10 February 2021 Case Studies

Conversation with...
Antony Appleyard, Technical Director Regulatory, talks about working with ATMPs

Summary
With experience of over 50 clinical development programmes in ATMPs, we’re in conversation with Antony Appleyard on Diamond Pharma’s approach to this complex area and the learnings so far.

In the article below we discuss:

  • What makes the team stand out
  • Beyond Glybera and YESCARTA®: Diamond’s 15 years in the ATMP field
  • The roadmap to success

How would you characterise Diamond’s approach to ATMP (cell and gene therapy) work, what do you think makes your team stand out?

Well our involvement with ATMP programmes has been extensive, all the way from initial strategy to operational delivery – getting programmes over the line. I think a key to this is the experience in the team and the excitement that I see in our consultants driving these new treatments towards patients, so they’re pretty passionate about the work!

In terms of our approach, we always start by building a strong roadmap to identify risks and opportunities for the programme, and central to this is working with the end in mind: we’re always thinking about the steps towards approval. That plan is a starting point – a reference to help adapt the approach - ATMPs are a rapidly developing area so we all need to stay agile and assess the programme on the go.

Diamond has helped clients with over 50 clinical development programmes for ATMPs, so what would you say are the biggest learnings so far that you’d pass on?

If you plan on being in more than one territory, start dialogue early. This doesn’t need to take huge time or investment, but steaming ahead with let’s say, FDA dialogue without at least some conversations with others like the EMA can cost you dearly later on.

Don’t lose sight of the goal, always keep the end-patient benefit high in your mind and reiterate it often and remember with these emerging technologies the roadmap continues: where rare indications are being targeted it is important to engage in discussion about what is approvable against the data that is emerging.

What would be your top tips for a CEO looking to save time and money en route to ATMP approval?

First, draw up that roadmap: it’ll help you spot hurdles and opportunities quickly. It also helps your team to get behind the mission.

Second, talk to the right people at the right time, which generally means earlier than you think. Develop your position, evolve and improve as you go.

Thirdly, a specific tip, based on our experience so far: check and challenge what key stakeholders like your clinical trials and manufacturing teams are telling you, especially if they’re outsourced. Watch carefully as you scale, as manufacturing moves between suppliers and methods change subtly. Always be ready to defend your comparability studies – because if a faster track route to market is possible you want to make use of all your data.

What are you proudest of around Diamond’s ATMP work ?

Up to now I’d say our work on YESCARTA® and how we keep rolling into the next frontier in regulatory science. Right from inception of this CAR-T programme up to the MAA, Diamond was there as the EU team, truly integrated and part of their global regulatory team. That sense of integration made me proud, and it’s a sign we’re doing our job well.

It’s still less than 10 years since we helped get Glybera over the line as the EU’s first-ever approved gene therapy so ATMPs are still frontier work, especially when you think about gene editing, highly personalised therapies and everything else that is coming down the line. So, I guess generally I feel proud of our track record, that we’re as experienced as anyone can be in a new therapy: I know we’ll very likely build a good roadmap and set the client on the right course with a plan that’s strong enough to react to the unexpected.

 

THE UPSHOT

Start dialogues early and start with a strong roadmap – don’t lose sight of the end goal, always keep the end-patient benefit high in your mind. And work collaboratively with an experienced team that’s passionate about its work.