Session Notes: Market Trends – Optimizing Post-Trial Access (PTA)

Apr 23, 2026
Market trends- Optimizing Post-Trial Access (PTA): Why the pharmaceutical industry is streamlining  - Transcript
00:00:00
 
 : break. Let's see if we can get some more people in the in the in the room. So, um while we wait for the rest, I was just introduced myself. I am Ana, head of expanded access at WP Clinical. Um WP clinical is a specialized CRO that um for expanded access program access confession legal trial market access. So we have a very complete zero and also one of our strengths of strategy. So what I want to do today um with my session is to combine um what we learned so far and apply it to what we can do to optimize a clinical trial. I have a list of a small takeaways that I took from the other presentation that were very interesting and that can very well relate to what I'm going to present. One of the thing that really stressed me yesterday was that making R&D faster and cheaper is one of the challenge that everyone has faster and cheaper the R&D and there is a lot of pressure on R&D to be more efficient and another element that I also was very u striking yesterday is where the founding is one of the biggest challenge in this moment especially for the biotech which we all agree in the past two to three years founding has been really really tough and with those two points that I took now I want to start that presentation because I brought here a case study that actually takes exactly those two points that we were
 
 
00:01:53
 
 : listening so far so the case study that I want to present today is a small biotech with relatively small number of facets that is developing a therapy for um a chronic disease quite large number of patient ongoing on clinical trials and they all have those patient finishing a clinical trial to open lab extension. So so far no particular problem. What happened? They came to us last year and they were like, "We need a wave to cut. Somehow someone of the cleanups team was speaking with someone from big farmer and they discovered that big pharma keep patient on treatment quite differently. They don't do open label extension. They don't have patient on clinical trial protocol updates for seven years. Big pharma work differently. So they came to us and they say look we have this problem. We have extremely pressure from the board. We need to cut costs. We are also looking for funding and we need to restructure how we are dealing with the clinical trial. What we can do?
 
 
00:03:12
 
 : So the first assessment that we did is like okay so you definitely are doing everything correct but you are doing clinical trials there are other options. The second assessment included challenge like you have a huge budget pressure. The other challenge a lot of patient on treatment in clinical trials. We are not speaking about a rare disease and the other is the rate mutation. They commit to keep those patient on treatment for a long while. You cannot go back from that. I mean you can but you need to deal with the consequences of your image. So with that we sit together with the team and we took a little bit of a closer look of the project. What happened is that when you have something like that something that we never need to forget it's like we are here for the patients and this is my third time in this conference I must say and again for the third time I am the only person who will say patient come first. We sometimes lose ourself in pharma with tools, strategy, people management, change management, business case, money.
 
 
00:04:43
 
 : But at the end we are in the business of saving life and saving life is patience. And in this case they need to put the patient in the middle. So there is no other option. You say okay first of all what are the patient doing? How are the patient behaving? Are they doing good with their treatment? Yes. Then they need to stay. This is the thing. The second it's money because uh we are not in the business of NGO. None of us business is business and then to keep a a company afloat you need money and money is investors shareholders depending. But we need also to be blend. We cannot do this with teams. The other thing always image I mean this is not only about the patient what they think about the company is what the hospital think about the company what the regulatory authorities think about the company and then what the public think about the company because if you want to exit or be acquired you need kind of a good reputation.
 
 
00:05:48
 
 : And then the last thing is expertise. And if you have that then somehow you can restructure a bit with your cost. And this is the framework that we use for this biotech to say okay let's work around this and then make your patient on clinical trial to be more efficient. So before I start speaking about post-trial access, I need to pose a moment and make sure that everyone has the same knowledge. Meaning most of you know very well that patient in clinical trial don't remain in clinical trial forever. At a certain point the clinical trial ends last patient last visit data lock there is an end of the trial. However, if you are in oncology, rare disease or neurology for the patient is not the end. They maybe are doing very good on the treatment and from an ethical perspective you should keep them there. But then is the end of the trial but what happened to the treatment? So two different things. Now if we go a moment outside the clinical trial there are alternative options which is called it post relaxis option to keep patient on treatment surveilling the safety clinical the clinical team is still very happy decides to but those patient are not in another clinical trial and for us in the jargon is called it posttrial access and that option is very expanded and this is what the big farm are doing.
 
 
00:07:29
 
 : I come from rushini. We were doing this all the time. Patient come out and you put them on a different setup. You keep surveying safety. You keep supplying them but you don't have all the costs that you have with the trial. Why this is a trend? This is this is why I'm here to do the presentation. So what we are seeing as a specialized zero is like in the past three years we have a surge of requests like the other biotech we had like at least 10 same story coming to us okay we need to restructure our cost for the clinical trial open exential very expensive what can we do but why is that is because in the past years the declaration of has evolved and more and more that patient were left behind after trials In some countries, no. In some countries, yes. And to make sure that it was unequitable access to the treatment and the trials. The declaration of unsyncing has added two years ago a specific article and say sponsors, you are responsible to what is happening to the patient after the trial.
 
