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Decades after Congress passed the Orphan Drug Act, only about 10 percent of patients with rare diseases actually receive treatment with orphan drugs, a new report finds.  

So what’s keeping patients from much-needed medication?

For starters, patients can’t take a medicine that doesn’t exist.  Though congressional action spurred the development of more than 500 orphan drugs, the vast majority of rare diseases still don’t have approved treatments.

For others, the need for a lower-cost option remains.  Only about half of the orphan drugs now eligible for generic competition face any.  More generics could expand treatment options.

And for some rare conditions, off-label use of newer, “non-orphan” medicines have reduced the demand for creating a specific orphan treatment.

Then there’s another reason for limited use of orphan drugs:  Access challenges.

The vast majority of orphan drugs are expensive.  To protect profits, health plans have adopted policies to help them “better manage overall expenditures on medicine” in recent years.  Translation: utilization management.

For example, insurers place complex drugs like biologics on specialty tiers.  Instead of paying a modest $10 or $20 co-pay, patients using higher-tiered drugs are responsible for a percentage of the drug’s price.  This can mean hundreds or thousands of dollars out of pocket each month.

Many insurers expanded their use of prior authorization as a way to control costs.  The practice requires physicians to submit extensive paperwork to ensure a prescribed medication will get covered. Meanwhile patients wait for the medicine they need.  Insurers may deny the initial request, prompting appeals and longer delays. For patients with rare diseases like cystic fibrosis, these delays can cause irreversible damage.

A third tactic involves insurers outsourcing the administration of their drug programs to pharmacy benefit managers.  Health plans claim this helps ensure patients are paying the lowest price for their prescription.  But with record profits, some wonder if patients are reaping the supposed benefits.

Policies like these lead to higher out-of-pocket costs and piles of paperwork, and they ultimately hinder patients’ access to existing treatments.

Policymakers laid the foundation for rare disease treatment research with the Orphan Drug Act of 1983.  But health insurers and drug manufacturers must continue the work to ensure patients with rare diseases have treatment options – and that their options are affordable and accessible.  Patients with rare conditions, like cervical dystonia, rely on medications to control their symptoms so they can perform everyday functions and live their lives.

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