Clinical trials constitute medical experiments on human subjects, the objective of which is to prove efficacy and safety of use of a given medicinal product. The procedure of conducting clinical trials in Europe is strictly controlled by regulatory bodies designated for this purpose. In Poland these agencies are: The Office for Registration of Medicinal Products, Medical Devices and Biocidal Products and relevant bioethical committees. Clinical trials sponsored by MABION S.A. are conducted in line with carefully specified rules and local executive and legislative acts of law. The above-mentioned institutions are obliged to provide constant oversight of ongoing trials and issue opinions on newly submitted applications. Actions of these institutions aim at protecting the rights and welfare of patients who decide to participate in medical experiments. Each clinical trial should be conducted in accordance with Good Clinical Practice (GCP) which constitutes a set of international standards in medicine, ethics and science applied to planning, carrying out, monitoring, and documenting trials conducted on human subjects and presenting their results. Compliance with these standards guarantees credibility and authenticity of collected data and protection of the trial participants' rights. Institutions such as the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) are responsible in the European Union and the United States, respectively, for material evaluation and monitoring of safety of medicinal products which are developed by pharmaceutical, biotechnological, and biopharmaceutical companies.
Medicinal products under ongoing clinical trials constitute a form of experimental therapy which has not yet been granted marketing authorisation. Efficacy, quality and safety of medicinal products which are subject to clinical trials are each time confirmed in a series of basic and pre-clinical trials. Basic research and the pre-clinical phase include laboratory tests and animal testing, respectively. Clinical trials sponsored by MABION S.A. are conducted in research centres and carried out by highly qualified medical staff who provide professional healthcare to participants. The clinical phase begins only after the pre-clinical phase is concluded with a positive result and there is a high likelihood that the benefits resulting from the use of the Investigational Medicinal Product by humans significantly outweigh the risk associated with its use. The Principal Investigator (PI) plays a major role in successful conducting of clinical trials. PIs are responsible for taking medical decisions with regard to patients enrolled into the trial, ensuring their safety and supervising the implementation of the trial, as well as ensuring that its course complies with the Good Clinical Practice guidelines, the trial protocol and the provisions of applicable local laws. Principal Investigators have the option of delegating some of their obligations to the medical staff constituting the Research Team. The success of any clinical trial depends to a great extent on cooperation between the Patient, the Sponsor and the members of the Research Team.
THE OBJECTIVE AND SCOPE OF MABION S.A.'S CLINICAL ACTIVITY
MABION S.A. is a leading biotechnology company which dedicates its activity to conducting multicentre clinical trials until phase III. Currently we are implementing projects in Central and Eastern Europe, in 11 countries and nearly 100 health centres. The high standard of technology developed and owned by MABION S.A. allows for manufacturing of cutting-edge biological drugs based on monoclonal antibodies. Commencing of the marketing authorisation procedure depends on the ability to demonstrate bio-equivalence between products developed by MABION S.A. and the reference product. Actions taken by MABION S.A. offer significant chances for improving the quality of life of patients, thus ensuring greater access to biotechnological drugs. Adequately designed trials, a highly educated team and constant, objective evaluation of the course and quality of ongoing projects guarantee meeting the anticipated goals.
POTENTIAL BENEFITS RESULTING FROM PARTICIPATION IN A CLINICAL TRIAL
MABION S.A. is a leading biotechnological company which focuses on the development of medicinal products which are used in the treatment of advanced oncology and autoimmune disorders. Clinical trials sponsored by MABION S.A. are conducted in Central and Eastern Europe at the highest international level, in compliance with the same high standards in all countries. All actions are carried out in accordance with the protocol, internal procedures introduced by MABION S.A. and in compliance with the provisions of local and international law. Additionally, MABION S.A. has successfully completed a joint Scientific Advice procedure in the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) in terms of qualitative and clinical development of MabionCD20. The data resulting from actions taken in connection with conducting clinical trials must constitute a reliable assessment of efficacy and safety of use of the new drug, which in turn should serve as a solid basis for completing the marketing authorisation procedure in the future. The wide range of potential benefits which may result from participating in clinical trials sponsored by Mabion S.A. include in particular:
- THE POSSIBILITY OF USING A TREATMENT WHICH IS UNAVAILABLE OR DIFFICULT TO ACCESS ON THE MARKET
By getting access to a treatment which was previously unavailable to the patient, he or she gains a chance for a new and better effect of the applied therapy. Medicinal products which are subject to clinical trials sponsored by Mabion S.A. are characterised by huge potential for becoming an effective alternative to treatment in many disorders.
