For Patients

Learn how your participation in a clinical research study, offering active biologic medicine at no cost to you, could help expand access to more affordable biologic medicine for patients around the world.

Considering a biosimilar clinical research study?

Click on the “+” sign related to each question below to learn more, and then, if you are interested, talk to your doctor about the potential benefits and risks of participation.


What is a clinical research study?   +

All medications must be tested in clinical research studies before they can obtain regulatory approval and be marketed to the general public. Once a study demonstrates that a medication is safe and effective, a pharmaceutical company can apply to regulatory authorities, such as the US Food and Drug Administration (FDA) and European Medicines Agency (EMA), for approval to market their medication in a region or country.1–4 Until a medication is approved, it is considered an “investigational medication”.

Doctors conducting clinical research studies must follow a protocol, which is a detailed plan that clearly states which patients can participate and what procedures need to be followed to collect important information about the investigational medication. Most importantly, the protocol is designed to protect the safety of patients and must be approved by regulatory and ethical authorities in each country before the study can take place.

Study doctors must provide potential study participants with an Informed Consent Form, which explains the investigational medication, purpose of the study, risks and benefits of participation, and what to expect during the study. This form must be voluntarily signed by patients to confirm that they understand the study and willingly agree to participate. 
What is a biologic medicine?   +

To understand what a biosimilar is, we must start with biologic medicines and how they differ from chemical medicines.

Most medicines are made by combining chemical ingredients according to a “recipe”. Biologic medicines are different – they are made up of proteins and other substances produced by living cells. Biologics are much more complex and difficult to produce than chemical medicines.

Biologics are used to treat a number of life threatening and disabling diseases. They extend the lives of patients with certain cancers, reduce disability for patients with rheumatoid arthritis, and provide lifesaving replacement proteins for patients with rare diseases, such as hemophilia, who cannot produce these proteins themselves.

The challenge with biologics is their cost – because they are difficult to produce, they can be very expensive. Consequently, many patients around the world do not have access to biologics because they simply cannot afford them. 

Adapted from Kozlowski et al. 20115

What is a biosimilar?   +

When any new medication is approved for use in patients, only the original company is allowed to make and sell the medication until the patent expires (a patent is a license that gives the original company exclusive rights to make and sell the medication, preventing other companies from doing so). After the patent expires, other companies are permitted to make similar versions.

A biosimilar, as the name implies, is a similar version of an approved biologic medicine. Because biosimilars are copies of already approved biologics, they are less costly to develop than the original biologic, so the biosimilar can be sold at lower prices. And lower prices enable access to more patients around the world.

For a chemical medicine, the recipe can be duplicated fairly easily to make exact copies, called generics. For a biologic medicine, it is not possible to make exact copies because it is produced in living cells and the manufacturing process is complex. Therefore, biosimilar companies must aim to make medications that are “similar” to the original biologics, offering the same health and safety benefits.

Biosimilars are relatively new compared with generics, but they are gaining approvals in the major markets. In Europe, more than twenty biosimilars have been approved for eight different biologic medicines since 2006.6 Currently, no safety concerns have been reported in connection with biosimilars. 

Why are clinical studies needed to develop biosimilars?   +

All biologic medicines are produced in living cells, so we expect small differences between batches that are manufactured – this is true of the original biologics, as well as biosimilars. As a result, regulatory authorities, such as the FDA and EMA, require manufacturers to manage these differences within acceptable ranges.1–4

Because an exact copy of a biologic medicine cannot be made, regulatory authorities require that biosimilars undergo extensive testing in both the laboratory and clinical studies. Testing compares the biosimilar with the original biologic in what is called a “head-to-head” comparison. To obtain approval, these tests must show that the biosimilar is similar in quality, provides the same health benefits, and is as safe as the original biologic medicine.1–4

Without clinical studies, biosimilars cannot be approved for patients. 
Why should I participate in a biosimilars study?   +

Consider the potential benefits of participation in biosimilar studies.

  • You would receive active medication at no cost. Half of patients receive the approved biologic medication – the other half receive the biosimilar (investigational medication).
  • There are no costs to participate. Study medication and procedures are provided at no cost, and expenses for transportation and parking may be covered.
  • A biosimilar is a copy of an approved biologic with known health benefits, safety profile, and side effects.
  • Before a biosimilar is tested in patients, it will have already undergone extensive testing in the laboratory to show that the medication quality is similar to the original biologic.
  • You can expect high-quality healthcare throughout the study through regular visits with the study doctor, and tests and assessments to monitor your health.
  • By taking part in a biosimilar study, you would be helping to make more affordable versions of biologic medicines available to people around the world, some that would never have access to these medicines due to cost.
What are the potential risks?   +

As in any clinical research study, there are potential risks.

While the approved biologic or biosimilar may help to treat your disease, improvement cannot be guaranteed and you may experience unpleasant (and potentially serious) side effects.

Clinical studies require patients to make visits to the study center for health evaluations and assessments, or tests, according to a planned schedule. Attending these visits is important to closely monitor your health and collect important information for the study. Carefully consider the visit schedule and what is expected of you before deciding to participate.

You should always make informed decisions about your healthcare. Before you enter the screening process to evaluate if you qualify for a particular clinical study, the study doctor will provide you with an Informed Consent Form that includes potential benefits and risks. This form will also explain that you can leave the study at any time, for any reason, without negatively impacting your care.

So, ask questions and make a decision that is right for you!

Want to learn more?   +

Explore our website! Learn more about Biosimilars by Disease and Biosimilars by Region.

Biosimilars Knowledge Connect was founded by Quintiles, a global clinical research organization, to educate investigators about biosimilars. Investigators are physicians who participate in clinical studies, enroll patients, and monitor patients’ health throughout the study period. This website is designed to encourage their participation in biosimilar clinical studies to bring more affordable medications to patients around the world.

References   +

  1. US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Scientific considerations in demonstrating biosimilarity to a reference product. Guidance for industry. April 2015. http://www.fda.gov/downloads/DrugsGuidanceComplianceRegulatoryInformation/Guidances/UCM291128.pdf. Accessed 5 February 2016.
  2. US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Quality considerations in demonstrating biosimilarity of a therapeutic protein product to a reference product. Guidance for industry. April 2015. http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm291134.pdf. Accessed 5 February 2016.
  3. European Medicines Agency (EMA). Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues. December 2014. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2015/01/WC500180219.pdf. Accessed 5 February 2016.
  4. European Medicines Agency (EMA). Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: quality issues (revision 1). May 2014. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2014/06/WC500167838.pdf. Accessed 5 February 2016.
  5. Kozlowski S, Woodcock J, Midthun K & Berhman Sherman R. Developing the nation’s biosimilars program. N Engl J Med (2011) 365:385–388.
  6. EMA European Public Assessment Reports. Biosimilars. http://www.ema.europa.eu/ema/index.jsp?curl=pages%2Fmedicines%2Flanding%2Fepar_search.jsp&mid=WC0b01ac058001d124&searchTab=searchByAuthType&alreadyLoaded=true&isNewQuery
    =true&status=Authorised&keyword=Enter+keywords&searchType=name&taxonomyPath=&treeNumber=&searchGenericType=biosimilars&generics KeywordSearch=Submit
    . Accessed 5 February 2016. 
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