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  1. 28 Σεπ 2021 · A majority of the highest-expenditure drugs in Medicare—some of the best-selling drugs in the world—have been granted at least one orphan designation, qualifying their manufacturers for ODA financial incentives.

  2. The increasing number and high prices of orphan drugs have triggered concern among patients, payers, and policymakers about the affordability of new drugs approved using the incentives set by the Orphan Drug Act (ODA) of 1983.

  3. The purpose of this report is to examine stand-alone Medicare Part D prescription drug plan (PDP) coverage of a set of drugs and biologics that treat rare diseases or conditions—which we will refer to collectively as “orphan drugs”—in the Medicare population.

  4. 9 Οκτ 2015 · The availability of orphan drugs depends on individual country’s legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing.

  5. 15 Αυγ 2023 · To inform debates around how the IRA may affect development of treatments for rare diseases, we examined how many orphan drugs approved by the US Food and Drug Administration (FDA) have had supplemental (follow-on) approved indications.

  6. 15 Φεβ 2021 · With a robust pipeline of orphan drugs anticipated to enter the market, including gene therapies and other high-cost, 1-time treatments, many health care stakeholders are questioning if they can afford to continue to cover them for their populations. What this study adds.

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