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INTERNATIONALMEETING ON PULMONARYRARE DISEASES AND ORPHANPROGRAMDRUGSMILANO ITALY CONGRESS CENTER PALAZZO DELL STELLINEFEBRUARY 24 25, 2017PRESIDENT OF THE CONGRESSSCIENTIFIC COMMITTEESERGIO HARARI
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To fill out for orphan drugs, follow these steps:
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Begin by gathering all the necessary information for the application, such as the drug's name, purpose, and potential benefits.
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Research and identify the appropriate regulatory authority or agency responsible for reviewing and approving orphan drugs in your region.
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Prepare the application form provided by the regulatory authority, ensuring all required fields are completed accurately.
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Include supporting documents, such as clinical trial data, evidence of the drug's effectiveness, and any additional research findings.
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Pay attention to any specific guidelines or regulations outlined by the regulatory authority and ensure compliance.
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Submit the completed application along with the supporting documents to the designated authority.
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Keep track of the application status and follow up with the regulatory authority as necessary.
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Once the application is reviewed, await feedback or a decision from the regulatory authority.
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If approved, fulfill any remaining requirements set by the regulatory authority, such as manufacturing processes or labeling guidelines.
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Begin the distribution and marketing process for the orphan drug, following all applicable regulations and guidelines.

Who needs and orphan drugs?

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Orphan drugs are specifically developed to treat rare medical conditions or diseases, affecting a small portion of the population. Therefore, the individuals who need orphan drugs are those suffering from these rare conditions.
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In addition, healthcare providers, physicians, and specialists who treat patients with rare diseases also have a need for orphan drugs.
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Regulatory authorities responsible for approving drugs for rare diseases also play a crucial role in determining who needs orphan drugs, as they evaluate the drug's potential benefits and assess the level of need within the affected population.
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An orphan drug is a medication that has been developed to treat a rare medical condition.
The pharmaceutical company that has developed the orphan drug is required to file for orphan drug designation with the relevant regulatory authorities.
The pharmaceutical company needs to submit an orphan drug designation application to the regulatory authorities, providing detailed information about the drug and the rare medical condition it is intended to treat.
The purpose of an orphan drug is to provide treatment for rare medical conditions that would not otherwise receive attention from the pharmaceutical industry.
The pharmaceutical company must provide information about the drug's safety and efficacy, the rare medical condition it is intended to treat, and the proposed manufacturing process.
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