Our Coalition joined with more than 80 organizations, led by the March of Dimes, to call on policymakers to include the “Newborn Screening Saves Lives Reauthorization Act of 2021” (H.R. 482) as part of the FY22 omnibus spending bill in support of improving the health of babies born with rare diseases.

Each year thousands of babies are born with rare diseases that may not be apparent at birth. Fifty years ago, these infants’ disorders would have gone undetected until symptoms appeared, leading to possible death or lifelong disability. Today, a simple set of newborn screening tests performed at birth can detect these life-threatening illnesses before any symptoms begin, allowing crucial time for early diagnosis and treatment.

Click here to read the full letter submitted to Congressional leadership.

Contact your member of Congress by clicking here.

By: Betsy Ricketts, Chair of the Rare Disease Company Coalition, and Vice President of Policy, Government and Public Affairs at Ultragenyx, a company member of the Rare Disease Company Coalition


Congress’s proposed limits to the Orphan Drug Tax Credit (ODTC) in the Build Back Better Act (H.R. 5376) could severely hurt development of potential treatments for rare diseases that impact 30 million people in the United States, half of whom are children. It is difficult to understand how Congress could agree to the drastic reduction of this proven resource that supports the development of new treatments for 90 percent of the approximately 7,000 known rare diseases that currently do not have an FDA-approved treatment, many of which are life-limiting or fatal. 

To understand the significance of the ODTC, it is important to understand the unique challenges — and promises — in taking rare disease drugs from research through development, approval, manufacturing and delivery to patients. Rare diseases have small patient populations, which makes developing drugs to treat them inherently more difficult, costly, and risky than those for common medical conditions. The ODTC incentivizes biotechnology and pharmaceutical companies to invest in the development of treatments that are not otherwise economically viable. 

In 1983, recognizing rare diseases historically have attracted minimal attention, Congress established the Orphan Drug Act (ODA), creating the tax credit for advancement of rare disease treatments. At the time, only 38 drugs had been approved by the U.S. Food and Drug Administration (FDA) for rare diseases. Now, thanks to this effective and long-standing public health policy, there are more than 650 drugs approved to treat rare diseases. Most importantly, this has led to a significant and consistent decline in the number of annual deaths from rare diseases. 

Congress should not be focused on snatching hope from those living with rare diseases; our representatives should instead support and enhance existing rare disease policy because it is working. Of note, the FDA affirmed the value of the ODA and this incentive in an Office of Inspector General (OIG) report issued in September. Rep. Anna Eshoo (D-Calif.) also recently called for the complete restoration of the ODTC after it was reduced from 50 to 25 percent in 2017. 

Continue Reading at The Hill.com

By: Zachary Brennan / August 3, 2021

The controversy over the FDA’s accelerated approval pathway is heating up.

Last week, the FDA’s top oncology official Rick Pazdur said the pathway is “under attack,” largely due to the agency’s recent accelerated approval of Biogen’s controversial Alzheimer’s drug and the surrogate endpoint used in that decision. In the meantime, three accelerated approval indications have been pulled since July 1 (two from Bristol Myers Squibb and one from Merck in recent weeks), even as Pazdur called on critics of the pathway to not miss the more positive, big picture, with some cancer drugs proving to be enormously helpful and approved years before their confirmatory trials were completed.

Now, a coalition of rare disease drugmakers is seeking to defend the use of the pathway, raising concerns with a recent call from the nonpartisan MACPAC to increase rebates around drugs approved under the FDA’s accelerated pathway. The rare disease firms claim such a move on rebates would disincentivize drug developers from pursuing therapies in otherwise intractable disease areas, the coalition said in a recent letter to top congressional health committees.

By: Gabrielle Wanneh / August 2, 2021

Drug makers researching rare disease treatments are urging congressional health committees to reject calls by Medicaid payment advisers to update reimbursement policies for drugs granted accelerated approval by FDA. Patient advocates also are pressing lawmakers to reject the proposals, saying they would curtail access to innovative products and stifle development of rare disease treatments.

At issue are proposals by the Medicaid and CHIP Payment and Access Commission (MACPAC) and some state Medicaid programs to limit the amount public and private insurers pay for costly accelerated approval drugs until drug makers can prove their efficacy.

The Rare Disease Company Coalition, like the patient advocates, argues the proposals would decrease access to new innovative medications, particularly those meant to treat rare and life-threatening diseases that might be too difficult to study using the traditional pathway for approval.

By: Kate Goodwin / May 13, 2021

There are more than 7,000 rare diseases identified in the world today. While “rare” means that less than 200,000 people in the US are affected, to suffer from a rare disease is not actually rare at all. 

Around 10% of the American population suffers from a rare disease. A shocking 90% of rare diseases do not have an approved treatment on the market. 

Source: BioSpace