Executive Director Amanda Malakoff and Lisa Feng, the Senior Director of Policy at Alexion, AstraZeneca Rare Disease (an RDCC member company) joined the Vital Health Podcast with Duane Schulthess for a discussion on the accelerated approval pathway for rare disease treatment development.

Recent proposed federal and state policies to restrict coverage and access to biomedical innovations approved through the FDA’s accelerated approval pathway would harm patient access to rare disease treatments. Vital Transformation completed a comprehensive impact assessment of these proposed changes and what they could mean for patients currently lacking treatments and future innovations. According to this research, up to 66% of accelerated approval treatments could be at high-risk of not coming to market or being developed at all if the FDA’s accelerated approval pathway were negatively impacted.

Listen below for the full discussion and click here for more information on why protection of the accelerated approval pathway for rare disease treatment is urgently needed.

Executive Director Amanda Malakoff joined the Global Genes RARE Cast Podcast for a discussion with Daniel Levine. In this podcast, Amanda covers a range of topics, including why the Coalition was formed, the current policy landscape for rare disease therapies, and our policy priorities for the year ahead grounded in the continued advancement of R&D for rare disease treatments so that patients can have the best access to the treatments they need.

Listen here:

By Gabrielle Wanneh / November 22, 2022

This article was published by Inside Health Policy.

Rare disease advocacy groups hope the new Congress will prioritize investment in the development of rare disease treatments by passing bills that restore and reinforce seven-year exclusivity for drugs being developed for specific rare conditions, require regular reporting to improve the approval process of orphan drug designation and ensure patient input is counted throughout the development process.

With Republicans set to control the House and Democrats to maintain control of the Senate, and new committee leaders yet to be formally announced, it’s too early to tell how legislation aimed at accelerating the development of rare disease treatment will be handled, sources say, despite bipartisan support for some measures.

Amanda Malakoff, executive director of the Rare Disease Company Coalition, is hopeful that bills like the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act and the Helping Experts Accelerate Rare Treatments (HEART) Act have the bipartisan support needed to be pass in one or both chambers next year.

The BENEFIT Act would make sure patient experiences, patient-focused drug development and other related data are considered as part of FDA’s risk-benefit assessment, while the HEART Act would require FDA to consult external experts and stakeholders while also providing reports on how FDA is handling applications for a drug to be designated for the treatment of a rare disease.

“There’s just a lack of expertise in general in [the area of] rare disease, so we really advocate for the FDA making sure they’re bringing people with the most timely and relevant knowledge into the process,” Malakoff told Inside Health Policy.

“Anything that can be done to streamline the process at FDA will help give that confidence back to investors on rare disease and biotech, and those two bills are just examples of steps Congress can take to help make that happen,” Malakoff added.

Another bipartisan bill, Cameron’s Law, would increase the tax credit for clinical testing expenses under the Orphan Drug Act from 25% back to the original 50%, years after the Tax Cut and Jobs Act reduced the credit in 2017. Malakoff said restoring the tax credit to its original parameters would make it more economically feasible for drug companies to develop rare disease treatments and could also send a positive message to investors.

Malakoff noted that provisions from both the BENEFIT Act and HEART Act had been included in Congress’ user fee reauthorization bill at different points in time before it passed in September. It’s unclear whether measures from either bill will make it into the upcoming year-end legislation.

Heidi Ross, vice president of Policy and Regulatory Affairs at the National Organization for Rare Disorders, said that reinforcing the seven-year exclusivity period for rare diseases and conditions under the Orphan Drug Act, and specifying that this period prohibits the approval of other drugs for the same use or indication, is another issue that may be able to garner bipartisan support. The Retaining Access and Restoring Exclusivity (RARE) Act, co-sponsored by Sens. Tammy Baldwin (D-WI) and Bill Cassidy (R-LA), would do just that.

Cassidy is expected to be the ranking Republican on the Senate health committee.

The bill would reverse the September 2021 Catalyst v. Becerra decision that upended FDA’s longstanding narrow interpretation of how and under what circumstances the exclusivity period should be granted.

“Our concern is that you will end up in a world where if you are awarding exclusivity to just cystic fibrosis, then you’re not going to have companies have the proper incentives to study those more challenging patient populations. The biggest population might be genetic mutation XYZ, but the patients that have genetic mutation ABCD also deserve to know that a therapy is safe and effective for them,” Ross said.

Similar language that codifies the FDA’s prior interpretation of how to award orphan drug exclusivity was included in the version of the Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act and the Food and Drug Amendments of 2022 passed by the House passed in June. The provision ultimately did not make it into the final clean user fee package that Congress passed in September, but many of the FDA reforms that were left out of the final package could still be included in year-end legislation.

If not included in Congress’ year-end legislation, Ross said it’s also possible the RARE Act alone could garner bipartisan support next Congress should Cassidy become the ranking Republican of the Senate health committee. — Gabrielle Wanneh (gwanneh@iwpnews.com)

A recent study finds that FDA’s accelerated approval pathway can lead to faster drug development and more treatment options for patients with rare diseases that are difficult to study. Once a drug obtains FDA approval – whether through a traditional or accelerated approval pathway, patients can access the therapies through Medicaid benefits, saving lives and easing the medical burdens of many. 

However, suggested proposals to limit reimbursements for accelerated approval therapies within Medicaid would limit access to—and threaten the future development of—orphan drugs that rely on this trusted pathway for viability. These proposed policy changes would cause both immediate and lasting harm to the rare disease community – and disincentivize future use of the valuable pathway for certain rare diseases.

RDCC calls on Congress to reject these unfounded proposals and instead support policies that would promote greater patient access to FDA-approved therapies. 

Read more about the study’s findings and how to protect accelerated approval for rare disease treatments in this overview.

Accelerated Approval image of article

Research and development (R&D) is at the heart of rare disease innovation. Due to the complex nature of rare diseases, a comparatively higher percentage of operating expenses is dedicated to R&D for the over 93% of rare diseases without an FDA-approved treatment. 

In 2021, our 21 Coalition members alone invested more than $12 billion into R&D, representing on average over half of their annual budgets. In addition, many of our members with products on the market are continuing to spend more on R&D than they make in revenue, investing profits back into future treatment development. The result of these endeavors have built a remarkable hub of innovation, growth and investment across life science companies. 

However, a harmful tax change that if not reversed, will hurt rare disease companies’ ability to advance lifesaving innovation while limiting additional job creation and development here in the United States. Up until January 2022, a business in the United States could deduct 100% of their R&D expenses in the year during which those expenses incurred.  

Now, a change in the tax code that took effect this year requires businesses to spread those deductions over a period of years—the so-called amortization requirement—making investment in innovation more expensive to conduct.

This week, RDCC joined over 400 organizations across the greater business community to urge Congress to repeal this harmful tax change. Doing so is vital to ensure that rare disease companies can continue to innovate, advance continued and ambitious research and development programs, and bring treatments to patients. On the cusp of significant breakthroughs in molecular, cell and gene therapy advancements, we must continue to support medical innovation taking place at rare disease companies to support delivery of life-altering treatments to rare disease patients.

Read the full letter to Congressional leadership here.

Our Coalition joined with more than 70 organizations, led by the March of Dimes, to call on the U.S. Senate to advance the “Newborn Screening Saves Lives Reauthorization Act of 2021” (S. 350). The House version of the bill (H.R. 482) passed with overwhelming bipartisan support last summer. Given the very limited availability on the Senate calendar, now is the time for the Senate to act on this bill and advance it to the floor.

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.

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.