Rare Cancer Treatment Portal

educational tool for patients
with rare Cancers in europe

find out more about your
Disease and treatments
in your country


Every pharmaceutical substance or active pharmaceutical ingredient cited on this tool has been listed by its International Nonproprietary Name (INN).
Please check the 'What To Search For' section for more information.

The Rare Cancer Treatment Portal is an educational tool for patients and healthcare professionals that is intended to provide up-to-date information about the accessibility of treatments for rare cancers in Europe. The tool is not designed for specific product promotional purposes and will not favour any manufacturer of products. To ensure an unbiased approach, this tool reflects all publicly available information on clinical trials, compassionate use programmes, patient partnerships, approved treatments and special access arrangements in designated disease areas.

The selection of initial disease areas for the Rare Cancer Treatment Portal pilot project is based on a preliminary questionnaire, approved by Rare Cancers Europe members, which identified appropriate cancers to use as pilots. The choice of cancer types to use as pilots for the Rare Cancer Treatment Portal was based on their likelihood to provide an opportunity to utilise all the parameters of the tool and the potential need for information for patients in that disease area. The pilot cancers were also chosen because they are covered by specific European Reference Networks (ERNs) and there is currently a selection of treatments available for them.

Pilot Projects

Based on these criteria – the following disease areas have been chosen for pilot projects:

By including all reimbursed treatments in these selected areas, this tool offers no advantage to a single product or company but simply reflects the existing state of access to a product, based on publicly available data. Data may be provided, for expediency, by industry but will not contain any proprietary or confidential information.

Every effort has been made to provide the most accurate and up to date information available on the pilot cancers in the Rare Cancer Treatment Portal. Periodic reviews will ensure that, as new treatments emerge, they will be added to the Rare Cancer Treatment Portal.

At the moment, the Rare Cancer Treatment Portal only covers systemic treatments and not treatments such as proton beam therapy, radiosurgery, devices, complementary therapies, etc.

    The information on this Portal was last verified and updated in February 2020, the next periodic review is due to take place by the end of the year.

    The Rare Cancer Treatment Portal is designed to help patients gather information on treatment availability, accessibility and on-going clinical trials for rare cancers in Europe.

    Using this tool is easy, the search function will help you connect any terms you may have heard regarding a disease with the specific disease page. Our search function is built by experts in the field and should accommodate a wide range of specific related terms. Once you have started to type your word, different page options will appear.

    You will then be referred to a subpage providing general information about the cancer family and the cancer type. There you will be able to select a country in Europe and check the availability of treatment, reimbursement status, ongoing clinical trials and the availability of compassionate use programmes.

    Every pharmaceutical substance or active pharmaceutical ingredient cited on this tool has been listed by its International Nonproprietary Name (INN). You can find the INN of the product you are searching for on the label and on the packaging of the product itself.  

    Clinical phase II and III

    The clinical phases of medicines development involve humans, and is different from the “non-clinical” or “pre-clinical phase” in which studies are performed in labs or on animals (as is the case for pharmacology/toxicology analysis). Clinical studies are usually conducted in four steps, called “phases” – each designed to answer separate research questions:

    • Phase I is often composed of small trials, recruiting only a few patients. Phase I trials are done to find out (1) how much of the drug is safe to give; (2) what the side effects are; (3) how the body copes with the drug;
    • Phase II objectives include: therapeutic effect; optimal dose; safety (toxicity); and proof of concept. Phase II trial is most often designed to find out more about the side effects of the medicine and to get more data on possible effectiveness.
    • Phase III aims at confirming the efficacy and safety of a product. Often Phase III consists of a randomized trial and needs a lot of participants to fully answer how effective and safe the medicine is.
    • Phase IV trials are done after a drug has been shown to work and has been granted a licence. Phase IV’s objectives include finding out more about the side effects and  safety of the drug and about its long-term efficacy. 

    Compassionate use

    Method of providing an unlicensed medicine (i.e. one with no marketing authorisation yet) to patients prior to final approval by a regulatory authority for use in humans. This procedure is used with very sick individuals who have no other treatment options. Often, case-by-case approval must be obtained for compassionate use of a medicine or therapy.

    European Reference Networks

    European Reference Networks for rare, low prevalence and complex diseases (ERNs) are virtual networks involving healthcare providers across Europe. They aim to facilitate discussion and recommendations on complex or rare diseases and conditions that require highly specialised treatment, and concentrated knowledge and resources.

    Expanded Access Programmes

    Sometimes patients can enter ‘expanded access programmes’. A company that makes a promising medicine, prior to marketing authorisation, may choose to run one of these programmes to allow early access to their medicine and to widen its use to patients who can benefit from it. For example, patients who have been treated with a medicine during a clinical trial and wish to continue treatment off trial may be able to do so via an expanded access programme. These programmes are often authorised by national authorities in the same way as clinical trials, and patients are followed in the same way as patients in a clinical trial.

    The FDA uses the terms "expanded access" and ‘compassionate use’ interchangeably. It is important to note that this is not the case in Europe.


    The rate of new (or newly diagnosed) cases of a disease. It is generally reported as the number of new cases occurring within a period of time.

    International Nonproprietary Names

    International Nonproprietary Names (INN) facilitate the identification of pharmaceutical substances or active pharmaceutical ingredients. Each INN is a unique name that is globally recognised and is public property. A nonproprietary name is also known as a generic name.

    Marketing Authorisation

    Marketing authorisation (MA) refers to the approval for a medicine to be marketed. A system for marketing authorisation was put in place to protect public health. Marketing authorisations are granted only when a competent authority (or ‘regulatory authority’) has conducted a scientific evaluation, and is satisfied that a medicine is sufficiently safe and effective, and of high enough quality to be licensed for use in humans. Different procedures exist to obtain an MA. The European Medicines Agency (EMA, the ‘Agency’) is responsible for the ‘centralised procedure’. A single application is submitted to the EMA for evaluation by the Agency’s scientific committees. If the assessment is positive, a single marketing authorisation is issued by the European Commission (EC). The Marketing Authorisation Holder (usually a pharmaceutical company) can then legally begin to market the medicine in all EEA (European Economic Area) countries [EU Member States and the four EFTA (European Free Trade Association) States (Republic of Iceland, Principality of Liechtenstein, Swiss Confederation, Kingdom of Norway)]. 

    Named-Patient Programme

    Doctors can obtain a promising medicine, prior to marketing authorisation, for a patient by requesting a supply of the medicine from the manufacturer, to be used for a specific patient under their immediate responsibility. This is often called treatment on a ‘named-patient basis’ and should not be confused with compassionate use programmes. In this case, the doctor responsible for providing the treatment will contact the manufacturer directly. While manufacturers do record what they supply, there is no central register of the patients who receive treatment in this way.

    Off-label use

    Off-label use of a medicine is the use of an authorised medicine by a healthcare professional to treat a patient in a way not covered by the Marketing Authorisation (MA) and not detailed in the Summary of Product Characteristics (SmPC) of the medicine.

    Off-label use of medicines is not regulated by EU legislation and no uniform definition is available. Advertising the off-label use of an authorised medicinal product is prohibited. All advertising must comply with the SmPC.


    Prevalence is the actual number of people cases alive with the disease either during a period of time (period prevalence) or at a particular date in time (point prevalence). Period prevalence provides the better measure of the disease load since it includes all new cases and all deaths between two dates, whereas point prevalence only counts those alive on a particular date.

    Randomised clinical trials

    A study in which the participants are divided by chance into separate groups that compare different treatments or other interventions.

    Rare cancers

    Rare cancers are identified as those with an incidence of less than 6 per 100,000 persons per year.

    Useful links: