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Rare Diseases And Orphan Drugs: Finding Clinical Trials

September 10, 2014

With the increase in pharmaceutical companies developing orphan drugs, there is a need for tools for rare disease patients and healthcare professionals to provide the capability to find clinical trials appropriate for their disease, geographic location, and other important factors. In clinical trials for rare diseases, the sample size is usually small, requiring innovative approaches to optimize the study design and data analysis.

I – is a free tool that helps consumers, patients, researchers, and healthcare professionals find clinical trials specific to their search criteria. It is a free registry and results database of publicly and privately supported clinical trials of human participants conducted around the world. It is run by the US National Library of Medicine (NLM) at the National Institutes of Health (NIH). The website currently lists approximately 154,000 studies with locations in the United States and in 185 countries.

Any research project receiving money from the United States government has to be listed here – it is a requirement. One can type in the disease name and find all sorts of information about the studies.

Each clinical trial that is listed on the website includes the following information:

•   Title, description, and design of study
•   Disease or condition and experimental treatments studied
•   Requirements for participation
•   Locations where trial is available
•   Contact information.

II – Brazil’s ReBEC

In Brazil, the Brazilian Clinical Trials Registry / Registro Brasileiro de Ensaios Clinicos (ReBEC) provides regularly updated information about federally and privately supported clinical trials. ReBEC is an open access virtual platform for registration of ongoing or concluded experimental and non-experimental studies performed in Brazil and elsewhere.

III – Reg4All

Genetic Alliance, a nonprofit that advocates for patients with rare genetic disorders, launches at the beginning of 2013, a new website Reg4All, with the purpose to attract more patients into clinical trials and disease research. Patients are given privacy controls and a greater say in how their data is to be used for research. Reg4All lets patients to identify how their information is to be used – sharing it with particular researchers, institution, or investigators studying a specific disease.

Although the initial focus of the effort is getting patients into clinical trials, Reg4All will also continue Genetic Alliance’s previous work on building registries and biobanks of patient DNA and other samples for research on the causes of disease.  

IV – PatientsLikeMe

PatientsLikeMe, a for-profit company, provides an online network for patients to talk to others about their same disease or condition. Through the networking, patients generate data about their disease that help researchers, pharmaceutical companies, providers, and nonprofits develop more effective products, services, and care. The organization is open about providing access to industry through data-sharing partnerships.

In 2013, PatientsLikeMe announces its free global clinical trials search tool at Europe’s Healthcare Innovation Expo. The tool “draws on open data to match patients from around the globe with clinical trials based on their condition and location. The U.S. prototype was launched last year …”. The new tool uses information from the US government’s website and individual trial listings. This new search tool gives patients the ability to find clinical trials they may want to participate in, and also helps pharmaceutical companies match their clinical trials to the appropriate patients.

V –

In an article from, a new free search engine launches that offers “…. consumers greater access to clinical trials around the world and helps researchers access qualified participants.” The development of the website, is supported and underwritten by the Biomedical Research and Education Foundation and the Bruce and Marsha Moskowitz Foundation. The search engine is updated in real-time 24/7 and is formatted to run on mobile devices. According to the search engine’s creators, the following features differentiates it from other clinical trial search engines :

• Access to clinical trials in the US and around the world
• Mapping function that shows exact location
• Push notification for patients who are searching for specific diseases, conditions, or wellness studies.

Plans are to add a social network, mobile applications, and to provide for the capability of patients with “rare diseases to find one another and a clinician or researcher of interest”.

The source of the clinical trial information is the publicly available information from and academic medical centers.

VI – Clinical Trial Seek app

Novartis Pharmaceuticals launches a free iPhone/iPad and Android cancer clinical trial app in the US called the “Clinical Trial Seek app”. The Clinical Trial Seek app gets cancer trial information from The search capability allows patients and healthcare professionals to find, navigate, and share in-depth clinical trial information.

VII – Clinical Trial Resource app

Lilly Oncology launches in 2012 a free smartphone app, “Clinical Trial Resource”, to help healthcare professionals find clinical trials (irrespective of their sponsor) of cancer drugs for their patients. The app is available for the iPhone/iPad, Android, and BlackBerry devices. The app lets the healthcare professional find clinical trials based on a number of different criteria :

•   Cancer type
•   Country
•   State
•   Drug
•   Phase of the clinical trial
•   Other factors.

Please Note:  Postcards and Magnifying Glass by Anna (Flickr: records) [CC-BY-2.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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