Rare diseases and orphan Drugs

256px-Postcards_and_magnifying_glassWith 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 – ClinicalTrials.gov

ClinicalTrials.gov 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.

Panellists at Pharmexcil's 10th annual meet propose to set up resource group ..  — pharmabiz.com
.. from Chandigarh, have not only discussed the various issues prevailing in India with regard to rare diseases and orphan drugs, they have also proposed solutions for the same and as an initial step they have agreed to set up a resources group on the ..

Popular Q&A

How the Orphan Drug Act Has Helped

Developing a new drug or device can be expensive. Because rare diseases affect a relatively small number of people, drug companies generally demonstrate little interest in performing research or development of new products to treat such diseases. Developers face a further difficulty in testing potential treatments because it is difficult to recruit a sufficient number of people to study safety and effectiveness.

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