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Real World Evidence

March 16th, 2018 - One Comment

We explore why observational studies matter and what role they play in the pharmaceutical industry.

Clinicians require a lot of supporting evidence-based data and well-designed studies to support prescription and patient management decisions.

Randomized Clinical Trials (RCTs) have been known as the ‘gold standard for medical research’ for a long time. Though RCTs are certainly crucial within the healthcare and pharmaceutical industry, in recent years, observational studies have become more and more important, due to the advances in statistical analysis and observational study methods. So, while RCTs are preferred to investigate the efficacy of new interventions, it is becoming clear that observational studies can complement and build upon the evidence and data procured from RCTs.

The following paragraphs cover what an observational study is, their different types, and why they are important, as well as what their advantages and drawbacks are.

What is an observational study?

Observational studies are a form of real-world evidence which explore how a given treatment would actually work and impact the daily life of a patient. These studies can be performed prospectively (going forward, data is collected on a group of participants) or retrospectively (the researcher mines existing databases and looks into the past to find data that is already collected).

Observational studies usually follow participants over a given period of time to investigate any and all associations between a patient’s treatments and certain health outcomes. This means that experts in the healthcare industry can uncover how treatments work in real world environments, so researchers can collect data based on different types of patients and a wide array of outcomes. Observational studies are also frequently conducted when RCTs are deemed either unfeasible or unethical.

There are several types of observational studies:

Longitudinal study — It requires repeat observations of the same variables over a long period of time

Cohort (or panel) study — It is a longitudinal study where a specific group of patients is closely monitored over a given period of time

Ecological study — An ecological study is an observational study wherein at least one variable is observed and measured at the group level

Case-control study — In this type of study, two existing groups (differing in outcome) are identified and then compared based on a particular attribute. Such a study can be remarkably important when it comes to identifying factors that may contribute towards a medical condition

Cross-sectional study — A cross-sectional study requires data collection from either a population, or a fitting subset, at a given moment in time

Why are Observational Studies performed?

Observational studies are used to better understand cause-and-effect relationships. They are useful in terms of real-world evidence and help understand how a drug or treatment impacts patients on a day-to-day basis. They are also helpful in times when an RCT would be a poor choice, such as in the following scenarios:

– An RCT isn’t always the right way to go in ethical terms. A commonly cited example is the relationship between breast cancer and abortion. If one were to investigate whether or not a causal link existed, an RCT would require dividing women randomly, with some participants receiving induced abortions and others not. Clearly, this would be highly unethical. To better understand this link, it would be more ethical to start with a group of female participants who already underwent abortions, comparing them to a group who had not

– The investigator of an RCT does not have the requisite influence to carry out a fair and accurate trial

– An RCT is impractical due to rarity. For example, if a researcher wanted to investigate the link between a given medication and haemophilia (a rare bleeding disorder), ethical considerations aside, an RCT would likely be impractical because the pool of participants is so small

What advantages do Randomized Control Trials (RCTs) have over observational studies?

Randomized Control Trials were considered the gold standard for a long time for good reasons. The main advantages of RCTs over observational studies are:

– RCTs provide better control over possible bias, confounders, and selection bias through the use of blind testing and randomization, i.e., it has high internal validity

– RCTs have well-understood standards of “Good General Practice”. There is a uniform industry standard that underpins this form of research — this does not exist for observational research. There have been attempts to address this over the years, but as of today, there remains no universal standard

– The pharmaceutical industry can be confident that results from RCTs will be trusted, while results from observational studies are occasionally viewed with skepticism

What are the main advantages of observational studies?

Sometimes, for a number of reasons, observational studies make the most sense for a given case. Here are a few reasons:

– Observational studies often play a significant role in investigating treatment outcomes, and providing information and clarification on the tolerability profile of a particular medicine

– One of the biggest strengths of observational studies is their proximity to real-life situations. RCTs have a very strict inclusion criteria along with rigid protocols, and as such, they might not reflect clinical practice. Observational studies, on the other hand, tend to have a greater heterogeneity of medical interventions, which are more true to clinical practice

– Observational studies are usually much cheaper than RCTs. Their designs are usually easily and quickly performed

– Observational studies can be used to investigate rare, unexpected outcomes that arise after taking a particular treatment. They can also be used to explore certain side-effects

– Observational studies provide a useful insight into healthcare interventions in the real world, which helps us gauge the gap between the efficacy and the effectiveness of a treatment. They can also be used to look at factors such as drug adherence and patient preferences

– Observational studies can be used to prove the validity of an RCT’s results, meaning that they are the perfect accompaniment to an RCT

Additionally, various meta-analyses have compared the results of RCTs and observational studies, and have shown that as long as the observational study has good methodological quality, the results are comparable.

There are clearly many advantages and benefits of observational studies, although they cannot be considered a replacement for a well-designed RCT. Observational studies play a key and, at times, a complementary role, in supporting and providing evidence for new drugs and therapies. Used together, these two forms of research studies can ensure that patient safety and care is always prioritized and at the forefront of our minds.

phamax is a research platform for scientific market access solutions. We build value for products through the use of real-world evidence and through HEOR studies. Get in touch with us today by emailing to find out how we can help you and your business.

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