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Standing Committee on Health, Aged Care and Sport
Approval processes for new drugs and novel medical technologies in Australia

TUFFAHA, Dr Haitham, Private capacity


CHAIR: Thank you very much for joining us this afternoon. Please introduce yourself, Dr Tuffaha.

Dr Tuffaha : Thank you very much for the opportunity. My name is Haitham Tuffaha. I am an associate professor in health technology assessment at the University of Queensland Centre for the Business and Economics of Health.

CHAIR: Are you appearing on behalf of the centre or as an individual?

Dr Tuffaha : I have to mention that I'm an NHMRC research fellow, so I'm funded through the NHMRC, and my submission was based on the findings of my research under that fellowship.

CHAIR: Wonderful. I'm required to alert you to the fact that these are legal proceedings of the parliament. The giving of false or misleading evidence is a serious matter and in some circumstances could be regarded as contempt of parliament. Today's proceedings are being recorded by Hansard and attract parliamentary privilege. I should also let you know that they're being broadcast live through the Parliament House website. Thank you for sending in your submission, Dr Tuffaha. Would you like to make an opening statement before we move to questions?

Dr Tuffaha : Yes, sure. I just wanted to give you an overview about the program that I've been researching over the past 40 years, which is funded by the NHMRC. It's called, Improving patient access to novel cancer drugs in Australia: striking the balance. What motivated this program was a Senate report in 2015 that found that there were delays in accessing novel cancer drugs in Australia, and the main reasons behind that were uncertainty in the evidence provided to evaluate those new technologies. The report called for more flexible and novel approaches to evaluating new technologies. So, in this program I applied and developed new methods that can be used to strike this balance between providing early access to novel technologies and maintaining evidence based evaluation of medicines to ensure efficient funding and sustainable use of resources.

CHAIR: Thank you. I might get you to elaborate on that. In your recommendations you propose, for the reimbursement process, expansion of managed access schemes. Are you able to elaborate on what you think the benefit of that is and what aspects of the current process would be available to managed access schemes? Or are we only talking about for rare conditions or orphan drugs? Perhaps you could elaborate a bit more.

Dr Tuffaha : A managed access program—or managed entry scheme, as it was previously called—is an approach that is based on providing the new medicines with conditional approval. So, we give the new medicines the benefit of the doubt and say, 'Well, we will fund that medicine conditional on collecting additional evidence.' And when I say 'evidence' I mean any evidence that is relevant to the decision-maker in terms of safety, clinical effectiveness and cost-effectiveness. So, instead of saying yes or no—making decisions that are dichotomous—we can say 'maybe', but we need to collect that necessary evidence over time. The agreement is often governed by certain conditions that are struck between the department and the sponsor of that new technology.

During my research we conducted the most comprehensive review of the agreements, from 2010 to 2018. We found that there is underutilisation of these agreements by the department. Only three medicines underwent such agreements—

CHAIR: Three managed access?

Dr Tuffaha : Three managed access programs that were concluded. And we provided some recommendations, as I just mentioned, particularly around the need to engage stakeholders, patients and sponsors in the development of these to have more-balanced agreements. Most importantly, we need to apply frameworks that can guide us as to when and how to start such an agreement, how to implement them, how to monitor them and how to exit them, if you want to exit, because the last thing we want is to list the medicine and to have it there without being able to withdraw it or de-list it.

CHAIR: So, with the current processes in place, effectively a company can apply to the department to basically have a managed access agreement? And what criteria do they have to demonstrate? You said there were three cases. If you know it off the top of your head, maybe you can outline what the circumstances were in those three cases.

Dr Tuffaha : According to the managed access program and managed access scheme guidelines, there should be a high clinical need and uncertainty in the evidence, and these would trigger discussions around the need for a managed access or managed entry scheme. The problem is that these are sometimes subjective and not very well understood by both parties. We noticed that most of the time managed access programs are the last resort. So, it's not where we start in the process, where we assess effectiveness and cost-effectiveness and at the same time say whether or not we want to engage in such an agreement. It's the last resort, when we've exhausted all negotiations, including price reductions, and then we would say, 'Okay: why don't we engage in such an agreement?' It could be triggered or started by the department or by the sponsor. So, there is no rule as to who should start that.

