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RWE in Pharma
The term real-world evidence (RWE) has generally referred to the key conclusions that could be derived from published studies in peer-reviewed journals. The information in these published studies comes from independent and company-sponsored research performed within targeted populations that often range from a few hundred to a few thousand patients. The process usually takes place over several years, and encompasses study concept, study funding, study execution, the summarization of results, publication of the information, and the definition of guidelines.
Traditional evidence has multiple uses today. Leading academic centers harness evidence and their own data to develop specific treatment approaches, pathways and order sets -standardized lists of treatment steps, diagnostics and therapeutics for a specific diagnosis - to make consistent and high quality treatment decisions. Health insurers and payers use guidelines and evidence as the basis for determining whether to support patient access and reimbursement coverage. Pharmaceutical, biopharmaceutical, and medical device companies need to constantly consult these studies to assess and determine their own strategies for undertaking follow-on studies, creating value dossiers and for medical communications.
On the other hand, real-world evidence is now becoming the foundation for new pricing strategies which are more explicitly linked to therapeutic value for patients and health outcomes benefits to health systems and risk-bearers. This evidence provides the support and confidence needed to undertake value-based contracting, deploy patient services, and to run the emerging new patient care management businesses that best manage care processes and resource utilization to optimize value.
In conclusion, real-world data and advanced analytics are changing how clinical trials are designed, how medical affairs experts may identify the ‘long responders’ to specific treatment approaches, and how commercial organizations are evaluating the effectiveness of patient services programs. Increasingly, sources of real-world data provide a focus on the total population within a region or country, encompassing the various questions and insights that distribution data or chart reviews may have provided in the past.
Hi Janice - I have talked to many people these past few weeks about this question and some the answers were involving evidence collected from pre-selected cohort studies, medical records, data from Insurance claims, social media forums, and mobility apps. This data can be used for RWE in pharma for drug toxicity, adverse events, efficacy. I was interested in other peoples take on this topic.
Let me start with a necessary step, defining RWE - I take it you mean Real-World Evidence.
The term may be new, but the activity is not. As I see it, it has been around under another term - Post Market-Approval Surveyance - for decades. In my direct experience, that activity monitors and deals with product-quality issues, side-effects, efficacy, etc., and reports it on a regular, scheduled basis to the authority, in this case the FDA.
What does RWE do?
Using a definition I have come across, it "provides significant insight into how a drug or drug class performs or is used in real-world medical settings". So in a way it does the same as post-market surveyance.
What may have changed is the process that quickly transforms real-world data sources such as claims data or electronic medical records (EMR) into evidence. I have no evidence that this actually goes on and in consequence improves health outcomes for patients. If it does, all is well.
Does the effort help biopharmaceutical companies develop better therapies more quickly? l doubt it; and if there is an effect in this direction, I expect it to be marginal. Meaningful improvements of this type are more likely based on new findings generated by basic research.
The real difference today, as compared to say 20 years ago, is our ability to compile data more quickly, and analyze the data more thouroughly. However, giving this activity a new name makes little difference.
In my experience, the key difference between RWE (also RWD for real world data) and clinical trials is the lack of control. Clinical trials control for every variable from demography to disease and treatment history. Patients check in and are expected to complete diaries that indicate proper usage of the drug at a set dose and use pattern.
In the real world, the drug is taken by patients often very differently than prescribed (eg, poor compliance), in conjunction with other meds that were exclusion criteria in the trial (so there may be DDIs - drug/drug interactions or safety issues), doctors prescribe different doses or use the drug in a different place in the algorithm than indicated in the trial (eg, first line or add on therapy).
Thus the concept of RWE is to see how the drug REALLY performs in a real world scenario. Often drug registries are used for this purpose.
I have been involved with the development of "botanical drugs", a specific drug regulatory classification (ie. multicomponent botanical mixtures based on traditional medicines). Here the FDA does consider RWE in determining where clinical development may begin.
As the definition of RWE's use in Pharma has been provided here, I wil expand on why pharma relies on RWD from RWE studies. RWE studies draw on RWD from real life practice, including data obtained from insurance claims, patient registries, or electronic health records. These data are more likely to reflect the experience of heterogeneous groups who may not have been previously engaged in RCTs. However, the limitations of RWD should not discounted. These include: biased data based on physician's preferences.For example, physicians alter the description of the disease to meet insurance coverage requirements, and do not disclose errors. FDA has been made aware of these considerations as they evaluate the quality of RWE to support market approvals.
I would argue RWE is not used by Pharma much. Although- I'm not in the biotech industry. I am however- part of the patient centerdness movement and an Ambassador fot the Patient Centered Outcomes Research Institute. What is incredibly inportant in ALL medical treatments is that patients have access to info on various treatments and be able to weigh all options and select the best one FOR THEM. It's changing- but health care is generally about avoiding death and mitigating "bad" things. Drug trials and things are already extremely pricey without increasing sizes of trial patient panels and things. To use a clear example- still today, a lot of surgeries result in loss of continence and loss of sexual function. Depending on the specific prostate issue- some surgeons can avoid these effects. Regardless- risks and impacts for the most part are not clearly explained to patients before surgery. Risks and benefits of different treatments are not explained. RWE vary great. Same is true for pharma. lots of work to be done!
And here is another idea.
Phase 0 to Phase 4 clinical studies are typically tightly controlled.
For getting real RWE data, why not set up and run Phase 5 clinical studies?
Design: follow dosing recommendations as per package insert.
Data: as recorded by patients (e.g., by filling a simple template to document compliance or a lack of).
Data: drug effects and side effects as reported by patients.
Clinical input: periodic scheduled medical exams to monitor overall outcomes (with test depending on indication & drug).