Real-World Data and Real-World Evidence: Current status in the APAC region (Focus: Australia)
The use of real-world data (RWD) to supplement randomized clinical trials has been a growing trend recently. Several companies started using RWD to make informed pricing and reimbursement decisions and are also exploring other ways to use RWD in developing their clinical pipeline. At the same time, several countries are deliberating on how to employ real-world evidence (RWE) in their drug approval process.
... Australia or anywhere else the biggest problem now is noise in the data.
Obviously, the rate of adoption of RWD varies across countries. Countries like the US embraced it and is currently working on a framework to integrate the use of RWD in their drug approval pathway. In other countries like Brazil, issues with data standardization and privacy restrict the potential for the use of RWD.
We, being an APAC focused consulting organization, naturally sought out to understand the status of the usage of RWD in the Asia Pacific region. With this objective, our team connected with KOLs across different countries in the APAC region. We aim to post the key intercepts from our team’s discussion with KOLs in this new interview series.
In the first interview post, our team members, Raja Mukesh Dokala, Senior Manager and Khusnuma Begum, Senior Analyst discussed the current status of RWD in Australia with Prof Dinesh K Kumar, member of The Advisory Committee on Medical Devices (ACMD) which advises the Therapeutic Goods Administration (TGA), Australia.
Prof Dinesh K Kumar
Professor of Biomedical, RMIT University, Melbourne Member of The Advisory Committee on Medical Devices (ACMD), Therapeutic Goods Administration (TGA), Australia
Considering the rapidly transforming healthcare space, what are your views on the usage of Real-World Data (RWD) in healthcare decision making?
The software used for data analysis now can identify errors in large data recordings. So, all data which might not be considered earlier due to errors is available now.
Also, the collection of data has become easier because everything could be recorded in some or other way like iPhone, iWatch or Fitbit or other similar devices. It is easy to record the data at a low cost.
One big advantage of RWD data comes in from its role in cost assessment for medical device or pharmaceutical product. Based on my personal experience from a project which I am running in collaboration with two of the premium institutes back in India, where we are recording a large amount of data of people, like their mobility, diet, size, etc. Using this data, we identify specific traits of the people for certain interventions for the treatment of diabetes and cardiovascular diseases.
There are two levels of dealing with the real -world data - (1) from the viewpoint of statutory bodies such as Therapeutic Goods Administration (TGA) in costing and reimbursement decisions; and (2) a pharma or a medical device company to use RWD in new product development.
With the introduction of the 21st Century Cures Act in 2016, USFDA has an additional focus on using Real World Evidence (RWE) in regulatory decision making. Coming to Australia, how is the regulatory landscape evolving in terms of usage of RWE?
The USFDA has come up with draft guidance for the industry regarding the process to submit RWD and RWE to FDA for drugs and biologics. I think this is the procedure document which has not yet been accepted but is like a white paper which is open for discussion. It might take a couple of years from this point to publish the guidelines and put them to use.
In Australia, the Pharmaceutical Benefits Scheme (PBS), which is under TGA, is taking RWE more seriously. However, I think the pharma companies who have been using RWE for a while are the ones to use it more aggressively because they can identify things proactively and deal with them before a real issue arises. These companies are going to drive the usage of RWE.
How are the key stakeholders of the healthcare value chain responding to the use of real-world evidence in decision making?
I think there are many discrepancies, some people don't completely understand the use of RWD, some consider this to be useless, and some use it as a means of revenue generation.
If you look at alternative scenarios, the entire model of Facebook until recent times is data collection. The same thing is happening in the medical world, as well. Some people provide you with free online services, but they collect your data.
For example, if someone is are going to get his/her cholesterol tested to know about their risk of cardiovascular disease. The service provider records this person's data and provides him/her the test for free or at a discounted cost. The data collected is marketed to whoever can use it. Of course, what is done with the data collected, the data confidentiality and other legal parts must be taken care of beforehand.
What are the common sources of RWD?
As discussed already, it can be from your day-to-day devices, health records, longitudinal studies, etc. Nearly all of them are voluntary. One example is the company which provided expensive software for a clinic to record the patients, to help them manage patients, but at the back-end data is being stored and saved. The data is used either for research or commercial outcomes. Some examples are the Framingham Heart Study from the US, which lead to the development of the Framingham Risk Score for estimating cardiovascular risk; the Blue Mountains Eye Study aimed to assess the progression of age-related macular degeneration in Australia. A recent example is a longitudinal study from the University of Canberra, Personality & Total Health (PATH) Through Life project, which examining the risk factors and relationship between depression and other psychological disorders.
Are there any specific indications or areas in which RWD is utilized more in Australia?
In Australia, there is more focus on Ophthalmology, especially for retinopathy. Centre for Eye Research Australia (CERA) has conducted one of the largest studies. The Rotary Club of Australia also conducted a similar Ophthalmological study. These are some of the big studies in Ophthalmology that I know, but that does not mean that these are the only ones. Some of them are non-commercial research, but many commercial pieces of research have been happening for a while
What according to you, are the key barriers for the use of real-world data in Australia? Australia or anywhere else the biggest problem now is noise in the data. One of the factors is that you do not know the source, where the data is from?. It is the same as the current media landscape where anyone and everyone can upload some information, and there is no way to identify whether that data is fake or real. So, that is a big problem, and to overcome, we must start reducing freedom, but that becomes a challenge. In some worst cases, people corrupt data of the competitor companies as they can go and load some junk data, and make it difficult to clean it later.
The huge supporting factor here is that the data collected from huge populations, and it is also inexpensive to collect. However, the problem is that we need people who can somehow identify the errors in the data, so people are not building algorithms to create data and it comes under a different category of thing, called as large data analysis or noise capture.
In terms of the publicly available data or government databases like electronic health records or any other insurance data from the government, to use them to generate RWE is data privacy an issue?
Of course, it is, unless in Australia we have much stricter than I think in many other countries. If someone intends to use such data, then he/she must disclose accurately, what is the data used for before collecting it.
So, data privacy in a way is a barrier to use of RWD now?
Yes, and the only way you can do it properly is to formulate a proper strategy of collecting the data by providing some information transparently to the patient. For example, if someone at risk of cardiovascular disease goes to a website wherein health-related data is collected, then there should be a clear disclaimer about the data collection before the person can enter the website. Consent must be taken for recording the data for the mentioned purposes and make sure that the person is comfortable to share it.
Pharma companies and medical device companies who want to collect this data, in the long run, must implement certain technical capabilities or driven the systems in place, in such scenarios do you think the cost is a barrier?
For a Pharma company that kind of cost which we are talking about should not be a barrier, we are talking about companies worth billions of dollars. Data is being collected using Fitbit now, and the device is recording your movement in GPS, and you are giving the data away for free. So, if Fitbit wants to use that data to identify people of Indian ethnicity of a specific size and shape, they can use the data taken from its users. They might develop methods to improve one's health, which is required, but privacy is not there. They can now take the data and use the data however, they want.
What is the outlook for the usage of Real-World Data (RWD) in the future in Australia in terms of regulatory approvals or reimbursement decisions?
It is going to happen for sure, and I think it will be the pharma companies that are going to lead because it is in their interest to use the real-world data to customize and individualize the medicines. Everyone’s record could be tracked and recommend the drugs and diagnostics that specifically suit that individual. It is not going to happen tomorrow, but it is not going to be like this there forever. Several factors are involved, and I think the statutory bodies must be more cautious and so will take some time.