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.
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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?
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
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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