Rare Disease Patients and Pharma and Biotech Meet
Patients with rare diseases, and the parents of children with rare diseases are the experts on their diseases. How can they transfer their understanding, experiences, and challenges with their diseases, if they never meet with those working in Pharma and Biotech to develop new treatments for their diseases?
Researchers and drug developers working in pharma and biotech, benefit from discussions with patients with rare diseases, and the parents of children with rare diseases. However, it is often extremely challenging for them to simply meet with patients to explore the challenges with their diseases. Why is there a challenge?
There are many challenges. One of the key challenges is the vulnerability of the rare disease patient population. They are vulnerable because of their disease process. Great caution must therefore be exercised when approaching them. It is for this reason that Pharma and Biotech prefer not to take calls directly from patients, unless this is related to the safety of their products. Even then, the professionals taking these calls are experts in handling communications with patients. They are usually Pharmacists, Physicians or Nurses.
The Biotech and Pharma companies that have been the most successful in designing clinical trials for rare diseases, are those that have built into their drug development processes, mechanisms for meeting with parents with the rare diseases that they are studying and developing new treatments for. These mechanisms may involve bringing in a small group of patients into a conference room, and allowing the team to ask the patients questions. There are challenges with this approach, including the fact that most patients would feel intimidated. Another issue is that the patients selected may not represent the spectrum of the rare disease. The professionals may therefore receive an unrealistic, or incomplete view of the challenges patients face with the disease.
Putting Rare Diseases Patients First!(R) are experts in rare diseases. The organization has also developed relationships with rare disease patient groups over a period of almost ten years. There is a depth of knowledge of the challenges patients face, and also a deep understanding of the drug development process. This understanding allows PRDPF! to bridge the gap between the two spheres.
Putting Rare Diseases Patients First!(R) is offering a new offering that will allow it to bridge the gap between the two spheres, rare disease patients and Pharma/Biotech. Pharma/Biotech interested in meeting with representative patients can request help in setting up a meeting or series of meetings with the patients with a specific rare disease. PRDPF! will coordinate the meeting, which can be held virtually in the first instance, but may involve some face to face component in the future. There will be modest costs involved, but the transparency in this respect will help to ensure the potential for conflict is greatly reduced.
Rare disease patient groups and Pharma/Biotech can find out more by emailing Dr. Lorna Speid at LSPEID@PRDPF.ORG.
Read Dr. Speid’s popular publication on how Pharma and Biotech can better serve patients with rare diseases:
Speid, L. (2016), Don’t Do Different Things – Do Things Differently! Drug Development in Rare Diseases: The Patient’s Perspective. Clin. Pharmacol. Ther., 100: 336–338. doi:10.1002/cpt.403.
Books
Dr. Speid recommends the book, Clinical Trials: What Patients and Healthy Volunteers Need to Know, if you are considering entering clinical trials. It will provide you with the questions you need to ask the Investigators before, during and after you exit the clinical trial, and much more.
Purchase from Oxford University Press. OUP.US, OUP.CO.UK.
Diagnosis: Rare Disease
“Rare Disease” the diagnosis that no parent ever wants to hear. “This book gives a heartbreaking exposé of what life is like for families that have a member with a rare disease,” explains Carolyn Paisley-Dew, ISMRD Board Member.
Rare Like Us: From Losing My Dad to Finding Myself in a Family Plagued By Genetic Disease
Taylor Kane was a daddy’s girl from the moment she was born, smiling and cooing whenever her father was around and refusing to sleep until he held her in his arms. But shortly after she turned three years old, the unthinkable happened. Her father was diagnosed with a rare, genetic disease for which there was no cure. It wasn’t long before he began to experience a number of bizarre and frightening symptoms, and young Taylor watched helplessly as the disease ravaged his body and mind, transforming him into a shell of the father she once knew
When Gail Campbell Woolley was seven, a pediatrician told her mother that Gail suffered from sickle cell anemia, a rare blood disease, and that she would be dead by age 35. While others may have responded to this horrifying news by descending into a fog of self-pity, Gail went in the opposite direction. Read her life story.
Book Series About Sickle Cell
My Friend Jen Book Series.
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The main aim of our children’s books on sickle cell is to promote sickle cell awareness and to give young children with sickle cell peace of mind and confidence to not feel limited by their condition.