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.
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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.
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Examples of Rare Diseases in a sentence

  • This action will contribute towards implementing the Rare Diseases Framework.

  • The Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder and as many as 15% remain in the undiagnosed category for at least 5 years as physicians labor to define cause and pathophysiology.

  • The Drug Safety Oversight Board may add members with relevant expertise from the Food and Drug Administration, including the Office of Pediatrics, the Office of Wom- en’s Health, or the Office of Rare Diseases, or from other Federal public health or health care agencies, for a meeting under subparagraph (D) of the Drug Safety Over- sight Board.

  • Rare Diseases and Orphan Products: Accelerating Research and Development.

  • Patient Access to Medicines for Rare Diseases in European Countries.

  • It comprises 2 test cases that involve 4 biomedical ontologies cov- ering the disease and phenotype domains: Human Phenotype Ontology (HP) versus 9 http://www.pistoiaalliance.org/projects/ontologies-mapping/ Mammalian Phenotype Ontology (MP) and Human Disease Ontology (DOID) ver- sus Orphanet and Rare Diseases Ontology (ORDO).

  • Also see FDA “Developing Products for Rare Diseases & Conditions,” at http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm.

  • The second main objective is to formulate recommendations to introduce rare cancers into the National Cancer Plans and the National Rare Diseases Plans.

  • Applicants that are invited to submit a second stage proposal are strongly encouraged to make use of the EJP Rare Diseases Clinical Support Services.

  • For information on where to find patient representatives and PAOs willing to be involved in research, please see:• Orphanet portal for rare diseases and drugs patient organization directory• Rare Diseases Europe (EURORDIS)• European Reference Networks (ERNs)• European Patient’s Academy on Therapeutic Innovation (EUPATI).

Related to Rare Diseases

  • Terminal disease means an incurable and irreversible disease that has been medically confirmed and will, within reasonable medical judgment, produce death within six months.

  • Waterborne disease outbreak means the significant occurrence of an acute infectious illness, epidemiologically associated with the ingestion of water from a public water system which is deficient in treatment, as determined by the Division.

  • Opioid treatment program means a detoxification or maintenance treatment program which is required to report patient identifying information to the central registry and which is located in the state.

  • Collaborative drug therapy management means participation by an authorized pharmacist and a physician in the management of drug therapy pursuant to a written community practice protocol or a written hospital practice protocol.

  • Occupational disease means a disease contracted in the course of employment, which by its causes and the characteristics of its manifestation or the condition of the employment results in a hazard which distinguishes the employment in character from employment generally, and the employment creates a risk of contracting the disease in greater degree and in a different manner from the public in general.

  • COVID-19 symptoms means fever of 100.4 degrees Fahrenheit or higher, chills, cough, shortness of breath or difficulty breathing, fatigue, muscle or body aches, headache, new loss of taste or smell, sore throat, congestion or runny nose, nausea or vomiting, or diarrhea, unless a licensed health care professional determines the person’s symptoms were caused by a known condition other than COVID-19.

  • Non-Administrator Substance Use Disorder Treatment Facility means a Substance Use Disorder Treatment Facility that does not meet the definition of an Administrator Substance Use Disorder Treatment Facility.

  • Anabolic steroid means any drug or hormonal substance, chemically and pharmacologically related

  • Disease means an alteration in the state of the body or of some of its organs, interrupting or disturbing the performance of the functions, and causing or threatening pain and weakness or physical or mental disorder and certified by a Medical Practitioner.

  • muscular dystrophy means a group of hereditary genetic muscle disease that weakens the muscles that move the human body and persons with multiple dystrophy have incorrect and missing information in their genes, which prevents them from making the proteins they need for healthy muscles. It is characterised by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue;

  • Cannabinoid edible means food or potable liquid into which a cannabinoid concentrate, cannabinoid extract or dried marijuana leaves or flowers have been incorporated.

  • sickle cell disease means a hemolytic disorder characterized by chronic anemia, painful events, and various complications due to associated tissue and organ damage; "hemolytic" refers to the destruction of the cell membrane of red blood cells resulting in the release of hemoglobin.

  • Infectious Disease means an illness that is capable of being spread from one individual to another.

  • Alcohol Use Disorder means a disease that is characterized by a pattern of pathological use of alcohol with repeated attempts to control its use, and with negative consequences in at least one of the following areas of life: medical, legal, financial, or psycho-social; or

  • Substance use disorder professional means a person

  • Medical cannabis means the same as that term is defined in Section 26-61a-102.

  • Substance use disorder means a cluster of cognitive,

  • Rural health clinic means a rural health clinic as defined under section 1861 of part C of title XVIII of the social security act, chapter 531, 49 Stat. 620, 42 U.S.C. 1395x, and certified to participate in medicaid and medicare.

  • COVID-19 Pandemic means the epidemic, pandemic or disease outbreak associated with COVID-19.

  • Biological diversity means the variability among living organisms from all sources including, inter alia, terrestrial, marine and other aquatic ecosystems and the ecological complexes of which they are part; this includes diversity within species, between species and of ecosystems.

  • Cosmetology means the art or practice of embellishment, cleansing, beautification, and styling of hair, wigs, postiches, face, body, or nails.

  • Infection means the entry and development or multiplication of an infectious agent in the body of humans and animals that may constitute a public health risk;

  • Progressive discipline means a process of applying and documenting disciplinary actions progressing from less to more serious depending on the employee’s history and the nature of the offense.

  • Medical cannabis pharmacy means the same as that term is defined in Section 26-61a-102.

  • Cannabinoid means any of the chemical compounds that are the active constituents of marijuana.

  • Rape Crisis Center means an office, institution, or center offering assistance to victims of sexual offenses through crisis intervention, medical and legal information, and follow-up counseling.