2019 Predictions for Late Phase Clinical Trials: Prediction #2

rare disease research, rare disease clinical trials, Orphan Diseases, Clinical Trials, Clinical Research
Group of medical professionals discusses clinical trials


2019 is here! In our latest blog series, we unpack four predictions for late phase clinical trials in 2019. Our next prediction discusses “orphanization” across therapeutic areas.

Group of medical professionals discusses clinical trials

Prediction #2: “Orphanization” Across Therapeutic Areas

Few companies are developing the 10th generation of an approved product. A rapidly developing understanding of disease-related pathophysiology increasingly divides traditional therapeutic targets into several highly discrete entities, frequently clustered under the umbrella of “personalized medicine.” Specialty medicine, including these orphan drugs, has become a growth engine in the pharmaceutical industry, and is emblematic of Worldwide Clinical Trials’ client base.

Specialty pharma focuses its efforts on one or two therapeutic areas with specialized physician populations. Specialty-based R&D usually is characterized as:

  • Small
  • Niche
  • Agile
  • Focused

These attributes are increasingly popular with investment teams. Competitiveness by a contract research organization therefore requires:

  • Scientific and methodological competency
  • Therapeutic acumen
  • A seamless and transparent operational infrastructure that also is small, niche, agile, and focused

Tax advantages, a regulatory environment providing dispensations for nonclinical data (frequently seen as limited value apart from data related to the primary pharmacology), and a substantively truncated clinical development program that uses real-world evidence data to complement a registration package are common. Moreover, there is a high probability of success leading to market authorization because of regulatory interactions facilitating development. Finally, most specialty-based therapeutics will have high value based upon price, an ability to implement direct-to-patient marketing, and require the creation of a sales distribution system that partners with patient advocacy groups.

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