Q: What regulatory requirement should be streamlined?
A: One of the most needed harmonization topics would be post-approval change. Right now a post-approval change can take up to four years before it is fully accepted by global regulators, which not only adds to the burden of the drug manufacturers, but also increases the opportunity for errors. These errors or failures can be manifold. For example, depending on the post-approval status, a manufacturer might produce the same product using different process technologies. One process could use a new update that has been approved by one regulatory agency, while another process requires old technologies, since another agency has not approved the updated technology yet. This resource and financial burden also may result in the hesitancy to modernize processes or facilities, resulting in aging facilities, which again have been recognized as a culprit for drug shortages.
Maik Jornitz is COO of G-CON Manufacturing and founder of BioProcess Resources. He has more than 25 years of experience.
Q: Has a medicine’s efficacy ever been demonstrated using a method other than a double-blind randomized design?
A: Yes, but it’s usually restricted to rare or life-threatening conditions. A recent example is lynparza for ovarian cancer, approved in December 2014. Here’s an excerpt from the FDA medical review: “The recommendation for approval is based on the single, open-label, nonrandomized trial in which olaparib demonstrated a robust overall response rate with a clinically meaningful duration of response in patients with deleterious or suspected deleterious germline BRCA mutation (gBRCAm)-associated ovarian cancer who had received three or more prior lines of chemotherapy.” This trial enrolled 193 patients with gBRCAm-associated ovarian cancer, including 137 patients who received three or more lines of prior chemotherapy and with measurable disease who were treated at a dose of 400 mg PO BID. The overall response rate in the patients with measurable disease was 34 percent with a median duration of response of 7.9 months.
DR. MITCHELL KATZ
Dr. Mitchell Katz has 30 years’ experience in the pharmaceutical and biotech industries, including preclinical research, pharmaceutical operations, and regulatory affairs. He is the Head of Medical Research and Drug Safety Operations at Purdue Pharma L.P.
What is the best leadership advice you ever received?
A: It was to “embrace diversity.” I learned this sage piece of advice from my grandmother who traveled from Scotland on her own across the Atlantic at a young age to make her way in the land of opportunity in the U.S. She saw firsthand the strength that America had was because of, not in spite of, its melting pot of cultures. I’m privileged to serve as the CEO of an organization that promotes the benefits of gender diversity, and I practice this advice daily. Studies show that every team and every company gains from diversity. No one person has all of the answers. We come at issues from different points of view based on our personal tapestry of experiences, and we solve problems using varying methodologies.
Laurie Cooke, BS, RPh, PGDip, CAE, is the CEO of the Healthcare Businesswomen’s Association (HBA), a global nonprofit professional association.