Rare disease drugs are transforming pharma market dynamics and patient hope
A closer look into how the current orphan drug 'revolution' came to be (Issue #309, 1,100 words, 5 minutes)
Therapies for rare diseases, once disregarded due to low prevalence and high research costs, now account for a substantial portion of new medications cleared annually. The shift is reshaping the pharmaceutical market, offering hope to patients and influencing broader drug development strategies.
While challenges persist, including the development costs of these specialized treatments, this evolution in the pharmaceutical landscape marks a significant change in how the industry approaches drug development and market opportunities for less common conditions.
According to Healthcare Brew, the U.S. Orphan Drug Act (ODA) of 1983 is spurring this orphan drug “revolution.” Before the ODA, fewer than 40 therapies addressed rare conditions, largely due to limited profitability in serving small patient populations. By incentivizing drug development with market exclusivity, tax credits, and fee waivers, the ODA catalyzed a surge in innovation, resulting in more than 1,100 U.S. FDA-approved rare disease indications and transforming orphan drugs into a dynamic market.
Despite significant progress, challenges persist. Healthcare Brew reports that the current focus on rare diseases has a negative angle. Some healthcare experts believe the ODA prompted drugmakers to overfocus on developing treatments for rare diseases instead of therapies for larger populations. A Clinical and Experimental Pediatrics study notes that diagnosing rare diseases remains arduous, often requiring extensive clinical time that may involve multiple misdiagnoses. Diagnosis is exacerbated by the complexity of genetic disorders, which account for approximately 80% of rare diseases.
Meanwhile, Clinical and Experimental Pediatrics notes that the economic burden of orphan drugs is substantial, with annual treatment costs often exceeding hundreds of thousands of dollars per patient, placing considerable strain on healthcare systems. According to Bloomberg Law, the recent enactment of the Medicare Drug Price Negotiation Program in the U.S. under the Inflation Reduction Act (IRA) has further complicated the landscape. While the program exempts orphan drugs with a single FDA-approved indication, it imposes price negotiations on therapies with multiple indications. This policy risks disincentivizing the development of expanded applications for existing therapies, jeopardizing innovation and the potential to address unmet needs.
According to Bloomberg Law, advocates argue the orphan drug market is financially sustainable because expanded patient populations can offset revenue reductions. Critics of the IRA contend the law undermines the incentives created by the ODA, which historically encouraged additional indications for rare disease therapies. Legislative amendments to preserve these incentives face significant hurdles, with stakeholders continuing to lobby for changes to safeguard research and development.
Observers agree the orphan drug market will see continued growth, driven by advancements in gene therapies, precision medicine, and innovative treatment approaches such as enzyme replacement and small-molecule therapies. However, aligning regulatory policies with the needs of patients and innovators will be critical. They say that ensuring affordability, mitigating financial barriers, and maintaining solid incentives will determine the future success of orphan drug development.
THIS WEEK 11/19/24
The U.S. FDA approved Syndax Pharmaceuticals’ revumenib (Revuforj) for treating adult and pediatric patients with relapsed or refractory acute leukemia with a KMT2A translocation.
PTC Therapeutics announced that the U.S. FDA granted accelerated approval of its gene therapy for treating aromatic L-amino acid decarboxylase deficiency.
The Canadian federal government launched the Youth Mental Health Fund to support community organizations and improve mental health services for youth nationwide.
The U.S. FDA granted marketing approval to Autolus Therapeutics' obecabtagene autoleucel (Aucatzyl) for treating relapsed or refractory B-cell precursor acute lymphoblastic leukemia in adult patients.
NOW LISTEN UP: HERE’S JONATHAN SOONG OF AMICUS THERAPEUTICS
In season 13 of the NPC Podcast, Jonathan Soong, General Manager of Amicus Therapeutics, gives insights into being at the forefront of rare disease innovations, patient support programs, and the importance of real-world evidence for rare diseases. Hear him in conversation with podcast hosts Mitch Shannon, Jim Shea, and Mark McElwain.
HEALTHBIZ REWIND
This feature of NPC Healthbiz Weekly looks back at some of the most insightful moments from previous NPC Podcast episodes. In Healthbiz Rewind, you’ll read bold life sci predictions made during the renowned “Prognostication Korner” segment of the NPC Podcast.
Rami Fayed
Vice President and General Manager, Canada
AbbVie
Montreal
Season 12, episode 07
Listen to this episode here
What bold predictions do you have about the life sciences industry over the next 12 to 24 months or even longer?
Do you need predictions? Let me focus on a couple of critical points. One is the growing influence of patients, particularly in Canada. We’re seeing different players in the healthcare industry listening more closely to patients, which reflects this increasing empowerment. I’ll be part of a panel discussion soon with healthcare professionals and government agencies to address this topic in detail. Patients are becoming more vocal about their care, especially in the post-pandemic world.
Another important trend is the evolving roles of nontraditional healthcare professionals. It’s no longer just about family doctors, neurologists, or gastroenterologists—nurse practitioners, pharmacists, and others are stepping into expanded roles. This shift is reshaping the healthcare environment and offering a solution to the significant workforce shortages in Canada. These changes have the potential to greatly enhance the capacity and efficiency of our healthcare system.
INTRODUCING DERMATOLOGY.BUSINESS and the DERMATOLOGY.BUSINESS DEEP DIVE PODCAST
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