CHAIR: Do you know the details of the three that have been listed through this mechanism?

Dr Tuffaha : I can get that information—

CHAIR: Feel free to take it on notice if you haven't got it at hand.

Dr Tuffaha : I'm just looking at the three examples. One of them, crizotinib, was initiated by the PBAC, and the rest of them were initiated by the sponsor.

CHAIR: In the existing scheme, how is the price set?

Dr Tuffaha : This is something for which there are negotiations between the department and the sponsor, and most of the time these are confidential. Researchers and the public don't have access to most of the details of these schemes.

CHAIR: I'm interested in your reaction here. Various countries around the world have been highlighted to us for having almost a reimbursement system as soon as the drug is lodged for reimbursement. It's an interim reimbursement system, a bit like a managed access scheme. Obviously, if it goes through the assessment process and is found wanting, then it's removed from the list. The two areas about which I had a little bit of angst were, firstly, does such a scheme effectively then give the pharmaceutical company the upper hand, because it becomes a drug that is being used by people with a condition and, therefore, you have increased community pressure to continue the reimbursement of that drug? And, secondly—in a very related issue—how do you actually withdraw a reimbursement decision from a drug which may have started to be used by a reasonably large cohort of patients, and might have a material impact on their lives, but be withdrawn because it doesn't meet the cost-effectiveness criteria?

Dr Tuffaha : These are two excellent points. Again, we want to strike this balance between providing early access and at the same time maintaining rigorous evaluation and efficient funding. I'm not aware of any jurisdiction where they engage their list of medication directly after it gets the regulatory approvals. Most of the time, there is some evaluation and technology assessment before we come to the stage of starting a managed entry agreement, so this should be carefully thought of and considered after engaging with all stakeholders, as I've just mentioned. Now, with this transparency, and using the frameworks that we are calling the PBAC to use, such as a value of information framework—and this has already been incorporated in Canada, in the guidelines of the Canadian Agency for Drugs & Technologies in Health—we can provide an objective way to show the public why we decided to engage in such an agreement. Then we can develop, together with stakeholders, that agreement to make it balanced. In that agreement, we should have a plan. We've called it 'mexit', like Brexit, for 'managed entry exit'. Whoever designs that plan should make sure it clearly states, 'In two years, we will collect evidence if the company fails to provide that evidence,' or 'If that evidence does not meet the expectations, there will be steps 1, 2 and 3 in terms of withdrawal.' We can plan the schemes in a better way to serve its purpose and also the needs of stakeholders.

CHAIR: Thank you. Dr Freelander, do you have any questions?

Dr FREELANDER: Yes. Thanks very much. I'm just interested in this managed access scheme. Isn't it really a provisional registration scheme? You're giving the company the provisional right to market the drug, and the government pays for—

Dr Tuffaha : The managed entry?

Dr FREELANDER: Yes, the managed entry. The government pays an agreed price. Is there then a time limit to collect the evidence?

Dr Tuffaha : Normally there is. The three schemes that we have reviewed had a time limit. In two years or three years we have to follow up on the evidence collected. We will revisit that evidence, and the sponsor, as part of the agreement, has to provide that evidence. Failing to do this is a breach of the agreement.

Dr FREELANDER: So it's their responsibility to collect the evidence over, say, three years and pay for the collection of that evidence. Are they the ones who finance it?

Dr Tuffaha : Yes. However, that shouldn't always be the case. Most of the time the sponsor or the company will pay for the data collection, and also for the clinical trial. Sometimes it's not feasible or not ethical, because the drug is already listed or registered and it may not be ethical or feasible to do another randomised controlled trial. However, there are other ways of collecting evidence. That's one thing. The other thing that we've called for in this research program is a linking of research clinical trial funding to such agreements. So, even if the company or the sponsor is not able or not willing to sponsor data collection, there should be some options whereby the government, through NHMRC or MRFF, may be able to commission research to answer important questions that can inform.

Dr FREELANDER: It would also be a pathway for the orphan drugs. You said there was one in the scheme, I think. Did the TGA promote this one product?

Dr Tuffaha : That's at a later stage to the TGA approval. The drug is already TGA approved, and now it is going to the Pharmaceutical Benefits Advisory Committee for evaluation.

Dr FREELANDER: But you said this was sponsored by them. I think you said two were sponsored?

Dr Tuffaha : No, that's the Pharmaceutical Benefits Advisory Committee, not the TGA.

Dr FREELANDER: Okay. So the PBAC sponsored one?

Dr Tuffaha : It initiated one.

Dr FREELANDER: It initiated one.

Dr Tuffaha : The question was about who initiated or started it, if I understood you correctly, not who sponsored it.

Dr FREELANDER: Sorry, I probably wasn't clear. What I mean is: who paid for the initial process?

Dr Tuffaha : It was the sponsor. The sponsor paid for the data collection. It was the responsibility of the sponsor to collect and provide the data.

Dr FREELANDER: Yes, but you said that in the three that were completed, two had sponsors that paid for the process and one had the PBAC.

Dr Tuffaha : Yes. To reiterate what I said and to clarify that, there were three. One initiated. The party that suggested that we should engage in this agreement was the PBAC, and on the other two occasions it was the sponsor. However, the data collection itself, after the agreement was reached, was the responsibility of the sponsor on the three occasions.

Dr FREELANDER: They paid for it all. So there were none that were actually paid for by the government.

Dr Tuffaha : No. On one occasion, I think there was some sort of double-checking of the data—it was from the registry data; that's a different source of evidence—just to make sure that the data collected by the sponsor was reasonable or reliable. But it was the responsibility of the sponsor to collect the data.

Dr FREELANDER: Do you think there is room to increase this scheme as a way of getting things to the market more quickly?

Dr Tuffaha : Yes. This scheme is one tool to improve patient access and to get things to the market more quickly. However, it shouldn't be the only tool that we have. Increasing the utilisation of the scheme should be considered as part of a framework of when we should engage in such a scheme, how and for how long.

Dr FREELANDER: But do you think that it could be a large part of how we get things to market more quickly, or do you think it will always be a small part?

Dr Tuffaha : I would say that using a framework to assess the need for such an agreement should largely be adopted, because that will be a triage point where we can say, 'Do we need this or not?' And if we realise that we need such agreements, we need to make use of that program more often, instead of engaging in sometimes endless negotiations until we reach a certain price.

Dr FREELANDER: Thank you.

Ms BELL: Thank you for being here today. It's an interesting area that you're working in as an academic and I appreciate the time and effort that you've put into that. You mention in your submission that the research is focused on novel cancer drugs, specifically in that area. Obviously, cancer's a very important part of research. Do you think there's any difference in how well the HDA process assesses cancer drugs compared to drugs for other diseases, and to what extent do you think cancer needs to be treated separately to other diseases for HDA purposes?

Dr Tuffaha : That's an excellent question. The reason I focus on cancer drugs is that I am a pharmacist. My background is of specialising in healthy economics and I was a clinical oncology pharmacist. So this is why it was something I wanted to explore at the personal level. The other thing is that most of the technology, in terms new drugs, is targeting cancer patients. However, the same principles, the same tools and methods, should apply to any health technology and journals. In that review where we looked at managed access programs, we looked at all drugs not necessarily cancer drugs.

Whether or not we should treat cancer drugs differently, if we want to consider equity, I don't think we should provide special treatment for cancer drugs or cancer patients. A life is a life, no matter whose life it is. What we need to do is consider the needs of different people the best way we can but not at the expense of the system or other patients—because there are always needs for other patient groups. I don't really support 'special treatment' for one group or another, being cancer or another group. We need to be fair and equitable for everyone, but we need also to consider that certain groups have special needs.

CHAIR: From one pharmacist to another, Ms McBride.

