Innovations in Drug Discovery and Pharmaceutical Manufacturing for Novel and Orphan Diseases

In the healthcare and pharmaceutical manufacturing world, many companies are quietly driving significant change, advancing treatments, and helping to address some of the most pressing health challenges. While the public may not always know their names, the innovations emerging from these organizations are shaping the future of healthcare. Recently, we had the opportunity to interview experts at a company in this space, Pikralida, a biotech firm working on treatments for rare and complex diseases. This conversation, combined with my background in healthcare as a former respiratory therapist, has made me reflect on the transformative work being done in drug discovery and its profound impact on patients’ lives.

In this post, I’ll explore the exciting areas where innovation is taking place and highlight some of the tools that are powering these advancements in the pharmaceutical industry.

1. Advancing Research for Novel and Orphan Diseases

Orphan diseases, which affect smaller patient populations, have long been underfunded and overlooked. However, a renewed focus on these diseases is helping to change the landscape of healthcare. For example, sickle cell disease, a condition that disproportionately affects Black communities, is seeing progress. Recently, a patient from New York became the first to be seemingly cured of sickle cell after receiving gene therapy, a groundbreaking moment for the field. This type of research, alongside ongoing work into genetic and biological treatments, shows just how far we’ve come in the fight against diseases once thought impossible to treat.

The impact of these developments is especially personal for me, as someone who has witnessed firsthand the toll that chronic conditions—like sickle cell, diabetes, and certain types of cancer—can take on individuals and families. The progress being made gives me hope that we are moving closer to breakthroughs that could offer life-changing treatments for those with limited options.

2. Tools Powering Drug Discovery and Medical Treatment Innovations

Abraham Lincoln famously said, “Give me six hours to chop down a tree and I will spend the first four sharpening the axe.” This sentiment applies well to pharmaceutical manufacturing, where even the best intentions and most well-charted plans are not executable without the right tools. These tools are critical to advancing research and achieving success in drug development.

While many companies are contributing to the evolution of drug discovery, one area of focus is the adoption of advanced technologies that streamline manufacturing and increase precision. Technologies such as 3D printing, twin-screw extrusion, and continuous granulation are helping researchers and drug developers create more efficient, customized solutions.

• 3D Printing: Techniques like Direct Powder Extrusion (DPE) allow for the rapid, customized formulation of drugs without the need for solvents or complex post-processing. This speeds up development and enables highly tailored medications that can meet individual patient needs.
• Twin-Screw Extrusion (TSE): This technology is critical for ensuring the precise mixing of active pharmaceutical ingredients (APIs) and polymers, allowing for consistent and reproducible drug formulations.
• Continuous Granulation: This process is key for creating oral solid dosage forms (OSD). By improving manufacturing efficiency while maintaining critical quality attributes, continuous granulation is transforming how solid oral medications are produced.

These technologies are pushing the boundaries of what’s possible in drug development, enabling faster, more reliable production of high-quality medications.

3. Addressing Complex Health Challenges: The Role of Polypharmacy

Beyond treating rare diseases, the healthcare industry is also focusing on a growing issue: polypharmacy. Polypharmacy refers to the use of five or more medications simultaneously, which is common in older adults and those with multiple chronic conditions. While polypharmacy is essential for managing complex health issues, it also comes with significant risks, including adverse drug interactions and increased hospitalization rates.

Research shows that up to 57% of adults over 65 experience polypharmacy, often due to cancer treatments or other chronic conditions. The pharmaceutical industry is working to reduce the negative effects of polypharmacy by developing fixed-dose combination drugs and improving the solubility of active ingredients.

An article on Polypharmacy in Cancer discusses the prevalence and impact of polypharmacy among cancer patients, highlighting how critical it is to address these issues as part of improving patient care.

Conclusion: A Bright Future Powered by Innovation

Reflecting on my journey from healthcare practitioner to marketing technologies in pharmaceutical manufacturing, I’m incredibly optimistic about the future of health. The innovations happening today, driven by advanced technologies, are paving the way for a healthier world.

The work being done to tackle orphan diseases, improve drug formulations, and address the challenges of polypharmacy is a reminder that the future of healthcare is filled with promise. These advancements are not only improving the quality of life for vulnerable populations but also setting the stage for more personalized, effective treatments in the years to come.

References and Additional Resources

Links Discussed:

1. Interview with Pikralida

1. • URL: Interview with Pikralida – https://youtu.be/fFg3bZvScF8?t=33
   • Summary: Video overview of Pikralida’s work on orphan and novel diseases.

2. CDC’s Page on Sickle Cell Disease

1. • URL: CDC – Sickle Cell Disease Data – https://www.cdc.gov/sickle-cell/data/index.html
   • Summary: Provides detailed information on the prevalence and impact of sickle cell disease.

3. Pharmacy Practice News Article on Polypharmacy in Cancer

1. • URL: Polypharmacy in Cancer – https://www.pharmacypracticenews.com/Clinical/Oncology/Article/03-25/Polypharmacy-Cancer-Adults-Prevalence/76474?ses=ogst
   • Summary: Discusses the prevalence and impact of polypharmacy among cancer patients.

4. 3D Printing in Pharmaceuticals Review

1. • URL: 3D Printing Transforming Pharmaceuticals – https://doi.org/10.3390/pharmaceutics17030390
   • Summary: Reviews the advancements and applications of 3D printing in pharmaceutical manufacturing.

5. Thermo Fisher Scientific’s Powder Rheology Accessories

1. • URL: Powder Rheology Accessories – https://www.thermofisher.com/us/en/home/life-science/lab-equipment/rheometry-viscometry/accessories/powder-rheology-accessories.html
   • Summary: Provides insights into powder rheology and its importance in pharmaceutical manufacturing.

Additional Suggested Links:

1. Thermo Fisher Scientific’s Pharmaceutical Manufacturing Solutions
   • URL: Thermo Fisher Pharmaceutical Solutions – https://www.thermofisher.com/us/en/home/industrial/pharma-biopharma/drug-formulation-manufacturing.html
   • Summary: Showcases a range of solutions for pharmaceutical manufacturing, supporting innovation and efficiency.
2. Global Transformational Health Research – OSD Formulation
   • • citation: Global Transformational Health Research Team at Frost & Sullivan. Growth Opportunities in the Oral Solid Dosage Formulation Industry. April 2024.
     • Summary: Highlights growth opportunities in the oral solid dosage formulation industry.
3. Advanced Process Automation of Continuous Wet Granulation
   • • URL: Powder Technology – https://doi.org/10.1016/j.powtec.2023.118936
     • Summary: Discusses advanced automation and process control in continuous wet granulation manufacturing.