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      What is an orphan drug?
      A female scientist conducting research in a laboratory, focusing on orphan drugs for rare diseases

      What is an orphan drug?

      The need for these rare disease therapies is strong, and access to them can be lifesaving. Here’s how Johnson & Johnson is innovating to help give patients options.

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      An orphan drug is a drug intended for use in a rare disease. The World Health Organization defines a rare disease—sometimes referred to as an orphan disease—as one that affects fewer than 65 per 100,000 people.

      More than 7,000 rare diseases have been identified, and an estimated one in 10 Americans lives with a rare disease; half of these are children. Still, about 95% of rare diseases don’t have a treatment approved by the U.S. Food and Drug Administration (FDA), according to the Rare Disease Company Coalition.

      Rare disease definition graphic

      To encourage manufacturers to address this unmet patient need, in 1983 the federal government passed the Orphan Drug Act (ODA). Under the ODA, a company can request orphan drug designation from the FDA. If granted, the company qualifies for research grants, tax credits for qualified clinical trials and potential seven-year market exclusivity after the drug’s approval, among other benefits.

      The European Union, Australia and Japan have similar programs offering various incentives including market exclusivity, research and development grants and tax credits, plus regulatory benefits to further support an increase in the development of orphan drugs.

      In some parts of the world, the definition of a rare disease differs slightly. In the United States, a rare disease is one that affects fewer than 200,000 people. The EU defines an orphan disease as one that affects no more than 5 per 10,000 individuals.

      Despite the differences in what constitutes a rare disease, the ODA and other initiatives worldwide have inspired much-needed research and development for effective, life-altering treatments. “With these incentives and the commitment from the FDA, we’ve seen a vast increase in what’s available,” says Victoria Dohnal, Director, Global Regulatory Policy and Intelligence, Johnson & Johnson.

      Before the ODA was passed, the FDA had approved just 38 orphan drugs to treat rare diseases. Today, more than 650 orphan drugs have been approved by the FDA—with more in development.

      Committing to orphan drug innovations

      Orphan drug incentives continue to drive research of new treatments for rare diseases. Developing drugs for rare diseases requires Johnson & Johnson Innovative Medicine to utilize new approaches and creative development program designs. The company is focused on areas of medicine that can make the biggest difference, which includes various rare diseases like pulmonary arterial hypertension, rare inherited retinal diseases, rare maternal/fetal diseases, blood cancers (including multiple myeloma and non-Hodgkin’s lymphoma), AL amyloidosis and myasthenia gravis.

      “These interventions give patients treatment options to help mitigate symptoms of rare diseases, helping to improve their quality of life,” says Helen Spain, Vice President of Regulatory Affairs, Pulmonary Hypertension & Rare Disease, Johnson & Johnson.

      Today, more than 650 orphan drugs have been approved by the FDA—with more in development.

      While ODA initiatives are making a difference for patients with rare diseases, “we still have a long way to go,” says Dohnal.

      “We are very active and engaged in thinking through, ‘What do we need to move the science forward?’” explains Dohnal, adding that the company is committed to working with the FDA, patient advocacy groups and other stakeholders to advance the development of orphan drugs.

      “We want to make sure we’re getting the best options to patients as quickly, as efficiently and as safely as possible,” says Dohnal. “At the end of the day, trying to create treatments and cures is what we’re all about.”

      Adds Carina Righetti, Director, Global Market Access & Policy, Rare Diseases, Johnson & Johnson Innovative Medicine: “Improved rare disease awareness, sustained scientific innovation and public policies that support research and development and patient access to new therapies allow us to work together to transform the lives of patients with rare diseases.”

      Are you living with a rare disease?

      See if you might be eligible to participate in a study that could potentially help lead to more effective treatment.

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