Table 3.

Quotes Regarding Investments to Patient Engagement in Medical Product Development.

Investments	Quotes from Patient Group Representatives	Quotes from Industry Sponsor Representatives
Dedicated Staff Time and Expertise	Our staff is small, so it’s like, our knuckles are sbleeding right now doing the work that we're doing. We don't have a lot of staff to accomplish these jobs, so everything [involves] quite a bit of investment for us. It's time, power and money that I don't have to hire somebody else and get the job done	So we actually have a whole new team. So I am focused on patient engagement, but there’s actually also other people on the—I have peers that are focused on like investigator and site engagement as well. So it’s a really kind of dedicated effort to think about from not just the patient perspective but also from the site and investigator perspective, how do we just do a better job? My role also came out of just a company wide recognition that we need to be thinking about patients differently. And not just sort of the end user Putting a team in place that can assist the clinical team with establishing those networks and opportunities for actually engaging with the patients or the patient groups or that list of different ways or different populations that you might work with to reach the patient I guess a big investment is just dedicated staff to run the activities, to maintain the partnerships with the external groups
Financial Resources	The big thing is money, and to raise more money. Our annual budget, with the creation of the registry and the care center network, has gone up substantially. And the more patients you enroll, and the more centers that you engage, the more money it costs. I'm focusing a lot of my energies right now on development and fund raising	We invest funds; so we have a sponsorship and a grants program that invest dollars directly to do these programs. We invest direct funds through partnership programs Well investments in terms of finances for sure. The FDA suggested that we hold a patient-focused drug development meeting. So we had to give a very sizeable grant to an advocacy group
New Infrastructure, Processes, and Organizational Policies	For other diseases, many already had experience in this arena, but we didn’t, and so we were starting from scratch. Because we created this from the ground up, it’s really building it from nothing. It’s an enormous amount of work, effort, learning, getting up to speed, meetings, funding, whatever it may be. Starting from scratch takes a lot of investment	The key investment is internal education, and for us at [company], that's really been a cultural change. Within the organization, to really put the patient at the center of what we do has been a significant change in how [company] operates and where we put our focus. That's required a resource investment on the part of [company], both by bringing on additional personnel or realigning personnel within the organization who will dedicate their full effort Although very cool and important, a lot of these [activities] seemed like a very heavy lift from a resource perspective in terms of getting kind of agreement across the organization so that we’d be able to do these things
Time, Effort and Burden Placed Directly on Patients	There are logistic investments in some of these things, and you’re working with people with [name of disease], it’s not easy for them to travel. You have to consider that aspect of it	No comments
Reputation or Ethical Principles	We’ve tried not to sell our soul, and that’s a very important issue for us. I’ll give you an example. Years ago, we brought our board together, and the issue was a clinical trial for a drug… and the clinical trial might have placebo. We met for a day. I remember it was a very unusual board meeting. We had board members in tears as they agonized over the following question. We had come to the conclusion that we felt that the placebo was not necessary, and that we thought that the placebo was not very good science. We didn’t want to know whether their drug was better than nothing. We also felt that we did not want to put patients who have [name of condition] in that kind of a situation. The discussion was if we oppose this clinical trial, it’s going to cost us a relationship with [the sponsor]; including a financial relationship in terms of further support. If we don’t, it costs us our soul. We voted for our soul. In fact, it did cost us. We adopted a principle that, so far, we’ve been able to follow. The principle was: does it meet the litmus test of what one needs to do to keep patients alive and well? … If it doesn’t meet that test, we’ll take the consequences of it	No comments