SHE Pod Season 3 Episode 2: Jessica Pace on Accelerated Access to Medicines

7:11AM Apr 14, 2022

Speakers:

Kathryn MacKay

Jessica Pace

Keywords:

medicines

patients

processes

people

important

access

jess

funding

questions

stakeholders

systems

medicine

guess

pandemic

project

approving

provide

tga

interviews

medication

Hello, and welcome to the SHE Research Podcast. I'm your host, Kate McKay. And today I'm joined by Jessica Pace, because Jessica has recently been awarded her PhD from the University of Sydney and Sydney Health Ethics. So we're gonna be discussing Jess' thesis today, which is entitled 'The Ethics and Politics of Accelerated Access to Medicines', some of which has been already published, and Jess will give us an overview of that. And we'll be talking about Jess' discussion section today. Hi, Jess.

Hi, Kate, thanks for having me.

Thanks so much for joining me. I wonder if you could give the listeners an overview of the project, or the results of this paper... thesis.

Sure. So in the things that kind of drew from my work as a clinical pharmacist, I guess, and it was... So I've, I've worked for... I've been registered as a pharmacist since 2015. And I noticed in my practice, that there's a... kind of my practice wasn't correlating what I was seeing in the media about access to medicine. So I found that I was hearing a lot of stories about patients who just couldn't access treatment they need. And I found that in my practice, that kind of mostly wasn't the case. So like, I work in hospitals, there's, there's committees, there's funding programmes, there's ways for patients to get, like 99% of the time that patients need access to medicine it seemed that the system was providing that. So I think the project came out of wanting to explore those contrasting perspectives. I ended up focusing on stakeholder perspectives of accelerated access to medicines. And what we mean by that, I guess, is official processes such as our TGA medicines registration processes, and our PBS funding processes that aim to provide patients with a faster access to medicines, or access to medicines that otherwise wouldn't be available through those... through those processes. And a lot of these have been introduced into those... in response to those calls by patients and other stakeholders for those faster access to medicines. So like I said, the project was largely focused on engaging with stakeholders. So I interviewed some doctors in hospice and patients, both in interviews and focus groups to get their perspectives on this phenomenon of accelerated access. And we published a couple of papers with the results of our interviews with doctors. And also, with those focus groups and interviews with patients, I understand.

What does it mean to have accelerated access, like does that mean in advance of TGA approval?

It can be, or it can also be just intim... initiatives that are introduced to kind of make those processes go faster. So there's something that the TGA that's called the 'Priority Review'. And that's when the TGA identifies a medicine as a potentially really important medication, and something that they have to take action on within a defined period of time. And that's faster than their general... than their general review processes. It could also be using different evidence or a different review process in order to provide access to those medicines. So for example, it could be using something like earlier data, so proving on the basis of a phase two trial rather than the phase the larger phase three trials that we usually look for, when we're approving or providing funding to medicines.

Interesting. And does this raise specific moral questions? Do you think?

Yeah, so I think I think it does raise a range of moral questions. The big one, I guess, is the idea of well-being and protection of patients. So if we're approving these medicines earlier in the development processes with the data that we're approving them on, there's a lot more uncertainty. So there may be side effects that we haven't... that we don't know about as yet, that further research would guide us to, and we also can't be sure about the effect that these medicines will have. So a lot of the times they've been made available on the basis of what we call 'Surrogate Endpoints'. So this is an endpoint, it's easy for us to measure. And we use it as like a marker or proxy for something that's clinically meaningful. So for example, if we look at cancer medicines, it could be something like progression free survival. And that's just means how long you how long you survive before your disease gets worse. But really, I guess what patients are mostly interested in with cancer is that they... how long is there going to survive overall? So is it really meaningful if we get them more progression free survival, but it doesn't actually extend their overall survival. So they would still pass away at the same point. I think that raises important moral questions about predicting patient's well being and protecting them from harm. And there's also broader community questions as well about allocation of resources. And I think this phenomenon... the ethical questions often come up, because a lot of these medicines are really expensive. They put a really big strain on our healthcare systems. So I guess questions about safety and protection still come up regardless of how much a medicine costs, but it's kind of amplified in the context of these medicines costing hundreds of 1000s of dollars each year, and we don't actually know if they're providing any benefits or worthwhile benefits. So in a context of limited healthcare resources, is this the best it is the best thing for us to be putting our healthcare resources into? Or could we as a community get larger benefits by funding other interventions for example, preventative healthcare?

