Patient advocates speak out on partnering with life science companies

What does patient centricity mean from a patient’s point of view, and what kind of support do patient advocates need from life science companies? Three prominent patient advocates shared their perspectives.

In 2010, Nick Sireau, a London-based father of two boys with the rare progressive genetic disease alkaptonuria (or AKU, also known as “Black bone disease”), had just quit his job in the NGO sector to focus exclusively on finding treatments for the condition. Like many so-called “kitchen table” patient advocates who were parents of sick kids, he’d poured the whole of his heart, soul, time and savings into the task. Unlike most, he’d already managed to identify a potential treatment, establish a patient group, solicit interest from the scientific community and raise funds for further research through the Royal Liverpool Hospital.

Patent rights to Sireau’s compound of interest, nitisinone, were held by Sobi, and the company had investigated developing it as a treatment for AKU. However, Sobi decided to focus its energies elsewhere following the 2009 failure of an NIH trial plagued by a too-small patient sample and poor endpoint selection. Sireau learned that the president and CEO of Sobi would be attending the inaugural World Orphan Drug Congress meeting in Geneva and set out to talk to him there.

“I contacted them beforehand and said ‘Look, could we please meet up, even just for a few minutes at the coffee break,’” said Sireau, “and we did, and I explained that we had a very good consortium, a good protocol, we had animal models, access to patients and would they be willing to consider working in partnership. And they said ‘Fine, let’s discuss it.’”

The resulting partnership between an international biopharma and a tiny, up-by-its-bootstraps patient organization produced a treatment that won EC approval last October and is now licensed throughout Europe (read this excellent Nature piece for more on Sireau’s nitisinone saga).

But back to that fateful 2010 conference in Geneva. “What surprised me,” said Sireau, “was that I was the only patient group there, and there were 350 pharmaceutical executives, and I was like, ‘I cannot believe that patient groups are not going out to interact.’”

What patients want from life sciences companies

Ten years on, stories like Sireau’s are becoming more commonplace, as genetic medicine advances and more biopharmas focus on rare diseases. The disconnect between companies and patients, however, can often remain substantial even today.

Sireau shared his story in a March 23 panel discussion alongside Henny Braund of Anthony Nolan and Claudia Hirawat of Voz Advisors, facilitated by Mike Ward of Clarivate. The topic: What patients want from life sciences companies. Here are some key takeaways:

Patient groups need help in raising funds, and with navigating regulatory systems and research worlds.

“In the early days, we were at sea,” said Sireau. “One of the things I realized is that even though in every rare disease the symptoms are very different, the challenges you face as a patient group are pretty much identical. It’s where do you get the funds, how do you identify the patients, how do you get them together as a community, how do you do the natural history studies, how do you work with them as an industry, what’s the regulatory process.”

Sireau proposes that patients and companies establish an independent foundation to distribute funds from industry to patient groups. “That would benefit everybody, because a well-constructed patient group can work absolute wonders when it comes to developing new treatments.”

Data-sharing can benefit patients and life science companies alike. Henny Braund, CEO of Anthony Nolan and the Association of Medical Research Charities, shared an example of a data partnership between Janssen and the Blood Cancer Alliance in the United Kingdom.“

There was a lack of a complete picture about hematological disorders,” said Braund, “so Janssen funded all of that data coming together in one place,” giving patient groups and the government a better view of the scope of the condition.

“Data is also power,” said Braund. “This isn’t just about the patient voice in terms of us getting their insights or experiences, it’s thinking about where there’s an unmet need that would actually help that community.”

Sharing power and decision-making with patients is essential. “This has to be seen as a true partnership,” said Braund, “and it’s a value set – it isn’t about profit. It’s about getting the best for that patient. Partnership means equal, doesn’t it?”

Patient centricity needs to be “a discipline across all stages,” said Claudia Hirawat, of Voz Advisors, “a procedure that’s systemic, that’s done across every single program of your portfolio.” Hirawat shared the example of Takeda, whose patient engagement office pulls the patient voice into functions throughout the company.

“It has to start with a partnership, and it has to start at the beginning. There isn’t one size that fits all. Just running a patient panel isn’t the answer to this. You have to think of different ways of bringing patients to the table.”

Henny Braund, CEO of Anthony Nolan and the Association of Medical Research Charities


Too many companies remain reticent to engage with patients and their advocates, for reasons ranging from a sometimes paternalistic corporate culture to fear of raising unmanageable expectations among patients, panelists said.“It is still a lot of underestimating the knowledge these groups have and their capability to be real partners,” said Henny, “and once companies have the courage to enter into these relationships, they’re usually extremely surprised, and those fears tend to dissipate naturally.”


View the entire hourlong conversation here, and don’t miss the next installment in our ongoing series on patient centricity, a conversation about achieving data-driven patient centricity at scale, on Thursday, May 20. You can register for that webinar here.