Presenting New Global Genes Toolkit: Drug Development Series

April 6, 2016

What’s taking so long? Is anyone working on answers? How can I get involved? How can I be heard? If drug trials are successful, when am I going to be able to access the therapy? What next?

The world of drug and therapy development is a complicated one full of questions. This new Global Genes toolkit series, From Molecules to Medicine, will help to break down the processes involved in bringing a therapy to market and answer common questions from rare patients and families.

The series includes an overview of how drugs and therapies produced; phases of clinical research required for a therapy to be approved and enter the market; and case studies that describe effective patient engagement at various points in the process. The goal is to help educate the rare community and to strengthen understanding of drug development and why it is essential for patients to get involved in clinical research and to share their experiences.

From Molecules to Medicine: How Are New Drugs & Therapies Developed? will shed light on the key elements and steps of the drug development process and answers questions like:

  • Is there a difference between the way drug development proceeds for a common condition versus a rare disease?
  • What is the approval process for a rare disease treatment?
  • What regulatory pathways help speed up the process for rare disease drugs?
  • What new technologies are changing drug development?

From Molecules to Medicine: Clinical Research breaks down each phase of the clinical trial process and describes its purpose. Prepare to learn:

  • What is the purpose of a clinical trial?
  • Who is involved in each phase of a trial?
  • How do I participate?
  • Why do some trials fail?
  • Where do I find results?
  • Are there new opportunities?

From Molecules to Medicine: How Patients Can Share Their Voices Throughout the Drug Development Process explores how rare disease patients can become involved each stage of the drug development process. Patients have many opportunities to participate, engage and even help define the process. This toolkit will cover how patients can use their voice in order to make advances for their community through the use of case studies. Explore topics like:

  • What is driving drug development?
  • How are communities being formed?
  • How can I encourage research?
  • How is researching being funded?
  • Is data being shared?
  • How can I participate in a trial or a committee?
  • How can I advocate for change?


Ready to get involved? Start with our Toolkit main page here and the Drug Development Roadmap.


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