Clinical vs real world data

Dr Kathryn Taylor-Barnes considers the importance of real world studies and how they differ from formal clinical trials

For the majority of the past two decades, while practising as an aesthetic medicine specialist, I have only had three toxins to use for my patients. It was exciting and somewhat daunting when suddenly three more toxins were released in the UK. This certainly made many of my colleagues and I inquisitive about how these newbies differed and what uniqueness they each brought to the table. In my clinic, I get to know a product by immersing myself in its capabilities; in doing so, I can decide if it delivers what is expected. I can then safely and confidently embrace it as a solid addition to my treatment toolbox.

When a new product becomes available in the UK, I learn about it through attending conferences, reading industry publications, via social media, web browsing, networking with colleagues, and visits from company representatives in my clinic. Toxin injection treatments are a relatively large part of my practice. When I heard about the influx of newer toxins into the UK, I did not want to bring them all into my clinic simultaneously, as that would likely confuse me. I realised that a comment I kept hearing from my patients was ‘I would like the treatment to start working quickly’ and ‘I want to look natural’. With that in mind, I felt the product from the USA-based performance beauty company Evolus, based in California, would be an excellent place to start. Its toxin is prabotulinum A, and clinical trials carried out in the USA and Canada specifically claim that the product did precisely this - act faster and produce a natural-looking result. Hence, my interest was piqued!

I wanted to dive in but felt that there had not yet been any UK-generated data to substantiate the formal US and Canadian trials on the toxin, so I was not convinced it would sit as well with the UK patient population; after all, the folks over the pond are pretty different! Also, the three toxins I have used for many years are produced by well-established, highly regarded pharma companies with proven track records and strong confidence among my colleagues. Taking a product into my clinic to be used by many of my patients needed me to be curious yet equally courageous, especially as the company behind this new toxin was utterly new to the UK and had a definite ‘newbie’ stance. I didn’t have the reassurance of turning to my UK colleagues to ask their opinion on their experience with it because no one had gained much experience with it over here at that stage.

I then decided to bring it into my clinic, and common sense told me that, in order to test the company claims, I would have to gain feedback from my patients and replicate the endpoints investigated in the formal clinical trials to date. I would have to do this as a ‘real world’ study.

THE IMPORTANCE OF REAL WORLD STUDIES

Real world studies have become increasingly important in providing evidence of treatment effectiveness in clinical practice. While randomised clinical trials (RCTs) are the ‘‘gold standard’’ for evaluating the safety and efficacy of new therapeutic agents, necessarily strict inclusion and exclusion criteria mean that trial populations are often not representative of the patient populations encountered in clinical practice.1

RCT exclusion criteria may rule out a significant proportion of real world patients. Patients excluded from RCTs are older, have more medical comorbidities, and have more challenging social and demographic issues than those included in these trials. Real world studies have the potential to assess whether results seen in RCTs would be generalisable to real world patient populations.1

Real world studies provide important information that can complement and/or even expand the information obtained in RCTs.1

Dr Kathryn’s patients in this study. Copyright Dr KTaylor-Barnes 2024

• Data gathered from a single clinic setting will remove the variable of the nuances of patient demographics influencing the results seen in RCT trials.

• Data generated from a single injector will remove the distinct impact on the results the RCT trials often cannot avoid when data is pooled from different clinics and different injectors in order to get favourable patient numbers through.

• Patients included in RCT trials will be having their treatments at zero cost as they are pharma sponsored, which creates significant bias that is wholly removed in a real world study such as the one discussed here, where all patients paid fully for their treatment with no discounts to incentivise their questionnaire answers and product feedback.

ADVANTAGES OF REAL WORLD DATA TO THE PRACTITIONER AND PATIENT

Real world data gives the practitioner the necessary information to relay to a patient who is in the delicate stage of deciding whether to take the chance of having a treatment which differs from their usual one. In my practice, the patient who is going to take the leap with a new clinic offering will usually ask if I myself have had it before. It’s not always feasible to say ‘yes’ to that one. Having robust and yet easily relatable data pertaining to the country in which the clinic is located is invaluable in helping that patient make a more informed decision.

OUTLINE OF EVOLUS PRABOTULINUM TOXINA (NUCEIVA) DATA

I carried out my research as a data collection exercise over three months as a single centre, single injector, having treated 457 patients with this toxin in my clinic. The patients were emailed a feedback questionnaire within two weeks of having their treatment. No patients were incentivised to participate. A 56% response rate [254 patients] returned the completed questionnaire. The mean age of respondents was 49 years. They were asked 12 questions, replicating those asked in the RCTs, as well as extra ones I included, regarding product preference feedback and psychological effects from having toxin treatment.

RESULTS

• 60% noticed a treatment effect by day three

• 34% reached peak effect by day seven

• 46% selected natural looking as a key treatment outcome

• 67% reported no adverse events

• 83% were satisfied with treatment

• 85% reported positive psychological benefit to having this toxin treatment

CONCLUSION

This piece of wholly independent research I have carried out is the first real world data on prabotulinum toxin A generated outside the USA. The patient group analysed was of a considerable size with data gathered from just one centre. I am hopeful that it will stimulate curiosity among my colleagues and give them the reassurance to try this toxin in their individual practices. I know that if I had been presented with this type of data at the start of my journey, I would have been more confident in discussing the product with my patients. I feel that, as aesthetic medicine practitioners, we should not be frightened to question the claims of clinical trial data and how it can be brought to reality in our own patient cohorts. I have certainly noticed that my patients have been further reassured by me taking steps personally to do a deep dive and generate my own data, which has then gone on to be published in a peer-reviewed scientific journal. There is a good feeling when you start the conversation with ‘this is what I have gathered from my own clinical data’. Being a UK ‘first’ is a bonus too!