The RDCC sent a letter to Senator Cassidy and the Senate Health, Education, Labor, and Pensions (HELP) Committee in response to the recent request for information (RFI) on ways to improve and protect access to gene therapies for Americans with rare ultra-rare diseases. Coverage and reimbursement is a critical issue for all patients living with a rare disease and their families. As the RFI recognizes, if a patient cannot access innovative treatments, to them it is as if that innovation never occurred. 

RDCC members currently have over 200 programs in development, many of which are first-in-class gene and cell therapies. With the appropriate policy framework, these therapies could ultimately prove life changing or life saving to patients with no other treatments available.  

But current coverage and reimbursement policies have not kept pace with the rapidly-evolving science. We urge policymakers to consider solutions to ensure the ecosystem is better-equipped to accommodate tremendous leaps in innovation in the development of gene and cell therapies and ensure timely access for patients living with a rare disease. 

Interested in learning more about the RDCC’s recommendations? Click here.

The Rare Disease Company Coalition (RDCC) strongly supports the ORPHAN Cures Act introduced by Senators Barrasso (R-WY) and Carper (D-DE) and Representatives Joyce (R-PA-13) and Nickel (D-NC-13). The bipartisan and bicameral ORPHAN Cures Act works to encourage rare disease research and development (R&D) by addressing a critical disincentive in the Inflation Reduction Act (IRA) that impedes rare disease innovation and investment.

Currently, the IRA’s Orphan Drug Exclusion offers a price negotiation exemption for orphan drugs that treat only one orphan condition. Rare disease companies rely on this exemption as a critical incentive for R&D, but in limiting it to drugs that treat only one condition the IRA discourages rare disease companies from further exploring promising research that could lead to additional treatment options. The ORPHAN Cures Act would instead expand the Orphan Drug Exclusion to allow for the research and development of existing products that could help find treatments for the more than 95 percent of rare diseases without approved therapies.

For RDCC member companies that invest over half of their annual revenues back into research and development, improving the Orphan Drug Exclusion will have a significant impact in delivering desperately needed therapies and hope to underserved patient and caregiver communities.

Real-life impact: 1 in 5 orphan drugs are Food and Drug Administration (FDA)-approved for more than one use, and 60% of those second indications are for another rare disease. By limiting the Orphan Drug Exclusion to medicines that treat only one indication, the IRA discourages companies from pursuing development of promising therapies.

The ORPHAN Cures Act strengthens the intent of the ODA: Since the enactment of the ODA in 1983, the number of orphan drugs approved by the FDA has increased from just 38 to more than 600 treatments for more than 1,000 rare diseases. While this has made a dent on the 95% of the 10,000 rare diseases that exist, continued progress requires both strengthening ODA policies and continuing to advance recognition of and support for the unique needs of rare disease R&D. The ORPHAN Cures Act achieves this by removing a disincentive for developing rare disease treatments.

The final word: The ORPHAN Cures Act addresses the concerning effects of the IRA’s Orphan Drug Exclusion and allows biopharmaceutical companies to meet the clear and urgent need for continued innovation in rare disease R&D. The RDCC thanks Senators Barrasso and Carper and Representatives Joyce and Nickel for supporting the rare disease community through this important legislation.

My name is Amanda Malakoff, and I serve as Executive Director for the Rare Disease Company Coalition, or the RDCC. To provide a brief background on us, we launched two years ago to represent the unique policy needs of 20+ rare disease companies working to develop and deliver rare disease treatments to patients. Collectively, our 21 members invest over $12 billion annually in research and development with over 200 new treatments in their pipelines.      

We want to thank the FDA for making rare disease drug development a growing priority at the agency. The RDCC applauds relevant programs passed in the user fee authorization bill such as the Rare Disease Endpoint Advancement Pilot Program, increased funding for FDA, ARPA-H, and NCATS for rare disease research, policies aimed at improving clinical trial diversity, and more. We also are thrilled to see CDER’s recent launch of the Accelerating Rare disease Cures program.

Despite recent cutting-edge medical breakthroughs in rare disease biotech, and these supportive regulatory effort, many challenges still remain in getting treatments to the rare disease patients who need them in an urgent manner.      

For example, rare disease company sponsors cite inconsistent feedback across review divisions as an ongoing barrier to meeting regulatory timelines. As FDA gears up to hire many new reviewers in PDUFA VII (7), and initiates a review of drug approval trends across review divisions, we recommend that the agency streamline the approach to training new reviewers on rare disease drug development. In addition to internal consults, external consultation with patients, patient groups, disease-specific experts, and small population study experts will help the agency make well-informed decisions and avoid roadblocks in providing patients with timely access to new treatment options.

We also encourage our agency partners at the FDA to use the flexibility granted by Congress with respect to standards for conducting rare disease trials in a consistent manner. One critical tool for rare disease drug sponsors is the accelerated approval pathway, which can offer hope to patients by providing life-changing, and sometimes life-saving, treatments years earlier. Although a significant percentage of the agency’s accelerated approvals have been for oncology, we believe this can be a critical pathway for rare diseases as well and would like to see the agency provide the appropriate support for rare disease drug sponsors who opt to use this regulatory pathway. As many rare diseases are progressive in nature, and around half affect children, time is crucial. The RDCC looks forward to serving as a constructive partner on rare disease drug development issues with the agency and key stakeholders in the rare disease community. Thank you for the chance to engage in today’s important conversation.

Passed 40 years ago, it may be time for ODA 2.0

By: Steve Usdin, Washington Editor, BioCentury

As the Orphan Drug Act slides into middle age, patient advocates and biopharma companies are applauding its accomplishments, lamenting legislative setbacks that have limited its utility, and developing proposals for expanding its reach.

In the 40 years since the ODA was signed into law, FDA has approved more than 600 drugs for orphan conditions, up from 10 in the prior decade. The success has been tangible, but new policies will be needed to increase the pace of drug development if society is to come close to meeting the needs of the millions of patients living with thousands of rare conditions that have no treatments.

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.

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. 

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 95% 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.

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

Originally Published at TheHill.com (December 29, 2021)

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. 

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.

Source: Endpoints News

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.

Source: InsideHealthPolicy.com

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