Drug discovery and manufacturing is rapidly changing across the pharmaceutical industry. The increased focus on rare diseases, along with breakthroughs in biologics, gene and cell therapy, and other forms of personalized medicine, have empowered bold innovation among drug developers. Many companies are leveraging these scientific advancements to pursue a new aspirational focus on finding cures for the most-difficult-to-treat diseases.
As big pharma increasingly relies on development partnerships and acquisitions to build its product pipelines, small biopharma companies have assumed the role of R&D innovators. Buoyed by record levels of venture capital funding, the biopharmaceutical sector is bringing forward novel drugs at a healthy pace. According to a report from global healthcare consultancy IQVIA, emerging biopharma companies patented 38 (66%) of the 59 new drugs approved by the FDA in 2018 and registered 47% of them.
FDA fosters innovation in the public good
To help biopharma companies speed their medical breakthroughs to market and more rapidly fill unmet patient needs, the U.S. Food and Drug Administration (FDA) has established several regulatory initiatives, such as the breakthrough therapy designation, that are designed to help shorten the development time of a potential new therapy.
Similarly, companies developing a regenerative medicine therapy, which includes a cell therapy, therapeutic tissue engineering product, or human cell and tissue product intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, can apply for the regenerative medicine advanced therapy (RMAT) designation, which supports expedited development and regulatory review.
The FDA’s focus on helping speed the delivery of novel drugs for unmet and underserved patient needs, particularly in the rare disease area, was reflected in its swift reflection action as part of its 45 drug approvals in 2019.
Impact on quality and regulatory compliance
The FDA’s efforts to streamline the approval process for new medicines and speed their path to commercial availability has also raised the bar for quality and compliance in drug development. The shortened time horizon for the development and approval of novel drugs has brought the quality function and regulatory filings under greater scrutiny, including the increased need for robust safety and efficacy data sets, particularly for personalized, precision medicines.
The FDA’s demand for stronger quality systems also extends to GMP manufacturing processes, including increased quality validation requirements and greater transparency across contract development and manufacturing (CDMO) vendor relationships. Another evolving requirement, especially with complex biologics, personalized medicines, and cell and gene therapies is the increased need for real time monitoring of material quality.
Advice for biopharma companies
Finding the right quality professional who can operate effectively in the evolving biopharma development and manufacturing environment is a business imperative. In working with growing biopharma companies in my practice, here’s the advice I give on adding the talent that will help them build their quality infrastructure and align their organization with the changing GMP model:
- Know your priorities. Finding the right person starts with assessing the priorities that are critical to your organization and your development lifecycle. This will help determine if you need a quality professional who is strong in an area of particular need – such as clinical or technical operations – or someone who can bring cross functional expertise to your organization.
- Hire a beneficial quality stakeholder early-on. For smaller biopharma companies, the catalyst to hiring a quality thought leader is typically following the release of positive Phase 1/2 clinical trial data. As your organization invests further in bringing its asset closer to commercial availability, the addition of a quality professional can help establish the infrastructure and culture that will be critical to the completion of upcoming milestones.
- Look for an effective persuader. A critical success factor in the quality function is a professional who has the combined technical and business acumen to influence all stakeholders to follow standard operating procedures in order to heighten overall organizational inspection readiness.
- Find a creative thinker. To further a collaborative environment where R&D flourishes, it’s important to find a quality professional who can “read between the lines” of regulations and create procedures with the flexibility that empowers innovation.
About the Author:
Tom Hayes is a Client Partner and leads Klein Hersh’s Quality Assurance and Regulatory Compliance practice. He assists clinical phase, mid-sized, and large biopharma companies clients in the identification and hiring of quality leaders. This includes vice presidents and heads of quality for small molecule, and traditional pharmaceutical companies, as well as biotech clients and service companies (CROs, CMOs).
