By NextLevel Life Sciences - March 09, 2018

Leading up to NextLevel Life Science’s 10th Edition PharmAccess Leaders Forum(Spring) 2018, we are conducting interviews with selected members of our prestigious speaker panel to learn more about their thoughts on this vital issue.

*Opinions below are those only of the individual and do not reflect upon corporate strategy or positioning.

For more information regarding NextLevel Life Science’s 10th Edition PharmAccess Leaders Forum (Spring) 2018click here!


Kieron Lewis, Global Business Development Director, Clinigen

NextLevel: What kind of work do you do at Clinigen?

KL: Clinigen is a company I have been with for four years. I advise on the strategy of early access programs, compassionate use programs, and pre-approval access to medicines where there is a high unmet need. Our company does the planning and the set-up stages all the way through to the delivery of the program to make sure they reach the objectives of the company and the patients in that therapy area.

NextLevel: How are early access programs impacting the collection of real-world data?

KL: It’s a good point and I think it is fair to say it is quite a new topic in the environment. We are seeing increasingly our client companies want more value from early access programs. Some companies are including real-world data collection as part of their early access programs. That's something that we do, we have an electronic data collection (EDC) system that, if clients want to include, we can build it into the program, and that enables clients then to collect some valuable data from both the physician and the patient on their drug prior to launch. Prior to approval that data can be used to support the value dossier and to support reimbursement processes. I think it is important to say that this is quite a new area and we are doing this now with a number of companies and we know that the EMA values/considers real-world data from Early Access Programs and it can add value to submissions - we know that lots of countries in Europe have regulations that allow for the collection of real-world data. It's a really evolving space.

NextLevel: Do you find that HTA agencies are giving scientific advice which is effective for collecting real-world data and for early market access?

KL: We do have examples where we see real-world data included in submission dossiers. We are seeing real-world data from early access programs copied in the submission package and we know that lots of posters are presented at international congress where data from early access programs is used.

NextLevel: Sometimes regulations or comparators are changing during the process of developing a new drug and data collection. Have you found that a challenge and can you suggest some strategies to deal with that?

KL: Not directly no, but I think that when a company decides they want to include real-world data collection as part of an early access program, there is a high degree of flexibility about what type of data is collected from which countries. The client would really drive what they want to collect and where they want to collect it from and then we would provide the solution that would allow them to do that.

NextLevel: Early access to innovative medicines poses an increased safety risk, how can early access programs best manage that safety risk?

KL: I think that's a good point because there are obviously risks associated with allowing patients access to an unlicensed medicine, there are naturally reservations that we see companies have. I think the most important thing is that it's a company's choice whether they make that medicine available, it's up to the company who they want to make it available to, and we increasingly see that companies do run early access programs, they have a great deal of controls over the criteria about types of patients and types of physicians they want to include in the program. So there are ways that risks can be mitigated by the companies if they want to go ahead. I will also add that in our experience we just don't see those unexpected safety signals from early access programs and that is backed up by the FDA. There are some statistics that the FDA have published for early access programs in the US where programs that where stopped due to unexpected safety events were less than 1%. There are some statistics on that, and I think you could almost argue that for a company with an unlicensed medicine, wouldn’t it be better to maybe know if there were those types of signals in a smaller patient population that can be controlled through an early access program, rather than wait for commercial launch? I think I would cover that point and try and cover it in a balanced way, accept that there are risks, accept that there are ways to control it, but also to find some examples of what we see and what the FDA has published on that subject.

NextLevel: What are you looking forward to most at our next PharmAccess event in London?

KL: Well I enjoy speaking on a subject that in itself is an important one. I enjoy preparing a presentation that is both engaging and relevant to the audience hopefully. I’ll do everything I can to tailor the subject so that it fits with the conference delegates and the briefs that people read. And I’ll make it as interesting as I can. I’ll hopefully provoke some thought and questions. Also I’ll get to network with quite a few people in the audience as well, that will spark an interest in early access programs and how it can relate to their market access plans.

NextLevel: Why do you think others should come to this event?

KL: I think what people will get here is a really broad range of topics, some of which they’ll know, and some of which will be new and I think it will help people involved in market access to identify new opportunities and new ways to launch drugs and prepare for the barriers that we know they will face.

For more information about this PharmAccess Leaders Forum please click here!