Rare diseases have attracted more attention in recent years, perhaps partly because of the high price tags associated with approved treatments. But the cost of rare disease drugs is dwarfed by the cost of not having them.
Our recent study shows that the lack of treatment for a rare disease is associated with a 21.2% increase in total costs per patient per year. This puts a significant burden on the healthcare system. While rare diseases affect a small population, the cost to society is staggering—we estimate that the societal cost for all 7,000 known rare diseases in the US could be in the range of $7.2–$8.6 trillion per year.
Historically, there has been a lack of shared responsibility for reducing the burden of rare diseases by various stakeholders including industry, government bodies, policy makers and society. But addressing this growing public health crisis requires a societal approach and will need to include better health policies. The good news is that the availability of treatments can reduce the substantial economic burden caused by rare diseases, and pharma and biotech companies are increasingly focusing on the rare disease population. Policymakers may be interested to know that, based on the study, productivity-related economic losses for both patients and caregivers fell from approximately $61,000 when treated to approximately $22,000 for patients and approximately $20,000 for caregivers with treatment. $22,000 and $5,000 were not available.
These and future economic data may help justify increased government investment to ensure broad patient access to safe and effective treatments and policy proposals that reflect the unique challenges in the rare disease community. And it looks like government leaders are listening. Current US policy encourages rare disease R&D, and new bills introduced to Congress would help advance rare disease drug development by, for example, increasing the orphan drug tax credit from 25% to its original 50%. Restoring access and inhibiting exclusivity for rare disease clinical trials during the pandemic. In addition, the bipartisan Bicameral Benefits Act would allow patients and advocates to play a larger role in the FDA’s benefit-risk framework for drug approval.
Regulators are also listening to the call to action. According to comments from Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), the agency is preparing a pilot program to encourage the development of new drugs for rare diseases, similar to “Operation Warp” for Covid. Speed”. -19 Vaccines.
Meanwhile, the Center for Drug Evaluation and Research (CDER) reports progress in its Accelerating Rare Disease Cures (ARC) program, which is designed to accelerate the development of effective and safe treatment options that meet the unmet needs of people with rare diseases. was launched to give and increase. As part of the ARC program, to help identify and address knowledge gaps when it comes to rare diseases and to better understand the challenges facing rare disease drug developers, the Rare Disease Drug Developers (LEADER There are also learning and education initiatives to enhance and empower 3D). ,
Both CDER and CBER are involved in the Rare Disease Endpoint Advancement (RDEA) pilot program, another initiative that helps drug developers develop effective endpoints for clinical trials in rare diseases. And most recently the FDA and NIH announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and developing new treatments for amyotrophic lateral sclerosis (ALS). To promote development. Other rare neurodegenerative diseases.
These are important steps but there is a long way to go. Accelerating approval of proposals that limit reimbursement for rare disease treatments poses a continuing threat to fostering and sustaining innovation, despite the positive economic return of these treatments.
Congress should increase funding for the FDA’s Orphan Disease Grant Program, increase NIH rare disease research and funding, empower rare disease physicians and researchers to review rare disease applications and advise regulatory agencies, rare pediatric patients The Priority Review Voucher Program must be reauthorized permanently, and fully enclosed. To better understand their unique experiences with the rare disease patient community.
Funding for rare diseases should be largely allocated alongside health conditions – which include diabetes, heart disease, Alzheimer’s disease, various types of cancer and arthritis – to reduce the associated significant societal burden. Social infrastructure must be optimized to increase caregiving resources and provide relief to families affected by rare diseases, as they bear particularly high indirect or non-reimbursable costs.
It is critical that pharma and biotech companies bring important new treatments to market and collaborate concurrently with government bodies, advocacy groups, regulators, and other stakeholders to ensure that policies enhance, not decrease, the use of new treatments. and patients have access to promising treatments.
Rare disease treatments provide clear positive returns to society, justifying increased government investment in rare diseases, not only for research and development but also for diagnosis, including screening. The sooner patients are diagnosed and placed on available treatment, the greater the benefits to society.
Giacomo Chiesi is Head of Global Rare Diseases at Chiesi Group, where he leads the team developing and commercializing treatments for rare and ultra-rare diseases.,
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