Reflections from the World Orphan Drug Congress

I'm sitting here on the final morning of the WODC reflecting on the many meetings, talks, panels and workshops that I've participated in?over the past couple of days.

As ever, this was a truly inspiring meeting. More than other events, WODC stands apart for me for the sheer passion of everyone who attends to make life better for people with rare diseases and their families. So many people with drive and passion to make a positive change.

Some talk on JCA (of course) but interestingly not as much as in previous meetings. Of course, unless you're an oncology or ATMP JCA won't begin just yet, but now is an ideal time to scope your PICOSs and look hard at evidence generation planning and execution.

?Not much on AI either.

What struck me more was the emphasis on the patient voice and early engagement, two topics I constantly bang my drum on. Some key takeaways here...

1. Patients should be partners, as early as possible and throughout the reimbursement process. Some of the best patient partnership we've been involved with recently have been really early and really late. Using cognitive debriefing and interviews to understand what really matters for patients and their families, and then using that to drive the entire access evidence generation programme. All the way through to patient groups reviewing sections of reimbursement dossiers to make sure they truly reflect their lived experience?
2. Tell a story. No surprises that I'll continue to shout about how important it is to paint a picture of the lived experience of a rare disease in all your communications. But I nearly stood up and cheered when I heard an HTA committee member saying that this is incredibly important in HTA decision making, and that industry doesn't always do it very well. Even if you don't get us to write your dossier for you, please push your HTA partner to do more here. Use the data, yes, but use it to paint a human picture of what the day to day reality of living with a rare disease is like
3. Early dialogue. Evidence generation in rare diseases has many, well-known challenges. Single arm studies and lack of robust natural history data being two big ones. There was lots of talk on early advice seeking, which I agree with. The advice was mostly to seek formal advice, via national routes or EU joint scientific advice. These have their place and are worth exploring. My personal view though is that they are often slow and expensive, and that you can often achieve better ROI and speed of advice through different routes (e.g. a steering committee or direct interactions with stakeholders). There's no right or wrong answer, but I would advise not falling into the trap of thinking formal advice is the only route

My personal takeaway across the sessions was the time dedicated to real world data. For me, particularly in rare diseases, the day of the RCT as trial-in-chief is over. In rare, it's often not practical or even ethical, so some level of RWE in an evidence package is almost certainly required. Finding the right data (if that even exists...perhaps relevant is a more appropriate word) is challenging. Which data source to use? Does one exist already? Do you need to generate data? What's the quality of the data? Does it have the right outcomes??

The challenge here is that HTA methods are still catching up, and I think that RWE is still seen as second best. We're moving in the right direction though, and I'm encouraged that the next 5 years should see a shift in the methods used to generate and incorporate RWE robustly into HTA, alongside an increased weighting of it in HTA decision making.

Lots to do still, but lots to be inspired by. And as ever it's great to know that the future of rare is in the hands of brilliantly smart and dedicated colleagues.