From TV to Reality

Sep 17, 2012
Thanks to a Quincy M.E. episode about rare diseases, the Orphan Drug Act was passed in 1983. Today, Express Scripts supports many of these life saving drugs.

Television programs aren’t typically credited with saving lives. Yet one show, now off the air for nearly 30 years, helped champion more attention for, and better treatment of, patients with rare diseases.

From 1976 to 1983, “Quincy, M.E.” starred Jack Klugman as a forensic pathologist investigating suspicious deaths. In 1981, an episode titled “Seldom Silent, Never Heard” featured the need for more treatments for patients with rare diseases. Such treatments, known as orphan drugs, received little attention at the time. Due to small patient populations, they were typically considered cost prohibitive to develop.

Thanks in part to that episode galvanizing public opinion, the U.S. Orphan Drug Act was passed in 1983. The act provided incentives to pharmaceutical manufacturers of orphan treatments, including tax credits for the costs of clinical research, seven years of patent exclusivity, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

In 1983, two drugs were granted approval as orphan drugs. Now, more than 2,600 other medications have been designated for study or approved for marketing as orphan drugs.

The Impact of Orphan Diseases

Orphan diseases affect an estimated 25 million people in the U.S., or 10% of the U.S. population. There are 6,800 known orphan diseases globally, and rare-disease patient populations can range from just a few people to 200,000. Some of these diseases are well-known, including multiple sclerosis, cystic fibrosis, sickle cell anemia and many forms of cancer. Others, known as ultra-orphan diseases, are extremely rare, with patient populations of 20,000 or fewer.

Improving Patient Access, Drug Safety and Health Outcomes

Orphan drugs and diseases received renewed attention in July 2012 when the Safety and Innovation Act was signed. The law included these provisions to further support the development of orphan drugs:

  • Accelerated patient access to new medical treatments
  • Resolution of conflict-of-interest provisions introduced in the previous PDUFA reauthorization
  • Accelerated development of “breakthrough therapies” that show early promise
  • Enhanced FDA consultation with rare-disease medical experts

Once considered as too costly an investment for research and development, orphan drugs have received more designations and approvals in the last decade than ever before. 

A recent Thomson Reuters study, The Economic Power of Orphan Drugs, documents the tremendous growth the orphan drug market and manufacturers have experienced. The study found the top orphan drugs were as follows: 

Top Orphan Drugs

Express Scripts supports each of these products through our specialty pharmacy and/or specialty distribution services.

In addition, manufacturers contract with UnitedBioSource Corporation (which now includes the HealthBridge Pharma & Biotech suite of services) to provide: 

  • Best-in-class Risk Evaluation & Mitigation Strategies (REMS) programs with sophisticated registries to identify and monitor patients
  • Leading reimbursement services
  • Dedicated care coordination teams
  • 24-hour access to disease-state-specific clinicians
  • Field nursing support network
  • Patient Assistance Programs
  • Clinical development

It’s safe to say that as orphan drugs rise in viability, so will the development of future medication for patients with diseases so rare that treatments are currently limited or even nonexistent. We tip our hats to Jack Klugman and “Quincy, M.E.,” and at the same time are pleased to know that we’re supporting that legacy through our industry-leading pharma services in support of orphan drugs and patients who take them.

Author Bio

Kevin Cast
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