{"id":98550,"date":"2021-04-28T11:21:27","date_gmt":"2021-04-28T10:21:27","guid":{"rendered":"https:\/\/clarivate.com\/?p=98550"},"modified":"2021-04-28T11:21:27","modified_gmt":"2021-04-28T10:21:27","slug":"patient-advocates-speak-out-on-partnering-with-life-science-companies","status":"publish","type":"post","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/blog\/patient-advocates-speak-out-on-partnering-with-life-science-companies\/","title":{"rendered":"Patient advocates speak out on partnering with life science companies"},"content":{"rendered":"

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.<\/em><\/strong><\/p>\n

In 2010, Nick Sireau, a London-based father of two boys with the rare progressive genetic disease alkaptonuria (or AKU, also known as \u201cBlack bone disease\u201d), had just quit his job in the NGO sector to focus exclusively on finding treatments for the condition. Like many so-called \u201ckitchen table\u201d patient advocates who were parents of sick kids, he\u2019d poured the whole of his heart, soul, time and savings into the task. Unlike most, he\u2019d already managed to identify a potential treatment, establish a patient group,<\/a> solicit interest from the scientific community and raise funds for further research through the Royal Liverpool Hospital.<\/p>\n

Patent rights to Sireau\u2019s 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.<\/p>\n

\u201cI contacted them beforehand and said \u2018Look, could we please meet up, even just for a few minutes at the coffee break,\u2019\u201d said Sireau, \u201cand 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 \u2018Fine, let\u2019s discuss it.\u2019\u201d<\/p>\n

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\u00a0(read this excellent Nature piece<\/a> for more on Sireau\u2019s nitisinone saga).<\/p>\n

But back to that fateful 2010 conference in Geneva. \u201cWhat surprised me,\u201d said Sireau, \u201cwas that I was the only patient group there, and there were 350 pharmaceutical executives, and I was like, \u2018I cannot believe that patient groups are not going out to interact.\u2019\u201d<\/p>\n

What patients want from life sciences companies<\/strong><\/h3>\n

Ten years on, stories like Sireau\u2019s 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.<\/p>\n

Sireau shared his story in a March 23 panel discussion<\/a> 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<\/a>. Here are some key takeaways:<\/p>\n

Patient groups need help<\/strong> in raising funds, and with navigating regulatory systems and research worlds.<\/p>\n

\u201cIn the early days, we were at sea,\u201d said Sireau. \u201cOne 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\u2019s 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\u2019s the regulatory process.\u201d<\/p>\n

Sireau proposes that patients and companies establish an independent foundation to distribute funds from industry to patient groups. \u201cThat would benefit everybody, because a well-constructed patient group can work absolute wonders when it comes to developing new treatments.\u201d<\/p>\n

Data-sharing can benefit patients and life science companies alike. <\/strong>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.\u201c<\/p>\n

There was a lack of a complete picture about hematological disorders,\u201d said Braund, \u201cso Janssen funded all of that data coming together in one place,\u201d giving patient groups and the government a better view of the scope of the condition.<\/p>\n

\u201cData is also power,\u201d said Braund. \u201cThis isn\u2019t just about the patient voice in terms of us getting their insights or experiences, it\u2019s thinking about where there\u2019s an unmet need that would actually help that community.\u201d<\/p>\n

Sharing power and decision-making with patients is essential.<\/strong> \u201cThis has to be seen as a true partnership,\u201d said Braund, \u201cand it\u2019s a value set \u2013 it isn\u2019t about profit. It\u2019s about getting the best for that patient. Partnership means equal, doesn\u2019t it?\u201d<\/p>\n

Patient centricity needs to be \u201ca discipline across all stages,\u201d<\/strong> said Claudia Hirawat, of Voz Advisors, \u201ca procedure that\u2019s systemic, that\u2019s done across every single program of your portfolio.\u201d Hirawat shared the example of Takeda, whose patient engagement office pulls the patient voice into functions throughout the company.<\/p>\n

\n

\u201cIt has to start with a partnership, and it has to start at the beginning. There isn\u2019t one size that fits all. Just running a patient panel isn\u2019t the answer to this. You have to think of different ways of bringing patients to the table.\u201d<\/p>\n