Orphan Drugs: Drivers of Market Growth (2019)

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Publication Date:
August 2019
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Orphan drugs: Building your profits or draining your resources?

Substantial growth prospects in orphan drugs (more than double the overall pharma market) have enticed many pharma companies to enter the fray. But all have faced challenges. From problematic relationships with payers and regulators to knowledge and evidence gaps, the obstacles in the way of success are numerous. So what are the most effective strategies for achieving the best results today?

In Orphan Drugs: Drivers of Market Growth eight senior orphan drugs experts from across the world share their views and experience and offer their recommendations on how to drive orphan drug market growth to the next level. 

Use this report to:

  • Make sense of the chaos: There’s plenty of noise around orphan drugs but what are the current drivers of market growth that pharma should be taking notice of and proactively pushing?
  • Build strategies to suit objectives not people: It’s easy to get distracted when payers, regulators and patient stakeholder groups all seem to be pulling in different directions. This report shows the areas of common ground and reveals a clear way forward for pharma.
  • Examine your motivation: Take time to understand which activities are driven by short term financial objectives and which are more likely to deliver better overall results over the longer term. You can then adjust your approach accordingly. 
  • Strengthen your growth plan: What are the most attractive opportunities being created as a result of advances in clinical research, evolving orphan drug legislation, increased competition and awareness? Make sure your thinking is right up-to-date.

What to expect from this report

Payers and regulators are keeping a close eye on pharma activities in the orphan drugs market, in part due to a few incidences where incentives have been abused. But increased scrutiny isn’t necessarily a bad thing because for some it presents the opportunity to stand out for doing the right thing. Orphan Drugs: Drivers of Market Growth makes clear exactly what the right thing is and offers practical recommendations on how to improve payer and regulator engagement. Those that do will move ahead of the pack.

How did we do it?

  • We identified 9 current issues affecting the orphan drugs market worldwide
  • We explored these during in-depth interviews, posing 17 targeted questions to 8 experts
  • We uncovered 54 unique new insights during these detailed discussions
  • Our unique insights are supported by 134 directly quoted comments

Example insight included in Orphan Drugs: Drivers of Market Growth

"Gene therapy is a space to watch for future orphan drug market growth." In recent years, major breakthroughs in science such as genomics and gene-editing technologies have facilitated the development of gene therapies, capable of precisely treating and even curing rare diseases. Whilst only a few have gained approval so far, there are many gene therapies at various stages of development. There is considerable scope for growth in this area and those companies that invest in gene therapy will be the ones to reap the long-term rewards."

Example quote included in Orphan Drugs: Drivers of Market Growth

"Regulatory agencies see many dossiers from many companies, so they are not experts in a specific area. Therefore, we create dossiers that have a lot of educational material therein. At the same time, we try to work with partners who have in-depth expertise within specific disease areas to inform and train regulators in a compliant and objective way. This is really important as it helps them understand the severity of those diseases and their impact on patients, caregivers, the economy, and on society."

The expert panel for Orphan Drugs: Drivers of Market Growth

  • Júlio Cesar Avella is the Biotech Unit Director (Fertility, Neurology, Immunology and Endocrinology) at the Merck Group, Brazil.
  • Claudio Marelli MD, F.A.C.C, Global Medical Lead Rare Metabolic Diseases, Takeda Pharmaceuticals Limited, Zug Switzerland.
  • Jerry Martell is the former Regional Business Manager of Rare Diseases at Shire, US.
  • Hector de la Riva is the Associate Director of Marketing for Gilead Sciences, Spain.
  • Parth R Shah is the Commercial Director (Haematology and Oncology) for Amgen, US.
  • Dr Andreas Trinkl is the Business Unit Director Haemophilia DACH at Swedish Orphan Biovitrum AB (Sobi), Germany.
  • Anonymous is a Global Marketing Director for Johnson & Johnson.
  • Anonymous is a Market Access Expert who has worked in the pharmaceutical industry for 10 years.

Why buy now?

There are usually no effective shortcuts to success in life and the same is true in orphan drugs in spite of the hugely attractive potential gains. As more pharmaceutical companies embrace rare diseases as the next big opportunity, legislators and payers ramp up their scrutiny and competition becomes more intense. It’s a volatile mix and it pays to keep a cool head when evaluating the current landscape, building longer term strategies and committing valuable resources. Get it right and the rewards are excellent; get it wrong and face financial and reputational damage.

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