Ms McBRIDE: Yes, thank you, it is good to hear from another pharmacist today. Dr Freelander was talking about the reimbursement process but I want to go back a step to the registration process. You mentioned international collaborative opportunities to improve the uniformity of drug registration requirements, and you went to the particular example of Project Orbis. Are you able to expand on that a little more and how that might inform other such collaboration?

Dr Tuffaha : I'm not very well versed about this program. It's a new program. My understanding is that this program was initiated by FDA Oncology and it involves countries including Canada, New Zealand and Australia. They're trying to unify the process of drug registration, so if a drug is evaluated in one of those countries there is no need to repeat the entire process of evaluation. This doesn't mean that we're going to delete the Australian TGA revision, but it might speed up the process and make it more streamlined towards the areas that we want to focus on in Australia. There's not very much from me about the program in detail, what's happening right now et cetera, but I'm aware of the program. I heard the leader of that program speaking at a conference and I checked their website, and I'm aware of that ongoing project.

Ms McBRIDE: You also mentioned the registration process, and others have commented on this as well, about better alignment between the TGA and PBAC process. Are there good examples that you've seen elsewhere or is there some sort of best practice that you would suggest or recommend?

Dr Tuffaha : It's called the parallel process, where new technology is considered simultaneously by the TGA and the PBAC. It is an extension of programs like horizon scanning, where we identify opportunities coming and then building on these, instead of waiting for the TGA to evaluate, assess, a drug and then move to the PBAC. At the same time, we look at that evidence for the TGA, which is mainly around safety clinical efficacy. We also start to anticipate what sort of evidence we need for the PBAC, which is mainly around effectiveness and cost-effectiveness. So there are lots of examples, currently, in Australia. It's something that, I think, is an established process but maybe we need to enhance it more. It is something that's not new to Australia. It's happening in many countries.

Mr ZAPPIA: Thanks for your evidence. Dr Tuffaha, getting back to your submission, I understand some 63 per cent of all the cancer applications for PBAC funding were rejected. Are you able to tell us how many of those applications had been approved in a comparable place like Europe, the UK or the USA? Equally importantly, were there any findings subsequent to the approvals in those countries that justified Australia's rejection of them here?

Dr Tuffaha : Thank you for the question. I just need to clarify that the program did not really consider comparing any acceptance or rejection decisions across different jurisdictions. That was not one of the objectives. Unfortunately, I cannot really give you that comparison you've asked for. It would be an interesting area to research. Maybe we can do this in the future. We did some comparison in the very narrow area of lung cancer. I can provide the committee later with some of the findings, but not a general comparison for that 63 per cent that you just mentioned.

The other thing with this rejection rate is it doesn't mean that those drugs did not enter the market or were not listed on the PBS. That's the rejection from the first decision. There is the first submission then there is a deferral and a second submission et cetera. Those rejections were rejections from the first submission, which is expected. Some of those drugs were eventually listed, so it is unfair to say that more than 63 per cent of the drugs were rejected.

Mr ZAPPIA: Okay. Do you have any information or knowledge of any rejections, regardless of whether it's that 63 per cent I referred to, where subsequently the decisions by the Australian authorities were proved to be justified on the basis that there were problems found with those particular medications or they were in fact withdrawn by other countries?

Dr Tuffaha : I don't have that evidence right now to provide. I have to commend the Australian system. The way we evaluate medicines with the PBAC is one of the most rigorous and excellent systems in the world. It is something we should be proud of. Each jurisdiction has its own circumstances and its own price negotiation channels et cetera. I don't have that to provide right now.

Mr ZAPPIA: Thank you.

CHAIR: Thank you for your evidence today. It has been quite valuable having your academic research presented to the committee. We do appreciate that. We'll provide you with a Hansard transcript of today's proceedings. If there are any corrections or additional information you'd like to provide, you can do that through the committee secretariat by 11 June. Thank you for your time.

Dr Tuffaha : Thank you very much.

Proceedings suspended from 14:19 to 14:34