A strong market access strategy aims to effectively convey an evidence-based value story to payers, ensuring rare disease patients have access to essential treatments. However, generating solid evidence can be challenging. By engaging directly with patients, pharma and biotech companies can gather crucial data that strengthens the evidence supporting their market access efforts.

To learn more about the importance of co-creating successful market access strategies for rare disease treatments, we spoke with Annabel de Maria, Alira Health’s Chief Patient Officer, and Pepi Hurtado-Lopez, Managing Partner, Market Access and Pricing.

What are the major challenges that companies developing rare disease treatments face at the price and reimbursement stage?

Pepi: One challenge in rare diseases, with high unmet needs and a small patient population, is a lack of statistical significance in the clinical trial data. Another challenge is the evidence gaps related to a lack of understanding of the patient journey and patient needs. After an understandable focus on generating data to meet the requirements of regulators, companies may find that they need additional data for price and reimbursement negotiations. Payers may say that they can’t make a final decision on a product because the evidence is insufficient and they need a better understanding of the benefit the treatment provides.

How can companies fill the evidence gap?

Annabel: Companies can address these evidence challenges by learning how to work with patients from the very beginning, in the preclinical stage if possible. They can work with patients in two ways: to understand the type of evidence that they should generate, and to generate this evidence.

Pepi: In rare diseases, patient value stories help payers to better understand how a product benefits patients. Patients are just patients when they are in front of a doctor. But in front of some payers in certain HTAs, patients can appear as experts. These payers might rely on that expertise to make decisions.

Annabel: Of course, there are other ways to close evidence gaps, but we believe that working with patients is the most effective way, especially in rare diseases where patients know more about their disease than most doctors.

How can companies work with patients to inform their market access strategy and overcome the evidence gap challenge?

Annabel: You can use quantitative market research when you work with patients to understand the answers to key questions. And you can conduct qualitative market research, which will delve into the nuances of the emotional journey for the patient, what it is to live with a disease, and a better understanding of any unmet needs. Then you can convert that assessment into strategic thinking for the value dossier. Payers can find this input powerful and insightful.

Based on our experience interacting with payers, we believe that they welcome additional input from patients about rare diseases. The qualitative information can help payers interpret the quantitative data and gives them a clearer picture of the disease.

How should companies change the way that their market access function operates in order to benefit from the collaboration with patients?

Pepi: The market access function should not focus only on the product because payers don’t put a product in the market, they provide patient access to the product. So companies should set patient access as the goal, and make it central to the entire company from the very early stages of product development. Market access should involve the three main stakeholders: the patients, clinicians, and the payers. Their insights and input can inform business decisions at every stage and make early scientific conversations with both the regulators and the HTA bodies more successful. By structuring the market access function from the beginning with this in mind, companies can ensure that every stage of the product lifecycle goes more smoothly.

What are the best practices for companies focused in rare diseases to close evidence gaps by working with patients?

Pepi: First, patients and caregivers can provide insight into the design of clinical trials to generate the evidence they believe is important. Their input may help inform secondary end points and/or exploratory endpoints that are usually related to patient reported outcomes. Second, companies need to understand the patient journey and the relationship they have with clinicians to capture how the patient influences prescription behavior. Third is the emotional journey, from which companies can create value messages that show the value of the product in clinical practice and complement the hard data in the value dossier. This helps the payers to interpret the disease burden and better understand the unmet needs.

At Alira Health, our experienced Patient Engagement and Market Access teams work collaboratively to ensure that companies working in rare diseases are co-creating their market access strategies with patients from the earliest stages of product development.