- NO COST FOR THE PATIENT
The patient does not incur costs related to treatment and diagnostics. All expenditure associated with participation in a clinical trial sponsored by Mabion S.A. which are incurred by the patient (e.g. travel expenses) can be refunded.
- TOP QUALITY OF MEDICAL CARE
Participation in a clinical trial sponsored by Mabion S.A. guarantees a thorough control of the patient's health prior to commencement of treatment with the Investigational Medicinal Product, in its course and after completed administration of the Investigational Medicinal Product. It allows for real-time control of the patient's health, and ongoing monitoring of both his or her safety and the treatment's results. Participation in clinical trials sponsored by MABION S.A. gives access to novel treatment methods as well as a team of world-class experts.
- PARTICIPATION IN DEVELOPMENT OF NEW PHARMACEUTICALS AND IMPROVEMENT OF PUBLIC HEALTH
Obtaining a marketing authorisation, accessing the market and maintaining wide availability of the drug is impossible without patients’ participation in clinical trials, even with high efficacy of the treatment. Each patient who takes part in a clinical trial partakes also in improving the level of public health.
Clinical data and the safety profile of MabionCD20 in patients with rheumatoid arthritis are constantly evaluated by an independent group of experts – the Data and Safety Monitoring Board. In line with procedures, members of the DSMB monitor the safety of patients and supervise the quality of trials. DSMB experts have confirmed that MabionCD20 is well tolerated both after the first administration and after the subsequent administrations. No differences in the safety profile of MabionCD20 have been observed in the analysed group of patients as compared to MabThera, the reference drug. Adverse effects occurring following administration of MabionCD20, the Investigational Medicinal Product, have a similar range and severity as the adverse effects characteristic of the reference drug therapy. No need to modify the trial protocol and procedures has been identified. In the opinion of DSMB, the trial conducted on RA patients can be successfully continued, thus offering a significant benefit to patients participating in the trial, and constituting significant support to the public health sector.
INSURANCE OF THE TRIAL
Every Sponsor is obliged to insure each patient participating in the medical experiment. In the case of an event which results in irreversible damage or injury, the patient has the right to compensation pursuant to applicable provisions of law.
HIGH-QUALITY COMPARATIVE TREATMENT
Even if as a result of randomisation, i.e. random allocation of patients to either the experimental or control group, the patient is allocated to the control group, he or she receives the reference drug and a guarantee of top level quality. An adequate comparator has high therapeutic value and despite the fact that it is a drug available on the market, it is often unavailable to patients, either due to high costs or the long waiting time.
SAFETY OF PATIENTS
- COMMENCEMENT OF THE CLINICAL TRIAL
Every clinical trial sponsored by MABION S.A. is conducted on the basis of a protocol which had been verified and approved by relevant authorities in every country in which the trial is conducted. Regulatory bodies are obliged to assess the merits, feasibility and the design of the clinical trial, as well as to analyse the expected benefits for the patient resulting from participation in the trial, as well as the risk associated with its conducting. A clinical trial can be commenced only after obtaining a positive opinion of experts regarding its merits.
- GCP – GOOD CLINICAL PRACTICE
The primary principle adopted by MABION S.A. is to conduct clinical trials in accordance with the Good Clinical Practice principles (GCP). It is an international ethical and scientific standard which guarantees protection of rights and safety of participants of clinical trials, as well as credibility and accuracy of the data obtained in the course of the trial, which then are subject to statistical analysis.