So what were some of the findings that you had from your stakeholder groups on these questions?

What struck me was that it... I was kind of expecting to see those extreme views that you sometimes see in the media. So you might see people arguing for, for just, 'let's get rid of all the red tape all the restrictions on medicines, because really, it's just stopping. It's costing lives, and it's stopping people from accessing treatment that they really need'. But all of my stakeholders were really considered and they and they recognise that this is a really difficult problem, and that there's a number of really important moral and political considerations that we need to take into account here. So I think, if I was to summarise my results, it would kind of be... when you are stakeholders, if these, if this game should be introduced most of their answers was, it... Well, it really depends. Like, in some cases, in some diseases, there might be a reason to introduce these alternative processes. But we really shouldn't be doing away with our funding and regulatory processes that have served us really well for the past, however long. People who were advocating for faster and more access, they weren't advocating to completely do away with our systems, their their responses, that is there are patients who these systems aren't actually serving very well. And we... and how can we make these systems more more efficient and fairer to those patients to provide them with treatment?

Hmm, that's really interesting. So one thing that stood out to me from the discussion section, is that you found that I think, like you were just sort of saying that the groups were actually quite considered and fairly... had like fairly balanced views, in fact, and that they wouldn't necessarily always put an individual over the common good, in fact that sometimes they would prefer to benefit the the common good, you say? And I wonder if you could say something about that. So what kind of like, what were the trade offs that people were considering making here? And like, what does it mean to be benefiting the common good in this context?

So the example that I really like is one from the focus, a focus group, and we kind of gave patients a scenario, there's a new medication, it costs a lot of money. We... the evidence is kind of iffy, but the early results are promising. Would... if you were advising the government, would you be advising them to find this medicine? And then when a lot of our focus group said even people who had had cancer themselves or personal family experience of cancer, and who had looked overseas or looked, or, wanted access to certain medicines that weren't available in Australia, they would say things like, 'well, health isn't the only thing that the government has to spend money on'. So they'll say things like, 'what about schools? What about roads? What about defence spending?' Like, we need to think of all members of our community and all of those spending priority, rather than just focusing all of our money on on these new medicines. I think there was the sense that there could always be more put into health care, just because health is important. And then and, and it seems that there's rarely enough resources to to meet the demands on healthcare. But there was also a recognition that health isn't the only thing. And medicines aren't the only thing that the community could and shouldn't be spending money on.

Hmm, that's really interesting. I think.

That kind of made me feel warm and fuzzy inside.

(laughs).

Because sometimes in interviews or focus groups, as you know, you kind of have to put, like, steer people in or ask people about certain considerations. But this was just like my participants themselves. I just sat back in and they were raising these issues themselves. So they, like it wasn't as if I said, 'Oh, what about the schools? Or what about this?' And there were like 'No, like, no, no, we have to be thinking about all these different considerations and all these members of the community.' Which was really nice.

Mhhm. So I wonder, what would you highlight as the main points of interest or sort of maybe the main findings that have come out of this project?

I think it is. And I know it might seem simple, but I think it can get lost in our media discourse sometimes is that, like, people, people are reasonable and rational, and they care about other people.

Mhm.

