Who are the stakeholders that can benefit from this?

Most stakeholders will benefit from the advantages of RWD/E that were outlined previously. Not only pharmaceutical companies and investors but also the general public and more importantly people with rare diseases such as those belonging to the CDG community. Moreover, depending on the kind of stakeholder, RWD can be used in specific ways, which we will address following.


Initially, regulators like the FDA and EMA have only utilized RWE for post-market safety and benefit/risk studies (10). From the last 10 years, the usage of Real World Data has been rising in the clinical world. Regulatory initiatives are not being left unnoticed. The FDA with the 21st Care Act (11) and their issued Framework (1) regarding RWE usage clearly puts RWE as a key enabler for regulatory decisions and market approvals (12). Nowadays, worldwide regulatory agencies most of the time still apply the gold standard RCTs and RWE is only used as a complementary source of information such as the epidemiology of a disease, individual-reported outcomes, etc. This complementary data provides novel insight on the possible outcomes in the performance of a novel disease (13). Moreover, as it was stated before RCT are not feasible in rare diseases clinical trials; therefore they might be a course of action to replace an unsuitable method.


The main factor that RWE has for the manufacturers is the clinical trials optimization as a way to accelerate the market access and pharmacovigilance. This optimization is due to a better understanding of the therapeutic unmet needs and the better understanding of diseases like CDG pathways in our biological system (10). It also allows investment in safety, and clinical effectiveness as well as helping in the modeling of economics towards a better prosper of the industry in question. Although, there are a few conditions that have to be met, such as the increase of investment in digital health infrastructure and the consensus on quality and reliability of data sources. It is also imperative to invest in new approaches towards the generation of evidence in earlier stages of the technology life cycle. One example of a project that works towards these goals is the EFPIA (European Federation of Pharmaceutical Industries and Associations) oncology health data mapping (13).

Healthcare providers

Healthcare providers can use RWD to leverage the use of technology in clinical practice (13). This will aid to “increase” their knowledge on different diseases such as CDG due to the accumulation of different aspects of a person's health status. In addition, they will be able to give more effective treatments, in terms of the drug dosage and specific pathways; so their clinical decisions overall are going to be optimised because of the growth in the implementation of evidence-based methodologies. All these RWD/E applications are going towards a more equal health access (10). 


RWD/E will lower the general cost of the trials like CDG, which are a growing area in the clinical spectrum nowadays. This is mainly because of the overall optimization of clinical trials (10): reduction of clinical trials duration, creation of external comparators in order to qualify drugs’ efficiency, cost per participant, and so on (13). RWE also enables insights and decisions for personalized reimbursement models based on usage, value and outcome. According to the payers perspective, this evidence is likewise important in safety monitoring as well as to conduct utilization management (14). Despite this usefulness, some payers also believe that this evidence is not suited to be used in pharmaceutical decision making since it has few opportunities to be applied and because RCTs pose a valuable and proven way of researching and testing orphan drugs (14). 

People living with a certain disease

RWD/E will allow people living with a certain condition to share their own real world experience and as a result raise more data openness and availability regarding their disease (13). Therefore, this permits to evaluate their unmet needs and potential outcomes if they are participants of an orphan drug research. This will set towards a more personalized medicine; so it will improve a CDG carrier lifestyle (10).


For a better understanding, the following videos are available:

  • The youtube channel OBRoncolog has developed a video named “ Real-World Evidence Compared to Randomized Clinical Trials” that is ideal for anyone who’s trying to understand the difference between RCT and RWE.
    Watch it below
  • The youtube channel AJMCtv from the American Journal of Managed Care has developed a video has developed a video called “Dr Jennifer Graff on Using Real-World Evidence to Deliver High-Quality Care” that is ideal for anyone who’s trying to understand the advantages of RWE in drug development
    Watch it below
  • The youtube channel AJMCtv from the American Journal of Managed Care has developed a video “ Dr Nicholas Robert Explains the Benefits, Challenges of Using RWE for Measuring Clinical End Points“ that is ideal to everyone who wants to understand the usefulness of RWD.
    Watch it here
  • The Video Journal of Biomedicine has developed a video that is a interview to David Thompson, PhD is Senior Vice President, Real World Evidence Advisory at Syneos Health about RWE. The video is entitled “Real-world evidence generation with David Thompson '' that is ideal for someone who wants to have a broad perspective of RWE, such as its benifits, regulatories agencies and so on.
    Watch it here



  1. https://www.fda.gov/media/120060/download Accessed March 2021
  2. Polak TB, van Rosmalen J, Uyl – de Groot CA.(2020) Expanded Access as a source of real-world data: An overview of FDA and EMA approvals. Br J Clin Pharmacol.86:1819–1826. https://doi.org/10.1111/bcp.14284
  3. Rudrapatna, V. A., & Butte, A. J. (2020). Opportunities and challenges in using real-world data for health care. Journal of Clinical Investigation, 130(2), 565–574. https://doi.org/10.1172/jci129197
  4.  Wu J, Wang C, Toh S, Pisa FE, Bauer L.(2020) Use of real-world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions. Pharmacoepidemiol Drug Saf.1–6. https://doi.org/10.1002/pds.4962
  5. https://www.lexjansen.com/pharmasug/2019/RW/PharmaSUG-2019-RW-345.pdf Accessed March 2021
  6. Tsutsumi, Shuntaro, et al. (2019). “Real-World Clinical Course of HTLV-1-Associated Myelopathy/Tropical Spastic Paraparesis (HAM/TSP) in Japan.” Orphanet Journal of Rare Diseases, vol. 14, no. 1, doi:10.1186/s13023-019-1212-4.
  7. https://graticule.life/2020/08/17/thoughts-on-advanced-real-world-data-and-rare-diseases/ Acessed March 2021
  8. https://www.fagg-afmps.be/sites/default/files/content/faq_1.5_20190726.pdf  Accessed March 2021
  9. https://www.nice.org.uk/news/press-and-media/nice-draft-guidance-conditionally-recommends-elosulfase-alfa-vimizim-for-treatment-of-very-rare-life-limiting-genetic-disorder Acessed April 2021
  10. https://www.dxc.technology/healthcare/insights/146938-how_real_world_evidence_transforms_the_entire_healthcare_ecosystem Acessed April 2021
  11. https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act Accessed April 2021
  12. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence Accessed April 2021
  13. Facey, K., Rannanheimo, P., Batchelor, L., Borchardt, M., & De Cock, J. (2020). Real-world evidence to support Payer/HTA decisions about highly innovative technologies in the EU—actions for stakeholders. International Journal of Technology Assessment in Health Care, 36(4), 459-468. doi:10.1017/S026646232000063X
  14. Malone DC, Brown M, Hurwitz JT, Peters L, Graff JS. Real-World Evidence: Useful in the Real World of US Payer Decision Making? How? When? And What Studies? Value Health. 2018 (3):326-333. doi: 10.1016/j.jval.2017.08.3013. Epub 2017 Oct 18. PMID: 29566840.
  15. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence Accessed April 2021

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Alexandre Gil and Pedro Granjo from Sci and Volunteer Program Nova School of Science and Technology 2021.


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