• 8Jun

    Patient centricity makes ethical and commercial sense

    Patient centricity is no longer a buzzword, it’s an essential way of conducting business that makes both ethical and commercial sense.

    That’s according to Jodi Wolff, head of patient advocacy in the US for Santhera – a company that attributes its success in developing drugs for rare diseases with high unmet needs to its pioneering engagement programmes. Jodi talked to pharmaphorum about the value of really listening to the patient voice during drug development, and how engagement is about meeting needs, not making money.


    She said: “Patient centricity isn’t just a buzzword any more, it’s really an essential way of conducting business”. “For Santhera, that means being led by the needs of the patients and always trying to incorporate their voice and needs into every aspect of the company.”

    To this end, the company employs two patient advocacy heads, one in Europe and the other in the US. The pair have been tasked with placing the patient voice at the heart of the company, so that unmet needs can be understood and tackled.

    This approach led the company to conduct the first clinical trial in congenital muscular dystrophy as well as one of the first studies to enrol non-ambulatory boys with DMD — both overlooked populations within an already very rare disease community.


    Jodi attributes much of Santhera’s recent success to its focus on listening to the end user of its medications. Patient centricity and profitability may be seen by some as mutually exclusive, but Jodi explained that this was simply not true. “They’re intertwined. To be successful as a company you have to make a drug that addresses a realised need of patients in a way that they want to take it and at a price that can be justified,” she said, adding that commercial success has put the company in a position to help more people affected by rare conditions.

    “You have to conduct trials that patients want to participate in. We’re in the business of treating devastating rare diseases, and you can’t do that without the patient community, without understanding their needs and involving them in the process.”

    Planning and conducting clinical trials with the input of patients makes commercial and, more importantly, ethical sense, and it is a privilege to be part of that, added Jodi.


    Part of Jodi’s role is to listen to the patient voice, and then make sure it is heard by the right people within the company. For this to work well, patient centricity needs to be embedded in the company culture — from top to bottom and across departments.

    It’s not just about drug development, though. Identifying and addressing educational needs in therapy areas in which it operates is also pharma’s responsibility. “There’s an education component when you’re working with disease groups who haven’t previously had access to therapies and trials,” explained Jodi.

    This is especially the case in rare diseases like DMD and congenital muscular dystrophy (CMD), where there is an incredible lack of awareness, even among paediatricians. To help combat this problem, Santhera has worked with the patient community to launch the Take a Breath DMD campaign.

    The website www.takeabreathdmd.com provides a US-based information hub for respiratory care equipment and decision making in DMD. Recently they launched a corresponding educational website in Europe (www.breatheduchenne.com).

    “We worked as a team to help create these sites that address the highest educational needs of families and carers, and to make sure the content is family friendly and addresses the areas of greatest concerns,” said Jodi.

    Patient centricity is about building relationships that allow companies to develop the products that will make a very real difference to people’s lives. The key to achieving this virtuous circle is effective and transparent communication between all parties.

    In all other industries this is called market research and widely accepted as the best route to profitability. Pharma is now waking up to the fact it’s the right thing to do – both from a commercial and an ethical standpoint – and Santhera is one of those looking to lead the way.

    Further information: https://pharmaphorum.com/views-analysis-market-access/patient-centricity-makes-ethical-and-commercial-sense/