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REAL WORLD EVIDENCE (RWE) IN CLINICAL TRIALS – DOCUMENTATION FOR MARKETING AUTHORISATION AND POLISH REQUIREMENTS

Real World Data (RWD) is data collected in the real world about patient health and healthcare delivery. This data can be collected routinely from a variety of sources including electronic health records, disease registries or patient generated and uploaded data. RWD allows for the collection of more data from actual users of medicinal substances. Real World Evidence (RWE) is clinical evidence that has been carried out on the basis of RWD analysis. They concern the benefits or risks of using a medicinal product. RWE is obtained from various studies and analyses such as randomised trials, including large simple trials, pragmatic trials or observational studies. They can be used to determine what action should be taken to reduce the risks and increase the benefits of a medicine for therapeutic purposes. RWE has been defined in Sec. 3022 (2) of the 21st Century Cures Act: ‘real world evidence’ means data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials. Clinical trials are now at the heart of the process of bringing medicinal products and medical devices into use. For the most part, they are conducted even before a product is placed on the market, but the final, fourth phase takes place after the product is marketed. This phase consists of determining whether the marketed product is safe in all manufacturer’s indications and for all patient groups. Conclusions from previous phases are further verified in this phase. In the earlier phases, studies are conducted on a group of volunteers on whom the effects of the substance are tested. In the final phase, information is collected on adverse drug effects that have been reported. In this aspect, RWE and clinical trials are similar. However, there is one important difference that weighs in favour of RWE. In the case of clinical trials, data are collected only from patients who have reported adverse effects and the information about them has reached the manufacturer. RWD can be collected from all patients who reported an adverse reaction to a medicine and the adverse reaction was reported in their medical records. The number of people from whom data on the effects of medicines are derived is therefore much larger than in the case of clinical trials. Manufacturers of medicinal products and medical devices use RWD and RWE to support clinical trials, especially their final phase. They are therefore not mutually exclusive modes of research, but rather designed to support each other.

Necessary documentation for marketing authorization

Introduction of a medicinal product or medical device on the Polish market requires authorization from state or EU authorities. Depending on whether the medicinal product is to be used only in Poland or also in other EU countries, there are several pathways to obtain authorisation for registration of a medicine: national procedure, Mutual Recognition Procedure (MRP), decentralised procedure (DCP), central procedure (CP) and parallel import. The first one is applied when a medicine is to be used only in Poland. Decision on marketing of such medicinal product is issued by the President of the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products within 210 days on the basis of documentation submitted by the applicant, which should include an application of the applicant for marketing authorization of the medicinal product and the relevant documentation with information forms. MRP takes place when the medicinal product has been placed on the market on the territory of another EU state and consists in transposing the authorisation to the Polish law. For this to happen, it is necessary for the Polish authority to issue a decision and for the registrant to submit documents in Polish, including information for consumers. The decentralised procedure looks similar, but the medicinal product has not yet been authorised for marketing, but only registration applications have been submitted in several states. Such a procedure takes 210 days and ends with the Polish authority issuing a decision on the basis of the submitted documentation and the assessment report of the reference country (RMS) indicated by the registrant. In the centralized procedure the decision to allow a medicine to be marketed in EFTA and EU is taken by the European Commission on the basis of the scientific opinion of the Committee for Medicinal Products for Human Use (CHMP) after examination of the application by the European Medicines Agency (EMA). Parallel import concerns medicines, which are authorized for marketing in Poland and other EU countries and are imported through the free movement of goods within the EU. The parallel import license is issued similarly to the decentralized procedure. Polish law distinguishes a medicinal product from a medical device. A medicinal product is intended to treat and prevent disease, whereas a medical device is intended to assist in the treatment and prevention of disease. Medical devices are therefore merely ‘facilitators’ of medicinal products. They are also subject to a different marketing authorisation procedure from medicinal products. The first step in registering a medical device is to assess the device’s compliance with the essential requirements. If the medical device is non-sterile and without a measuring function, the manufacturer conducts this assessment itself, declaring conformity and marking it with the CE mark. If the product must have a higher risk class, the assessment is carried out by a body notified by the Minister of Health. In addition, the product should have a labelling and instructions for the customer in Polish. A medical device that has the required certificates must be notified to the President of the Office for Registration of Medicinal Products, Medical Devices and Biocidal Products within 14 days. The above-mentioned registration paths also result from various acts, as the provisions concerning medicinal products are contained in the Act of 6 September 2001 of the Polish Pharmaceutical Law (Journal of Laws of 2008 No. 45, item 271 with subsequent amendments) and regulations and concerning medical devices in the Act of 20 May 2010 on medical devices (Journal of Laws of 2015, item 876 with subsequent amendments). It is also important that in order to introduce a medicinal product on the Polish market, it is necessary to conduct clinical trials before obtaining the decision mentioned above. However, such an obligation does not apply to medical devices. Consequently, with regard to medicinal products, under Polish law it is not possible to completely abandon clinical trials, but RWD and RWE may support them in their final phase or replace them in this phase only. In the case of medical devices, the collection of RWD is not necessary for registration and subsequent marketing. Of course, RWD and RWE can only be used for the registration of a medicinal product if they come from another country where the product is already on the market.

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Advantages of RWD and RWE

The undoubted advantages of real world data and evidence are the low cost of obtaining them and the faster generation of data, as it is sufficient to have access to anonymised databases. Thanks to the wide circle in which data are collected, it is possible to detect less frequent adverse effects and a greater ability to capture information that may affect health outcomes. The advantages of using RWD and RWE have already been recognised by the US authorities by including them in the 21st Century Cures Act. Additionally, the US Food and Drugs Administration (FDA) is already using RWD and RWE to monitor the post-marketing effects of using drug products in the pharmaceutical market.

Sources:

  1. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
  2. https://www.politykazdrowotna.com/53330,rwe-coraz-wieksza-rola-danych-z-rzeczywistej-praktyki-klinicznej
  3. https://www.biostat.com.pl/dane-rzeczywiste.php
  4. https://www.badaniaklinicznewpolsce.pl/o-badaniach-klinicznych/podstawowe-informacje/jak-sie-prowadzi-badania-kliniczne/
  5. https://przemyslfarmaceutyczny.pl/artykul/wytyczne-dla-produktow-leczniczych-stosowanych-razem-z-wyrobami-medycznymi
  6. https://www.gov.pl/web/zdrowie/pozwolenie-na-dopuszczenie-do-obrotu1
  7. http://www.urpl.gov.pl/pl/produkty-lecznicze/zagadnienia-rejestracyjne/nowa-rejestracja/procedura-narodowa
  8. https://www.gov.pl/web/zdrowie/wprowadzenie-wyrobow-medycznych-do-obrotu-i-do-uzywania
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