An influential think tank funded by insurance companies and pharmacy benefit managers called the Institute for Clinical and Economic Review (ICER) is holding a policy summit in Washington, DC on Wednesday (May 31) to discuss “methods for assessing the value of new drugs for rare conditions.” Called the Orphan Drug Assessment and Pricing Summit, this meeting should make those in the rare disease community very nervous, as ICER is no friend of the Orphan Drug Act and the rare disease community. With the future of the U.S. health care system in flux, and groups such as ICER suggesting policy changes that could have devastating effects for millions of people suffering from rare diseases, the pharmaceutical and biotech industries need to work with the rare disease community to ensure their voices are also heard.
This Summit is especially important since Congress has yet to reauthorize the Prescription Drug User Fee Act. Every 5 years, Congress has to reauthorize the Prescription Drug User Fee Act (PDUFA) and so far it has yet to be done. The negotiations between industry and the FDA have been on-going, but there hasn’t been much attention on PDUFA as the House of Representatives was focused on the American Health Care Act, and the Senate is now working on its own version of the health care replacement bill.
The fees generated from the PDUFA agreement account for about 70% of the Food and Drug Administration’s (FDA’s) drug review budget. The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) wrote to Congress earlier this month, asking them to act swiftly on PDUFA to reauthorize the user fees. But so far, it has not been done.
All of this is important because without proper oversight and representation from the rare disease community, negative changes could be made to both the PDUFA and the Orphan Drug Act, leading to significant changes to the pharmaceutical and biotech industries.
The FDA’s definition of a rare disease is that it accounts for no more than 200,000 patients in the United States. Although this is a small number of patients for each individual disease, overall rare conditions afflict more than 30 million people.
According to the Pharmaceutical Research and Manufacturers of America (PhRMA) and the ALS Association (in their Medicines for Rare Disease report), the FDA has approved more than 500 orphan drugs since the passage of the passage of the Orphan Drug Act 35 years ago, and there are currently more than 600 therapies under development. Examples of the medicines currently in development include:
- 151 for rare cancers and 82 for rare blood cancers;
- 148 for genetic disorders, including cystic fibrosis and spinal muscular atrophy;
- 38 for neurological disorders, including ALS and seizures;
- 31 for infectious diseases, including rare bacterial infections and hepatitis; and
- 25 for autoimmune diseases, including systemic sclerosis and juvenile arthritis.
It would seem that the Orphan Drug Act would be credited as being a tremendous long-term success and an example of how good government policy can change an industry for the better, helping millions of people with cures for devastating illnesses. However, the Orphan Drug Act seems to have influential enemies, especially with ICER. In these uncertain times, rare disease organizations such as NORD, Global Genes, and CheckOrphan need to be out front championing the many successes of the Orphan Drug Act and the rare disease community.
