Orphan Drugs Find a Home

Guest Blog by Sharon Ferrer

Sharon Ferrer - Director of Pharmaceutical Contracting at BioPlus Specialty Pharmacy

The pharmaceutical landscape continues to get remapped. Between now and 2016, several blockbuster drugs are poised to lose patent protection. This means that the cost of prescription medications will nose-dive for millions of patients (and their insurance companies) as generic competition heats up.  

In just the next year alone, seven of the 20 best-selling medications will be available in generic versions for the first time ever (including the cholesterol drug Lipitor and the blood thinner Plavix). Generic versions for the treatment of high blood pressure, asthma, diabetes, depression, high triglycerides, HIV, and bipolar disorder are coming, as well. In fact, over the next decade, more than one hundred brand-name prescription drugs will lose market exclusivity.  

Granted, the cost savings of generics is good news for patients and insurance companies, but there is another side to this story. Pharmaceutical companies invest big money in R&D for patented drugs and are seeking to offset patent protection losses. It turns out that orphan drugs might be one way to do this. No matter the motivation, when orphan drugs get more Big Pharma attention, patients with rare diseases will benefit.  

Orphan drugs are medications that target rare medical conditions. The rarity of each condition hampers the cost effectiveness of research and development, compared to conditions that are much more prevalent. These normal economics, however, are being turned on their head with the need to offset sales declines associated with the loss of patent protection.   

Orphan drug examples:

• ·       Cinryze (treats HAE-hereditary angioedema)
• ·       Xenazine (treats Huntington’s disease)
• ·       Adcertis (treats HL and ALCL)
• ·       Cerezyme (treats Gaucher disease)
• ·       Fabryzyme (treats Fabry disease) 

The U.S. Congress encourages pharmaceutical companies to develop drugs to treat rare diseases, as evidenced by the Orphan Drug Act of January 1983 that gives orphan drugs exclusivity in the marketplace for seven years, as well as clinical trial tax incentives. In addition, orphan drugs have the added perk of expedited approval by the FDA and fewer patients needed in clinical trials. With the upcoming flood of generics, the incentives in this law are more attractive than ever before for pharmaceutical companies. To sweeten the deal even more, Congress is currently considering even broader exemptions for drug makers of orphan drugs sold to government health insurance programs. 

It’s heartening to see a win-win as orphan drugs find a home: as pharmaceutical companies research rare conditions, the fruits of their labors benefit the health of those with rare conditions.  

The story does not end, however, when a drug is approved. We believe that patients needing these kinds of specialty medications will see the best treatment outcomes under the care of a specialty pharmacy, such as BioPlus Specialty Pharmacy.  

We work with patients and the treating physician’s team to overcome challenges such as cost and the ins and outs of starting treatment (including side effect education and management, injection training, or needed lifestyle changes). As a specialty pharmacy, BioPlus has 20 years of experience providing special tools and services for the best possible outcomes in what is often a very complex process of treating a rare disease.

Guest Blog by Sharon Ferrer, Director of Pharmaceutical Contracting at BioPlus Specialty Pharmacy