Real World Data for Orphan Drugs: Meeting the Data Analysis Challenge

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Publication Date:
September 2020
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Creating meaningful insights from diverse data sources

The explosion in real-world data (RWD) from digital platforms is creating new insights into the understanding of rare diseases and patient experiences. The value and validity of RWD generated from registries, patient records, wearables and apps is increasingly recognized by pharma and stakeholders. Pharma's challenge now is less about data collection for their RWD programs, but more about how to effectively process and analyze data from the many varied data sources to create significant insights that inform and support patients, physicians, payers and regulators.

To explore the opportunities and challenges of harnessing the power of RWD for orphan drugs we interviewed rare disease and data collection/analysis experts. In Real World Data for Orphan Drugs: Meeting the Data Analysis Challenge they examine the current trends in RWD generation/analysis and the technologies, practice and strategies that are generating new critical insights.

Rare disease experts explore key questions:

  • How are regulatory authorities using RWD when assessing orphan drugs and what are its limitations in respect to regulatory reviews?
  • What benefits does RWD provide for payers in their assessments of orphan drugs?
  • What are the benefits and challenges of using a registry to collect orphan drug RWD?
  • What kind of data can electronic health records and claims data provide for orphan drugs?
  • To what extent can wearables and apps provide RWD for orphan drugs and what are their drawbacks?
  • What RWD can be found in unmediated social media channels and how is it best used?
  • In what ways will patients become increasingly influential in RWD generation?

What industry experts say...

"It's a lot of data….it is not a randomised controlled setting. It's all comers of data coming in. If the sample size is not right, and if you start to cherry pick on the sample size you could get a different answer than what was intended. If you utilise real world evidence, thinking that it's going to support what you've already seen in a clinical trial, then it has a lot of utility. But, if you were to notice a trend going the opposite way of what you expected, it would help you to pause, and think, what are the biases within this study that we devised? Not all data is collected equally, unlike in a randomised clinical trial. So, we have to be very thoughtful of that."
Sonal Bhatia

"Things are evolving really quickly. We've gone from being able to access electronic medical records to using some tools and technology to directly transmit, and translate, and upload that data straight into a patient case report form. EMRs also allow quick glimpses and low effort activities to identify things like how many patients are we actively seeing, how many patients have symptoms X, Y, and Z? It allows one to sort of scour through an entire hospital system to figure out whether these patients that we're looking for exist. A clinical trial might be looking for all kinds of different criteria and eligibility factors. Sometimes those eligibility pieces are decided without really knowing for sure if those patients really are out there walking around. So EMRs provide a really good reality check and a really good litmus test for us to confirm that there are plenty of the patients we are seeking for a clinical trial."
Scott Schleibner

"One can often better understand the natural history of the disease and disease progression via real-life registries. This information is sometimes lacking in rare disease, mainly because as soon as patients are diagnosed, they're put onto appropriate treatments. Therefore one cannot really understand fully the natural disease progression without treatment...this important information can be collected via registries"
Zoya Panahloo

What to expect

A detailed report exploring the current trends and future direction of the collection, analysis and use of real-world data in orphan drug clinical and commercial development.

  • An examination of 9 key issues that are shaping the collection and use of real-world data for orphan drugs
  • 25 targeted questions put to orphan drug and data experts
  • Their responses which provided 39 insights supported by 118 directly quoted comments

Deliverables: Includes PDF report and PowerPoint slide deck

Real World Data for Orphan Drugs: Meeting the Data Analysis Challenge is delivered as:

  • A detailed PDF report including all insights, quotes and intelligence exhibits
  • A useful PowerPoint slide deck providing a summary of the report's key findings for presentations and sharing with colleagues

Expert contributors

Experts contributing to this report have been screened to ensure they have:

  • Five to 20 years of experience in real-world data collection/analysis
  • At least five years of experience in the orphan drugs arena

Experts interviewed include:

  • Pedro Lendínez Ortega, KAM - Recordati Rare Diseases has over 14 years' experience in rare disease and orphan drugs. Pedro has worked in a number of rare disease areas, including cystic fibrosis and onco-haematology and possesses a wealth of hands-on experience from both the commercial and scientific side of the pharmaceutical industry.
  • Scott Schleibner, Senior Vice President, Center for Rare Diseases, PRA Health Sciences is an innovative clinical drug development executive with 25 years' experience in leadership roles across the CRO, biotech, and non-profit research sectors. He is passionate about leveraging technology to ensure clinical trials revolve around patients, reducing the burden of clinical trial participation and using data and advanced analytics to guide evidenced-based decision-making. Scott has had a varied career within rare diseases, working at a number of organisations in this area, with roles ranging from Clinical Operations Manager to Chief Operating Officer.
  • Dr Zoya Panahloo MD, Global Medical Director in Rare Diseases has worked in the pharmaceutical industry for over 20 years. She has held a range of medical roles, including Senior Medical Advisor at Roche, Head of Medical Affairs at CSL Behring and Global Medical Director, Rare Diseases at Takeda. She has published a range of articles on rare diseases and orphan drugs.
  • Sonal Bhatia, VP, Medical Affairs Lead for Rare Disease, Pfizer is an experienced Vice President with a demonstrated history of working in the pharmaceutical industry. Skilled in Medical Affairs across Cardiovascular, Diabetes, and Rare Diseases, she has over 15 years' experience in the pharma industry, starting as Vice-President, Medical Director for Cline, Davis & Mann and then moving to Pfizer. At Pfizer she spent 13 years in a range of medical roles such as Medical Director, Medical Affairs Product Lead and Senior Director, Global Medical Affairs.
  • Sheikh Usman Iqbal, Chief Medical Officer - Julz Pharma has over 15 years of broad-based health care experience, including clinical medicine, Global Medical Affairs, End-to-End Evidence and Value Development, Health Economics & Outcomes research across both large and small cap biopharmaceuticals. His pharmaceutical experience started at Amgen, where he held the role of Senior Manager Global Health Economics – Bone Health, and this was followed by several Evidence and Value Development roles at Sanofi. More recently, Usman has focused on rare disease with roles at Trevena Inc. and Acer Therapeutics.
  • Medical Lead, Rare Disease, Global Pharmaceutical Company is a trained physician with international long-term experience in Medical Affairs, Cardiology and Endocrinology, Diagnostic Imaging, Drug Research and Development, Orphan drugs and Global Disease Strategy. He has worked in the pharmaceutical industry since 1992 and has gained significant experience in rare disease and orphan drugs in that time.
  • Chief Business Officer, Biotech Company has worked in the pharmaceutical industry for over 30 years, holding a range of commercial roles at both large pharma and smaller biotech companies. His wide experience includes Product Marketing, Sales director and Chief Commercial Officer roles and, in the more recent term, he has focused on rare disease franchises.

To ensure full and frank opinions were expressed we have agreed to keep the names of some contributors anonymous.

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