While pharma companies engage with both healthcare professionals and patient groups to understand patients’ needs, they mostly speak to healthcare professionals, limiting their understanding of the outcomes that matter most to patients. In recent years, there has been a trend towards patient centricity, which might seem like a no brainer, but patient input is still not routinely sought, and efforts often do not go beyond grand statements. A genuinely patient-centric approach would transform patient insights into actionable outcomes.

Cell and gene therapy delivery is particularly complex and requires a deep understanding of patients’ needs

With personalized therapies like cell and gene therapies (CGTs), the patient journey is fundamentally different than traditional medicines. To successfully deliver CGTs, patients’ needs must be well understood and addressed. Most CGTs are for rare, often misunderstood, diseases. In addition, patients take on significant risk, for example, having one gene therapy often means they will never be able to have another. Patients also commit time and money, often travelling long distances including across borders to specialist treatment centres. As for payers, evidence that meaningful and sustainable outcomes is achieved for patients is very much top of mind given CGT costs extend into the millions of dollars per patient.

‘What really distinguishes treatments such as chimeric antigen receptor T cell therapies is that the patient is not just the recipient of a drug product. The patient is the product,’ explains Marc Boutin, Global Head of Patient Engagement at Novartis. ‘Patients are truly central to each and every activity as the patient’s own cells are the starting material for all manufacturing processed and, after genetic modification, form the essence of the final material.’

‘Beyond-the-pill’ programs have been more successful when patients have been involved in their design

In cases where patient communities have not been consulted on ‘beyond-the-pill’ for CGT – that is the initiatives and services to improve access to therapy – there have been difficulties. In early 2020, Novartis launched a global managed access program to make its gene therapy Zolgensma available in countries where it had not received regulatory approval. Novartis failed to engage properly with the SMA patient advocacy community on the design of the program, and was subsequently criticized in the media, by patient groups and EU health ministers for exercising an unfair ‘health lottery’.

Novartis has had more success when it has sought patient input. While devising a plan to implement widespread newborn screening for SMA, Novartis closely collaborated with rare disease advocacy organizations to better understand patients’ needs and expectations. Its efforts have helped to bring newborn screening online across North America and Europe.

A recently launched documentary project by BioMarin looks particularly promising – production teams will travel to the hometowns of hemophilia patients to learn about the realities of living with a bleeding disorder. The relationships and understanding of patients’ experiences should facilitate the development of pressure-tested and meaningful beyond-the-pill initiatives, supporting the expected launch of BioMarin’s gene therapy ValRox for hemophilia.

Patients’ insights should be systematically internalized for patient-driven decision-making

CGT companies leading the way are prioritizing the incorporation of patients’ insights into drug discovery, development, and crucially, delivery. Astellas, which is developing multiple CGT platforms, is one to watch – it follows a patient focused medicine development (PFMD) approach, but adds an extra D, for delivery. In this way, Astellas involves patients in the delivery stage, therefore implementing services that address real patient challenges.

Patients’ perspectives should not be sought simply during ‘one off’ consultations, but should be formally internalized via channels such as patient advisory boards. Systematically incorporating patients’ voices into all design, processes and decisions should facilitate effective CGT delivery, taking a step towards facilitating access to these notoriously inaccessible therapies.