Miracle of haemophilia drugs: Personal views about a few main players

Research output: Contribution to journalArticle

8 Citations (Scopus)

Abstract

Introduction: In the second decade of the third millennium there have been dramatic developments pertaining to the availability of highly innovative drugs for hemophilia care, notwithstanding a satisfactory previous scenario. Aim: I am going to emphasize the role of 2 main categories of players: scientist physicians who produced important translational research and the pharmaceutical industry, who developed, produced and made commercially available so many improved treatment weapons stemming from the translational research of the forementioned scientist physicians. Methods: PWH were enrolled through the University Hospital of the West Indies, Jamaica. The impact of haemophilia was assessed using a comprehensive battery of heath outcome measures that included the following: laboratory, clinical information and validated outcome measures of joint structure and function, activity, and health-related quality of life (HRQoL) to provide a health profile of the Jamaican haemophilia population. Results: Pertaining to the role of scientist physicians, I chose to mention first those who were successful in the 1980 in the production of recombinant coagulation factors. In addition, those who more recently helped to produce new non substitutive therapies given by the subcutaneous route, and recombination coagulation factors with an extended half-life. Conclusions: Current miraculous progress in hemophilia therapy is stemming from the research work of outstanding scientist physicians who acted in close collaboration with small biotechnology companies, leading to the early development of innovative therapeutic products, subsequently taken to the market place by the so called Big Pharma. I shall briefly provide my views to explain the fact that large pharmaceutical companies show more and more interest in such a rare disease as the hemophilias.

Original languageEnglish
Pages (from-to)557-562
JournalHaemophilia
Volume24
Issue number4
DOIs
Publication statusPublished - 2018

Fingerprint

Hemophilia A
Translational Medical Research
Blood Coagulation Factors
Physicians
Pharmaceutical Preparations
Outcome Assessment (Health Care)
Jamaica
West Indies
Physician's Role
Weapons
Drug Industry
Therapeutics
Biotechnology
Rare Diseases
Genetic Recombination
Half-Life
Joints
Quality of Life
Health
Research

Keywords

  • Coagulation factor inhibitors
  • Emicizumab
  • Hemophilia
  • Non-substitutive therapies
  • Recombinant coagulation factors
  • Translational research

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Miracle of haemophilia drugs : Personal views about a few main players. / Mannucci, P. M.

In: Haemophilia, Vol. 24, No. 4, 2018, p. 557-562.

Research output: Contribution to journalArticle

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