drug development and approval


Next frontier of drug development and approval

Will regulatory agencies align in moving policies forward?

Amid a barrage of medical data and new technologies, life sciences companies and regulators are improving the drug development and approval process. By establishing guidance and standards that consider scientific and evidentiary models as well as innovation, these organizations are helping to ensure safe and effective medications get to the market efficiently.

Improving the drug development and approval process

Future progress in BioPharmaceutical (biopharma) research and development (R&D) hinges, in part, on reducing regulatory uncertainties. Many biopharma companies and regulatory agencies around the world are continually exploring ways to evolve their processes, programs, and standards to reflect new scientific and evidentiary models occurring in drug development.

This paper examines three areas that multiple stakeholders consider to have the potential to substantially improve the drug development and approval process. If biopharma companies, regulators, and policymakers find common ground on the following areas, it could enable a smoother pathway to the next frontier of drug development and approval:

  • Benefit-risk assessment: Enhancing transparency among regulators and between regulators and biopharma companies.
  • Patient-reported outcomes (PRO): Encouraging continued stakeholder collaboration to develop procedures and policies around the use of patient-reported instruments and delineating pathways for patient involvement in the drug development process.
  • Real-world evidence (RWE): Identifying leading practices for RWE use and interpretation, and establishing a multi-stakeholder task force focused on standardizing data to make the evidence reproducible, interoperable, and operational for regulatory purposes.

Combined, the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) regulate most of the global pharmaceutical market. These agencies have implemented a number of policies and programs over the years to keep pace with scientific advances and drug development methods, and to work collaboratively with manufacturers to bring innovative new therapies to patients as soon as possible.1 The agencies also work closely with one another in certain areas.2

If regulatory agencies align to move these priority areas forward, and provide clear guidance for the biopharma industry to follow, the result could lead to significant change to the global regulatory environment. While there is general consensus that total regulatory alignment may never be achieved, or that such alignment would not be desirable across every country, many stakeholders agree that increasing alignment in certain areas of the drug approval process may produce positive results for the consumer, industry, regulators, and public health.3

1. F Ehmann, M Papaluca Amati, T Salmonson, M Posch, S Vamvakas, R Hemmings, HG Eichler and CK Schneider, “Gatekeepers and Enablers: How Drug Regulators Respond to a Challenging and Changing Environment by Moving Toward a Proactive Attitude,” Clinical Pharmacological Therapeutics, May 2013.
2. Pharmaceutical Manufacturers, “Regulatory Harmonization Failures. . . and Prescriptions for Success,” August 28, 2012.
3. M Rosolowsky, “Introductions and perspectives on international harmonization,” FDA/PQRI Conference on Evolving Product Quality, 2014.

Biopharma areas of uncertainty

The global biopharma industry operates in an extremely challenging environment. Biopharma companies are expected to grow revenue and boost shareholder return despite ever-increasing R&D costs, slow time-to-market, and pressure by governments, health care providers, and health plans to control costs and improve outcomes.

A dynamically changing scientific, regulatory, and business landscape is requiring companies to re-evaluate their traditional business and R&D models to:

  • Demonstrate value: Widespread efforts to slow health care spending growth, reduce variations in care, and engage consumers in self-care are increasing emphasis on demonstrating a drug’s value. As part of this focus, comparative effectiveness research is starting to play a role in product adoption and market uptake. In the future, products not proving to be more effective than their competitors may struggle to generate demand or attain reimbursement.
  • Bend the cost curve: Cost-conscious governments and other health care payers are imposing price controls and increasing incentives for physicians and patients to use less-expensive generic drugs and biosimilars.
  • Maintain regulatory compliance: The evolving regulatory landscape continuously challenges the biopharma sector. Although all regulators focus on patient health, safety, and privacy, the way policies are developed can vary widely from country to country.

Fertilization dish

The path forward

Biopharma companies, regulators, and the research community will need to integrate new technologies and a huge and growing influx of data into the decision-making process. Increased stakeholder input and collaboration should help to ensure that this data is leveraged so that early laboratory findings can be translated into therapies that improve patients’ health. The priority areas outlined in this paper have the potential to improve the efficiency of the drug pipeline, and to develop safe and effective treatment options for patients around the globe. We recognize that the path forward will not be easy. Industry and regulatory agencies may need to dedicate resources to advance their individual initiatives, and then identify opportunities to jointly define, communicate, and achieve shared goals.

Ultimately, it will be up to regulators to put forth consistent, aligned guidance around incorporating quantified benefit risk frameworks, PRO, and RWE into the approval process. However, the biopharma industry should consider continuing its efforts to align on approaches that could achieve the same goal. The Biotechnology Innovation Organization (BIO) has recently published guidance for the industry on how to incorporate patient perspectives into the FDA’s qualitative framework for benefit-risk assessment. Additional progress can be made if stakeholders use established venues and coalitions to define frameworks and continue creating and implementing approaches across all three priority areas.

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