And, and they, like, people can think outside of their own interests and their own concerns and, and see what's what's needed for the broader community. And then as you get I guess, if you give people time, space and information to consider these big issues, then they can have been really, really important things to say about them, that we that are really valuable for policymaking. And I guess, from a personal perspective, it was it was kind of nice just to see the way that the stakeholders views how different they were to what we what we see in the media discourse. And then again, there's that considered and that that altruistic view of a number of members of the community and then we can engage with these when we're... in terms of policymaking.

Can I ask you a question about that about the, what you said about giving people time, space, and information. And I guess this is kind of a methodology question. But but was... were those important parts of how you structured your stakeholder interviews?

Yeah. So the way that we structured our interviews that was that we tried to give... it was more important, I guess, for our consumers, rather than for our physicians, because I think our physicians know a lot more about how the healthcare system works, and how the processes for, for registering and finding medicines, and also the types of studies that are used at each stage of that process. But for our consumers, what we tried to do was to give them... to try and give them some information, and then give them some time to think about and then we changed, tweaked the information slightly. So we'd start off with what we thought was a pretty straightforward scenario. So we have a new medication, we have a large a large trials are involving 1000s of patients. There's quite good evidence to show that it does actually impact an important outcome. So it could be overall survival. Would you fund that medication? And then, even though the cost was high, a lot of people would say, yeah, there's the evidence is good. The outcome is important. And also there's an unmet need in the community. Yep. That's, that's something that's worth the funding. And then we've kind of, we kind of changed that scenario to try to make it a bit more uncertain. And we'd also provide information about how our medicines and funding systems work. So kind of start with this. We had some slides at the start to say this is the usual process for funding medicine. But there's been suggested changes, and this is how that would alter that process.

And did it change their answers?

So it's, when we introduce more uncertainty, it did change their answers, which was also nice to see. Because you'd hope that if you kind of take it back to a phase one trial of about 10, people make it really expensive. There's just so much uncertainty, you kind of hope that more people than not... would say, 'Look, we should think before we, before we make this really available'. It did work as we expected which, which was reassuring.

It is reassuring. That's good. I'm happy to hear this (laughs).

(laughs).

Um, well, thank you for speaking with me, we're kind of coming to the end of the time. I wonder if I mean, you've published a number of papers out of this project. And so maybe this is too big of a question, to ask for just one kind of takeaway message. But I don't know, do you have a sort of one or two kind of big takeaways from this project?

I think what my personal takeaway was, was that our systems are actually quite good, like, stakeholders are very, overall quite happy with these systems. Some stakeholders had particular things that they wanted fixed, and that's fine, like no political economic system is perfect. And, and if we weren't constantly adjusting and fixing these then, I guess that's not a good system of government, and of an administration. But yeah, most of our systems are working or working quite well. And people are generally satisfied with those. And I guess I think that that's kind of the COVID pandemic started just as I was finishing my PhD. And I found it reassuring that, in the midst of the COVID pandemic, we haven't discarded our current systems for medicines funding and regulation. So we may be doing things quicker for COVID. We may be looking at gathering evidence in different ways. But we still have maintained those systems and maintained those safeguards in order to provide access to COVID drugs in a really timely manner and to to fight the pandemic. But I know that sounds a bit of a cliche way of phrasing it (laughs).

(laughs).

But yeah, to provide that access to those COVID treatments, and we're seeing more COVID treatments becoming available. And the evidence to support those is quite good. And there's clear plans in place as well to collect further data and to make sure that these are safe and effective, and how we can best use these medications.

Excellent. Well, thank you so much for speaking with me, Jess, that sounds really interesting.

Thanks for having me.

No problem.

And thank you to everyone for listening.

Yeah, absolutely. Thank you for listening to this episode of SHE Research Podcast. You can find a couple of Jess' papers from this project linked in the episodes notes along with the transcript. SHE Pod is hosted by me Kathryn McKay and produced by Madeline Goldberger. You can find our other episodes on Spotify, Radio Public, Anchor or wherever you get your podcasts of quality. Thanks again for listening. Bye.