapy combinations and labeling strategies

While there is no effective standard of care with many rare diseases, stakeholders often are a source of valuable, if not critical insights into an improved understanding the disease, relevant biomarkers and clinical endpoints.

Applying Research to Maximize Commercial Potential

As stakeholder research helps to reveal key decision points and the development pathway, it is crucial that market access and drug development teams work together early in development.  One of the key outputs of this early, joint team’s efforts is the establishment and inclusion of strategic market recommendations in the clinical profile and prospective trial design. Working together with key stakeholders, sponsors can leverage real-world data to highlight the disease’s burden on patients, describe the product’s unique features to payers and better position the product to reach patients in need.