- GMP – GOOD MANUFACTURING PRACTICE
The Investigational Medicinal Product is manufactured and stored in line with Good Manufacturing Practice (GMP). It is an international standard which ensures the correct course of the medicinal product manufacturing process. GMP ensures repeatability and unification of manufacturing processes by way of strict supervision of the entire production process – starting from procurement of raw materials, through storage, production, packaging, labelling and ending with distribution of medicinal products. Implementation of GMP rules eliminates chance from production processes, thus determining the implementation of subsequent stages of the manufacturing process in line with strictly defined procedures and guaranteeing the highest quality of medicinal products.
- COMPETENCES OF INVESTIGATORS
In cooperation with the company CRO, MABION S.A. selects medical centres and Investigators who have significant expertise and experience, high qualifications and are well prepared and capable to adequately carry out the trial. This constitutes a guarantee that the project will be implemented reliably and effectively, thus providing the trial participants with adequate care.
As a pioneer in implementing innovativeness in Polish biotechnology, MABION was the first to develop a drug in the eukaryotic expression system, increased the scale of the drug's manufacturing from laboratory to industrial and implemented an up-scaled process for manufacturing in the GMP standard. Furthermore, for the first time in the Polish pharmaceutical market, MABION S.A. obtained authorisation to manufacture a biotechnological product in the monoclonal antibody class.
MABION S.A. PHARMACOVIGILANCE UNIT
All actions taken by MABION S.A. in connection with clinical trials aim at ensuring the highest ethical standards, while at the same time obtaining the highest level of scientific and clinical data. The MABION S.A. Pharmacovigilance Unit continues to take comprehensive actions aiming at identifying, assessing, learning about and preventing adverse effects and other potential problems related to the use of the Investigational Medicinal Product. In order to obtain the highest quality supervision over safety, MABION S.A. has established internal procedures which cover monitoring of the safety of treatment, reporting adverse effects, as well as procedures in the event of an unacceptable risk. MABION S.A. procedures are fully harmonised with applicable provisions of law on safety of patients in clinical trials. In cooperation with Investigators and the Medical Monitor, the PV unit provides, on a regular basis, reliable, rational, evidence-based information which constitutes a source of the drug's assessment with regard of its safety profile, thus constituting invaluable support to public health.
Confidentiality of data regarding participants of clinical trials sponsored by MABION S.A. is observed to the letter, in line with applicable provisions of law on personal data protection.
Due to the lack of complete data on safety, subjecting oneself to treatment with the Investigational Medicinal Product is always associated with a certain risk. However, the data collected so far suggest that the direct benefit to the patient significantly exceeds the potential risks. The ultimate goal of conducting trials sponsored by MABION S.A. is minimisation of the risk for the patient, both in the course of the trial and in the future, following marketing authorisation and marketing. The following issues need to be addressed with regard to each clinical trial:
- TREATMENT WITH ANY MEDICINAL PRODUCT, INCLUDING THOSE WITH MARKETING AUTHORISATION, IS ASSOCIATED WITH RISK
Potential adverse effects which might take place after the use of MabionCD20 can take place also following the use of the reference product. The collected clinical data allow for concluding with a great likelihood that the use of the treatment in question is as safe as in the case of the reference drug.
- INFORMED CONSENT OF THE PATIENT
Voluntary, informed and written consent is obtained from each patient participating in the trial before its commencement. Before deciding to participate in a clinical trial, the patient must fully understand the purpose of the trial, as well as the actions and medical procedures which must be carried out to achieve it. It is the doctor's role to explain to patients what their right and obligations are and to provide them with any information necessary to assess the relation of the potential benefits and the risk. At any time, in case of any questions or doubts, the patient can ask the doctor for additional consultations and explanations. Participation in clinical trials is voluntary and patient can withdraw from it at any time without stating their reasons. They also have the right to obtain up-to-date information which might impact their continued participation in the trial.
- RISK MINIMISATION
Not every patient with a given disease can be enrolled into a trial. Precisely selected inclusion and criteria aim at singling out those patients who will benefit most from the trial, at the same time minimising the risk of adverse effects. It is crucial to provide the doctor with all necessary information on the current health condition, history of the disorder, other medicinal products used, as well as all issues which might impact safety of the treatment.