Each year, on the last day of February, we recognize Rare Disease Day. It is an opportunity to shine a light on a class of diseases that do not often make in onto the front page of the newspaper, but whose collective impact on more than 30 million Americans, their caregivers and loved ones is significant.
Rare Disease Day allows us to take stock of the extraordinary progress made toward delivering treatments and cures to these individuals and to advance policy and regulatory changes to incentivize future innovations to treat rare diseases.
According to the journal Nature, the Food & Drug Administration’s (FDA) Center for Drug Evaluation and Research approved 21 drugs with orphan indications in 2015, up from 17 in 2014, and from 9 in 2013. Overall, nearly half (47 percent) of all of the drugs approved in 2015 were orphan-designated drugs for rare diseases. These included approvals for innovative drugs that advance the treatment of Multiple Myeloma, Cystic Fibrosis and a range of pediatric rare diseases.
Despite this success, we know more work needs to be done. Today there are only around 450 FDA-approved orphan therapies, but more than 7,000 rare diseases. That means that 95 percent of rare diseases currently have no treatment.
So how do we address this?
The good news is there are amazing scientific breakthroughs occurring across the biopharma industry. The science is galloping forward at a breathtaking pace. In just the last few years, we have seen enormous advances in both our understanding of biological science and in our ability to harness that science into innovative drugs and therapies that are transforming the medical standard of care for devastating and previously untreatable diseases. And it is only going to get better with exciting advances in areas such as cell therapy and immunology.
However, one of the things I always tell biopharma entrepreneurs, investors and my former colleagues in Congress is that no matter how good your science is, if the policy and regulatory environment is not keeping pace with what is happening in the lab it is extraordinarily difficult to succeed.
Throughout history, advances in science and drug development have increased when Congress passed legislation that supports, promotes and incentivizes the innovation being conducted in the lab. These include policies like the Orphan Drug Act, the Hatch/Waxman Act, The Prescription Drug User Fee Act, the Food and Drug Administration Modernization Act, the Biologics Price Competition and Innovation Act, the creation of the Small Business Innovation Research program, and the Jumpstart Our Business Startups Act.
Take the Orphan Drug Act. A recent study conducted by BIO, the National Organization for Rare Disorders, and Ernst & Young concluded that one-third fewer new orphan drugs would have been developed to treat rare diseases in the last 30 years if Congress didn’t pass the tax credit included in the Orphan Drug Act. Since passage of the Act—which also included incentives for rare disease drug development beyond the tax credit—more than 200 new orphan drugs have been approved by the FDA – compared with only 34 approved before enactment.
This is an example of the important role that smart public policy can play in supporting innovation and incentivizing the enormous amount of private investment that is required to get a new drug or therapy to patients.
Over the last 10 years, we have seen an increase in investment into rare diseases. This inflow of private capital is helping to fund nearly 500 rare disease clinical trials.
While this is promising, it represents only about 10 percent of the clinical pipeline. More can and needs to be done on the policy front to help speed the pace of drug development and to lower the cost of bringing new innovations from the lab bench to a patient’s bedside.
These include integrating the patient’s perspective into all aspects of drug development. The 21st Century Cures Act, passed by the House of Representatives last year, included several provisions that would help integrate the patient voice into areas such as the design of clinical trials and the FDA’s assessment of the balance between a treatment’s benefits and risks when deciding on final approval.
Placing the patient at the center of the drug development process will help spur new therapies for rare diseases and other unmet medical needs.
We also believe that advancing adaptive clinical trial designs and the use of surrogate end points will help speed the pace, and reduce the cost, of drug development.
Smart and effective public policy will help ensure that patients have access to new and more innovative treatments. It means that, hopefully, with each coming Rare Disease Day we can celebrate starting to move more rare diseases from the pages of medical books to something we only read about in history books.
Greenwood served in the House from 1993 to 2005. He is president and CEO of the Biotechnology Innovation Organization.