Rare Diseases definition

Rare Diseases means any disease or condition that meets the criteria for an orphan or rare disease or condition in a Major Market, including, for example in the United States, in §526(a)(2)(1) of the Federal Drug and Cosmetic Act (U.S. Orphan Drug Act (1983)); in Europe, in the Orphan Drug Regulation 141/2000; and in Japan, in the Orphan Drug Amendment (1993).

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Rare Diseases shall include adrenoleukodystophy (ALD) (including but not limited to AMN, CCALD, and all other variants of this disease caused by genetic mutations), beta hemoglobinopathies (including but not limited to beta-thalassemia and sickle cell anemia), [***]. Whether in the case of [***], such diseases shall be considered on a case-by-case basis and considered a Rare Diseases if the incidence or prevalence is similar to those diseases listed as Rare Diseases above. In these situations, Licensee and/or Affiliates shall provide justification as to whether such disease is a Rare Disease, in writing, prior to payment of the Milestone #4 or #5 as applicable.

Rare Diseases mean human diseases considered to be a “rare disease” in the applicable jurisdiction, e.g., as defined by the Rare Diseases Act of 2002 in the U.S. or by the European Commission on Public Health in Europe.

Examples of Rare Diseases in a sentence

• Starting from 2003 the Medical Genetics and Rare Orthopedic Diseases Department started a collection of data from patients and families affected by Rare Diseases (in particular skeletal dysplasia) and in the subsequent years stared to standardize this data collection and in collaboration with CLIBI Laboratory established the REM (Registry for Mutliple Ostechondromas Disease).

• Amicus’ scientific and clinical expertise in human genetic diseases is among the best in the industry, and we are pleased to be collaborators and investors in this exceptional company,” said ▇▇▇▇ ▇▇▇▇▇▇▇, Global Head of GSK Rare Diseases and a member of the GSK Corporate Executive Team.

• For clarity, neither Party may file for Regulatory Approval or Commercialize Products in (i) any Additional Indication for which Licensee has elected not to share Development Costs or (ii) any additional indication other than Rare Diseases unless the Parties mutually agree to do so.

• Human Gene Therapy for Rare Diseases: Guidance for Industry; Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research: Silver Spring, USA 2020.

• Either Party may, by written notice to the other Party (an “Escalation Notice”), refer disputes regarding Development of the Product in the Territory or regulatory issues relating to the Product in the Territory to the chief executive officer of Amicus (or his/her designee) and the GSK Head of Rare Diseases Unit (or his/her designee) (the “Senior Executives”).

• Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI).

• The TF on Rare Diseases (TFRD) and the newly established TF on Health Expectancies (TFHE) have very informative websites, and we hope to have one online for the TFMCD in the first half of 2007.

• For this reason several indicators will be developed by WP2 (Data Sets), WP4 (Access) and WP7 (Rare Diseases).

• The Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) has initiated the NIH/NCATS GRDRSM Program.

• The publication should include the following acknowledgement: Data used in the preparation of this article reside in the NIH/NCATS GRDRSM Program’s Global Rare Diseases Patient Registry Data Repository (GRDR).