Overcoming the Barriers to Rare Disease Treatments
Rare diseases affect millions of people, yet over 95% of these 7,000+ conditions still have no approved treatment. Developing therapies for rare and orphan diseases is challenging. Small patient populations make traditional clinical trials and commercial investment difficult. Limited funding and scattered expertise slow progress, and regulatory requirements can be complex when patient numbers are low.
Another critical hurdle is the lack of manufacturing partners willing to produce small-batch therapeutics – many developers struggle to find facilities for niche drugs. Despite these barriers, the rare disease community continues to push for solutions, refusing to let loved ones face hopeless diagnoses.
We believe the future of rare disease research is one where every patient can have hope for a cure, and that those cures will be affordable and accessible from day one. The many foundations, patient groups, and advocacy organizations are helping make that future a reality, and it is our mission to help them at every stage of development.
Turning Challenges into Solutions
Every barrier to drug development for rare and orphaned diseases presents a new opportunity for innovation, collaboration, and perseverance. The key is bringing together the critical mass of support, resources, and empathy to bring an end to the suffering inflicted by these myriad diseases. Rallying around these challenges will deepen our shared committment to health equity for all:
Collaboration Networks
Patient advocacy groups, researchers, and biotech companies form coalitions to share data and expertise, ensuring that even ultra-rare conditions have a coordinated research effort. Public–private partnerships and global consortia help pool resources and knowledge beyond what any single group could achieve.
Innovative Trial Design
With so few patients per disease, researchers pursue creative approaches like adaptive trials, umbrella protocols, and other novel methods to demonstrate a therapy's benefits. Regulators have begun offering flexible pathways for approval, recognizing the need for agility in rare disease trials.
Incentives and Funding
Philanthropic contributions from grassroots patient groups has always driven the core of rare disease research.
Policies such as the Orphan Drug Act provide vital incentives that spur further investment in rare conditions. In combination with grants and mission-aligned capital, funding is on the rise.
Specialized Manufacturing Considerations
Perhaps the most concrete obstacle for rare and orphan diseases is access to manufacturing services. Few vendors can affordably supply material at such a small scale for both clinical trials and commercial production, traditionally favoring large-volume projects to benefit from the economies of scale. This leaves rare disease researchers with few options, especially early in development when product quality is critical to the assessment of safety and tolerability.
This challenge can be overcome by establishing dedicated, flexible manufacturing resources, including facilities that specialize in small-batch production and prioritize patient impact over profit. By tailoring production processes to meet the specific needs of this patient population, these solutions can be used across many disease indications with similarly small patient populations. No project should be shelved due to lack of manufacturing support or the cost of a complex process.
Houser Labs - Your partner in Orphan Drug Manufacturing
Small-Batch cGMP Production Lines
Our clinical-scale production facilities are set up to deliver high quality products at any scale you require. Whether its a gene therapy for a handful of patients or a new biologic for a few dozen, our team is ready to help you bring it to patients.
The Nonprofit Model of Manufacturing
We strive to put patients before profits, and our cost structure reflects the committment to ensuring your drug is delivered at the lowest possible price. This also allows us to sponsor individual manufacturing runs to subsidize the cost even further.
End-to-End Support for Discovery Programs
Houser labs is your partner in drug development from the early stages of discovery to commercial production. Our seamless pipeline and constant support ensures as few barriers as possible between innovation and cure.
A Unique Bridge from the Lab to the Clinic
Developing a new therapy for a rare disease often feels like an uphill battle. Houser Labs was founded to level the playing field for rare disease treatments. We believe that no cure should be out of reach because of a “business case” or manufacturing roadblock. By providing world-class manufacturing capabilities in a nonprofit model, Houser Labs empowers researchers and patient advocates to translate breakthroughs into real-world cures. Together, we are proving that the challenges of rare disease drug development can be overcome with creativity, determination, and the right partners.
Whether you are a foundation with a promising discovery or a biotech startup tackling an orphan condition, Houser Labs is here to help turn your vision into life-changing reality. Let’s break down barriers and bring hope to every family affected by rare disease – one groundbreaking new treatment at a time.