In the past, reimbursement wasn't a consideration during protocol design, but that must now change.

By adopting a focus on on the needs of the patient, industry can evolve their trial designs and processes to pre-empt market access issues further down the line, and incorporate the patient voice into in all phases of research and commercialisation.

The culture of clinical research is starting to shift from one directed by researchers to one driven by the needs of patients. Digital technology that constantly monitors and communicates with patients in real time makes it possible to assess the results of therapies at a granular level and over a range of real-world conditions. Involving patients and care partners in developing new therapies and trial designs, and meaningfully engaging with them throughout the development and post-market process, helps to generate irrefutable evidence of the value of therapies to patients in the real world.

Real world evidence (RWE) can estimate the effectiveness of a therapy in actual clinical practice, reflecting a larger population and longer-term outcomes. As clearer direction from stakeholders is released, confirmed and put into practice, there is major potential in the implementation of RWE generation strategies. Devising a comprehensive RWE strategy can ensure that smart decisions are made in how to best choose, synthesise, and analyse available assets. By identifying what evidence will support regulators’ and payers’ decision-making, sponsors can develop an evidence generation plan across the product life cycle to leverage outputs and identify data gaps.

However, the use of digital technology to collect patient-reported data may not be right for every study or every patient. It is therefore important to analyse the patient population and consider factors such as patient age, health literacy, and socioeconomics. It is also imperative to consider what data is being collected and the rationale behind it.

At ICON, we have a process for working closely with our clinical trials teams at an early stage to make sure that the protocols meet the needs of HTAs and payers. Contact us to speak to one of our experts.


This blog is an edited version of “Improving Access and Reimbursement for Specialty Therapies” which appeared in the May 2020 edition of PharmaPhorum Magazine. To view the full article, please visit