 
00:08:42
 
 : Before was like more advice. Now it's really like okay, you need to get your act together. you need to get your protocol together. We don't want to see patient running out of the trial with no more options. So the market trend I was telling is exactly that. So now more and more of the small and mediumsiz company um are seeing this but because also the money is scarce so the investment is quite complicated. So they are seeing okay where can I optimize my cost and for the people here that are working on portfolio management and project management and you are maybe the lead of a specific product and a program for you is key. If you discover that there is a trial that you can cut five million and use those five millions to something else, I can tell you you will run through my door tomorrow. The problem is that you don't know because the decision for the budget of the trial has been decided at the very beginning and probably the budget includes the three or four years of open lab extension.
 
 
00:09:55
 
 : So you think that the full budget it's okay but if you go back and maybe challenge the Kenoff's team you say look are you sure that this is the best option. Are you sure? Because we are like looking for a couple of million here and there. Can we optimize it? And this is what we are seeing more and more now in the market. However, and this is one thing that also was registered yesterday. Why so much resistance on the PTA? And yesterday what I learned it was change comes with resistance and pain. And this is for the change management is key because the cleanups team only knows clean and clinical trials. Now if we go on the behaviors on the different size of companies quite simple we have big companies I was for rush I was in the team doing expanded access and PTA I know very well how rush is doing super optimized novart is the same I know GSK so let's take the top 10 pharma they get they have the act together so they have policy in place very clear strategy the team comes at protocol devel developments.
 
 
00:11:20
 
 : So we were at the protocol development already there saying okay what happened to the patient when they go out in the protocol was already included and the first patient out he already has a plan. So everything good but we are speaking about probably top 10 farmer and there you have the middle size here now that I am in the other side of the service provider I can see a variation there are middle-size pharma they are doing very good same as Russian artist but others is like okay they have kind of a policy in place some sort of strategy And then in the execution the problem starting to begin. And what we can see often here in the midsize pharma is that because maybe oncology and things like that the first patient comes out and they are rushing to the team from medical affairs. Ah you are doing compassionate cues can you take these two patient to you and this is it. So there is kind of this way or some just do by themsel and then they do it but we have a variation but my preferred of course are the biotech because the small biotech they have really other problems.
 
 
00:12:41
 
 : So they are existential problem in the biotech. So when the first two or three compounds start in phase one and phase two uh the last thing they are going to think is about what is going to happen to the patient after the clinical trial. very little number of biotech are really different and then if they are lucky and they have someone from big pharma someone is going to ring the bell but if not then it comes like last year when we had a number of company coming to us and say okay we are having those patient on no level extension we are burning millions but we don't know what to do and that is where we say okay let's sit down and see so we can see but some of the small biotech implement this faster than that's the thing goes. So let's talk about the change management and why the business systems because this is why the point is this. This is what the clean team sees clinical operational clinical trial of course. Reality is you can have patient that exit the clinical trials in other options.
 
 
00:13:59
 
 : You can put them on single patient pathways very similar to compassionate use. You can you have the cohort program depending from some countries like Italy um quite structured way to take patient out of the trial put it in a cohort that is not clinical trial but is a post program or you have very clear mandated countries like Brazil that tells you exactly what you need to do and then you follow which for some people is almost better because they tell you this is what you need to The problem that we can see here is the fragmentation and also everything that is here requires a high level of expertise in regulatory because this is completely fragmentated. Every country is different. will depend from number of patient, type of protocol, type of thing. You also have requirement in term of supply chain. So it is scary because at the end you are going out from a GCP world where you are very comfortable into a world where there is no nuances and there is more challenges and then it's sometimes there are not clear pathways and then you need really expertise and when the big pharma they build that they have everything in place the more you go down in term of size the more this become a huge challenge and then the resistant comes because it's like okay
 
 
00:15:33
 
 : why I need to take my patient out of something that I know I trust to something that I don't know but then you look at the big it's like okay but they are doing it so it should not be that scary and if we compare the old extension versus um kind of PTAP we can see clearly that the difference are not huge. There is definitely one GCP versus not. There is high cost per clinical trial versus maybe something less expensive. And then the implementation but still there is substantial change is big. Now let's go back to our case study at the beginning. what we did with that because they arrived um we advised them what we did we go back to patient at the center how many patients what is the volume what is the commitment I mean in the protocol you committed to something we really need to understand what it is because we need to honor that at least this is what we advise then every company do what they want but we advise if you commit to this you should go for then in that situation we took the protocols we took the information we sat down with the clinical operation team and then we analyzed country by country protocol by protocol and everything to understand okay what is possible and it's possible in this way and that way what are the vendors everything because it is quite a complex thing and then again on the change management.
 
