Rho: evolution of drug development reflects focus on quality

The highly complex field of drug development is constantly evolving. From regulatory developments, to available technologies, to market conditions, industry professionals continually face shifting challenges.

Outsourcing-Pharma (OSP) recently spoke with David Shoemaker, senior vice president for Rho, about how the drug development field has transformed in the three decades years since he entered the industry — and what might lie ahead.

OSP: You’ve been in the industry for quite a while—how has the field evolved since you first started?​

DS: Over the course of my 30 years in the industry, I have certainly seen it evolve tremendously. I came into clinical research prior to the Prescription Drug User Fee Act (PDUFA) and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), and I consider the introduction of this documentation to have brought significant influence and benefit to the evolution of the industry. PDUFA has made FDA much more transparent and accountable to the industry, and ICH has done its best to internationalize the standards for product development and marketing approval for all regions.

Additionally, the winnowing of the research and development arms of big pharma has thrust much more responsibility onto biotechnology companies, academia and government as the discovery engines for the industry.

Finally, I would say the industry’s transition from a focus on blockbuster products to products designed to treat rare diseases has had an enormous effect on the quality of the science that is being conducted at pharmaceutical and biotechnology companies around the world. For example, oncology has advanced during this time from experientially directed combination therapy to scientifically directed combination therapy, which has resulted in marked success for patients.

OSP: What are some of the key milestones both at your company and in the industry in general?​

DS: As a regulatory professional, I would consider some of Rho’s key milestones to always be around the successful marketing approval of products, whereby you are able to provide important, new medications to patients who – without these new treatments – would not be able to be cured. Rho contributes significantly to on average two to three marketing applications each year by assisting our clients with the regulatory meeting conduct, authoring the meeting documents – which may include marketing application materials – or analyzing the data that will support the efficacy and safety of the product.

The industry should be extremely proud of these approvals and how some of these treatments have helped improve the life expectancy – and even cure – some oncological and rare disease patients.  It is amazing to see how the advancement of potential gene transfer, editing, cell and tissue therapies has finally reached fruition after 30 years of searching for the “Holy Grail.” The next 10 years will no doubt see a flurry of approvals with these exciting technologies to bring relief and improved health to millions of patients.

OSP: How has the regulatory landscape changed?​

DS: PDUFA and ICH have brought major benefits to the industry, and these primarily direct the laying down of regulatory processes and establishing international regulatory standards for product development and marketing approval. Additionally, I would say the regulators’ perspective waxes and wanes throughout the years, with incidents like the death of a subject in a gene transfer clinical study in 1999 and the uncovering of the Vioxx scandal in 2004 increasing the conservative nature of regulatory authorities via an increased focus on safety in both the pre- and post-approval of products.

There has also been a dramatic increase in patient contributions to the marketing approval of a product. When I first started in this business, regulators did not give much weight to data derived from quality of life instruments when compared to clinical evidence derived from the clinical study. Since then, quality of life instruments have changed perspectives on the importance of patient-reported outcomes in products being approved and released into the market.

OSP: How has COVID-19 impacted the field?​

DS: The novel coronavirus (COVID-19) has had a unique challenge for the industry, as it has stopped some current clinical studies dead in their tracks while slowing the progression of others due to the transition to remote assessments – spawning an entire industry dedicated to the conduct of decentralized remote clinical trials. The industry may one day look back on COVID-19 as the single most important event responsible for forcing the industry to stop operating clinical studies in practically the same manner they have been conducted since the 1960s, resulting in the implementation of technologies and processes that expedite product development and market approval.

OSP: What solutions have professionals at your company and in the industry come up with to help mitigate the impacts of the pandemic?​

DS: It has been interesting to see the various creative solutions that have been implemented for companies that have been able to continue their studies during this time. I think the most creative of these that have come out of this pandemic have surrounded getting subjects to sites in the face of insurmountable difficulties and enlisting technologies to conduct assessments in the subjects’ homes.

Regulators have also exercised greater leniency in providing Emergency Use Authorization and other programs to help expedite the typical product development process. Regulators have also come up with other accelerative programs, such as the Coronavirus Treatment Acceleration Program at FDA, to assist getting worthy COVID-19 therapies into FDA sooner for collaboration on development and, eventually, accelerated approval.