Creating a Win-Win for Rare Disease Patients, HCPs & Payers

As many as 30 million Americans currently live with a rare disease. The U.S. Food and Drug Administration (FDA) defines an orphan (rare) disease as a condition that affects fewer than 200,000 people nationwide. Some rare diseases have patient populations of fewer than one hundred people.

According to Global Genes, the highly respected patient advocacy group:

  • Three of 10 children with a rare disease won’t live to see their 5th birthday
  • Rare diseases impact more people than cancer and AIDS combined
  • There are 7,000 distinct types of rare and genetic diseases

Collectively, the diseases affecting the 30 million Americans represent a serious public health concern. Finding new technologies, including gene therapies, to save or improve their lives should be a societal imperative.

Nearly 40 years ago, a bipartisan commitment to finding treatments for rare diseases spurred tremendous innovation. The Orphan Drug Act of 1983 worked exactly as designed; it created incentives for manufacturers to develop new therapies for very small patient populations, sometimes fewer than 100 people. During the first 25 years of the Act becoming law, 326 new drugs were approved by the FDA and brought to market.

The Challenge

Today the U.S. health care system is struggling to make medical breakthroughs available to patients. Our health care system was designed in the 20th century with elements conceived over 50 years ago. Yet, 21st century innovations are critical to rare patients in need and our health system was not designed to make them readily available.

  • Patients and their families (50 percent of people with rare diseases are children) struggle to afford the co-payments associated with medications whose productions costs cannot be absorbed by millions of patients, such as drugs and devices for heart patients
  • Health care professionals struggle to provide the best care available to their rare disease patients if they can’t afford the treatment
  • Private and public payers (such as Medicare and Medicaid) struggle to manage mounting financial burdens and budgetary constraints of supporting the medical and therapeutic needs of rare disease patients, particularly when a product might be used by only a few thousand, a couple of hundred or even a mere dozen individuals

Here are two critical issues that RAAP seeks to solve:

  • How do we as a society create incentives to advance therapies through discovery and development while also ensuring that patients can affordably access them once they are approved by the FDA?
  • How can we reduce these barriers and challenges in a way that balances all the competing imperatives, and maintain a commitment to finding new therapies for the 6500 rare diseases for which there are currently no effective treatment options?