 
00:17:22
 
 : An element that we could not underestimate is the education because we can do a fantastic slide deck on strategy but if we don't educate the senior management this is just a couple of bites into the computer. The people that take the decisions are not the clinical operation people. They are not. They execute. We need to convince sea level. You need to convince the one who approved the budget. All those people need to believe that this is a valid option costs and this is where the senior management summary comes in the meeting because we analyze the things and then we do a comparison of the cost. It's like okay you have this open li extension for three years commitment 3 years time let's compare with apostasal access in this and this and that you have your cost on this this is what you are going to like g in case you decide to move those patients to that that works very well I can tell and then the other element and this I must say is way More important than the cost is regulation because you can do the best plan in the world but if your sites are going to be hungry with you the phase three trial or the next trial is going to be complicated and this is not to underestimate.
 
 
00:19:04
 
 : This is why when we do this analysis we need to have a risk assessment and say okay if you decide to move those patient from an oil where the side know what to do they maybe also are paid and you go to a postraal axis with this different setup what is going to be the impact on the single sides especially if we know that those sites are going to participate to the next trial. Second, if you decide to go back to your commitment, which by the way happens, we had client that decided to say, "No, you know what? We want to cut the commitment for this trial from I don't know how many years to one." Okay. What is the reputation damage on the regulatory authority and ethic committees? you committed to maybe three years and now you want zero that is also an impact I mean it is a business decision of course but you don't need to underestimate that those decisions will take also a consequence later on on your next trial and then from a public perspective because again especially if you are a small company and maybe you are considering a couple of years an exit or someone else to buy you out you need to make sure that you have a reputation intact and some decision can be very unethical.
 
 
00:20:22
 
 : So you need to pay attention and this is why the risk assessment with the cost analysis and the expertise it's key in all of this because then as a single management you can say okay now I have the facts I can take the decision and is after all of this they still think let's continue with O because even if it's expensive the risk are too high but everyone then is okay they understand but if like no what let's move to PTA and then um we deal with uh the the fallout then it's okay. So this is basically what it is and this kind of case come to us more often than you think because in this moment the money is scars and this is what is happening. So the first trigger to have this analysis is the financial pressure. So take away and then I'm done. I think I have more time. Um because I also had something very interesting which was my team. Um choose your heart or the heart we choose you. This is exactly the same.
 
 
00:21:36
 
 : You need to take a decision on the cost, the operation and everything. But if you choose it then you are okay. If not what is happening the heart choose you meaning you have financial pressure and then of course never forget that if we do clinical trials is because we believe in the product and we believe that you can help patient with the disease and this should keep being like the life motivational treatment after the trial. So patient are there and they are not commodities they are people and we need to make sure that the people remain on treatment if they benefit from it they should and this is why ethically the the declaration of sini is also pushing the sponsors to do the same. Thank you very much. No, it's actually Yes. Yes. Hello. Yeah. Uh, two quick questions. The first is maybe I didn't really understand is where does the cost saving from come from in the GTA? The second kind of lead on question is given the patient benefit through that program.
 
 
00:23:09
 
 : Um does that influence or bias the trial results? I mean do you see an increased placebo effect because the patients want to see a positive response for example in the trial? The second is no. If so we are speaking here about oncology, rare disease and neurology. We are always speaking on situation where patient cannot go to a standard of care and and they are benefiting ethically. You need to go. We don't see a change in term of the data also because the data are locked and then we are just you know keeping the patient on treatment there is there are some instance where we have data collection but it's more about longterm data but no the first question is when you are in a clinical trial in GCP you have all the foil of a clinical trial monitoring testing like visits committee submissions this is very expensive on a PTA when you work with like expert people of a PTA mostly that fall all out you don't need monitoring because you are not collecting the data for um for regulatory submissions what you do is you supply the patients and then you collect the data for safety but the safety data collection is completely different there is no monitoring there is also no ethical committee submission no update protocol all that fall down so this is why we are speaking about half if not a third of the
 
 
00:24:34
 
 : cost. Next questions. Yes. Yes. Yes. If there is a compassionate use. Okay. So here we are speaking on PTA the majority is very similar pathway from a regulatory perspective of compassionate use. similar. However, this question comes very often. If you open a post trial access for patient on trial is this means that all spontaneous cases can also be included. This is a fear that comes all the time. The response is no because at the end what you are opening you are keeping patient on treatment on the trial. you have very clear inclusion criteria and the inclusion is the patient needs to come out from that trial to keep the treatment. If this is the incl

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