The end of the decade brought with it an exciting and critical trend for people living with rare diseases. Over the last eight years, the Center for Drug Evaluation and Research (CDER) approved more than twice as many orphan drugs as in the previous eight years (see graph below).
It is often the case that people living with rare diseases face a long and difficult journey to diagnosis. During this time patients are going from doctor to doctor in search of answers, treatments, and hope that they are on the path the health. Too often patients go undiagnosed or, perhaps worse, are diagnosed and sadly informed that there is no treatment for their particular rare condition.
The number of annual orphan drug approvals are trending in favor of the rare disease community, but not as quickly as is necessary for the people suffering daily from the implications of their conditions. Every orphan drug approval is certainly a scientific advancement and the epitome of hope for patients and their families who have waited years, maybe decades for a viable treatment option. Unfortunately, the majority of people living with rare diseases (roughly 95%) are still waiting for the creation and approval of drugs to treat their conditions. While every new drug launch inspires hope in the rare disease community, each approval is also a reminder of the many treatments yet to be discovered and many people who are not yet able to rely on tailored medications for their complex health needs.
PANTHERx is proud of our contributions to the strides made in rare pharmacy over the last decade and we are committed to greater advancements in the decade ahead. We continue to support our rare disease community and work fervently with our industry leading partners to offer more breakthrough therapies, more care programs, and more hope to patients and their families who deserve the best level of care and quality of life that our world’s ever advancing medicines can provide.