We keep our feet on the ground, our sleeves rolled up, and our ears open.
Let us share what we’ve learned and apply our thinking to your challenges.
Earlier this year, hundreds of advocates representing every state gathered in Washington, DC for the Rare Disease Week Legislative Conference. Their energy and enthusiasm raised the roof, as they shared experiences and prepared to visit their legislators on Capitol Hill.
Jean shares with the Rare Collective® her insights, experiences and advice that has helped countless others in our community become more effective in serving patients and organizations.
There are lots of ways to collaborate, but it all comes down to one thing: the patient organization will help you understand what it’s like to live with the disease. This helps the company understand what needs to be done to develop a therapy and get it to the patients who need it.
There are lots of ways to collaborate, but it all comes down to one thing: the patient organization will help you understand what it’s like to live with the disease. This helps the company understand what needs to be done to develop a therapy and get it to the patients who need it.
How many people live with a given rare disease? How many more remain undiagnosed? Can we develop a formula to predict improvement in diagnosis rates across diseases? A recent article by the International Society for Pharmacoeconomics and Outcomes Research attempts to answer these riddles.
Birthdays bring friends and families together to celebrate life, growth and progress. On the 11th anniversary of Rare Disease Day, the same holds true.
Clinical trials are one of the most critical steps in drug development. The Rare Collective took a step back to discuss the differences in clinical trials for orphan drugs and “mainstream” pharmaceuticals.
On May 19, the Wall Street Journal revisited industry and patient relationships leading up to FDA approval of Sarepta’s eteplirsen. Chris Smith weighs in.
Last month’s World Orphan Drug Congress generated discussion on a wide range of hot topics, from gene therapy to pricing policies. The Rare Collective™ offers their insights.
As hundreds of gene therapies advance through clinical trials, a genomic revolution is taking place before our eyes. Are we ready?
What is a rare disease therapy worth? David Lapidus discusses this pivotal question for pharmaceutical companies.
At a time of divided politics and diverse opinions, easing the burden of rare diseases is emerging as a cause that everyone can understand and support.
Jean Campbell reflects on why the patient voice needs to be not just heard but acknowledged.
When pharma treats patients as partners, it’s much easier to reach our shared goal of better health. In our final J.P. Morgan blog post, Chris Smith weighs in.
When it comes to orphan drugs, particularly gene therapy, the pricing problem stems from visibility rather than dollars.
At this year’s J.P. Morgan Healthcare Conference, David Lapidus spoke with biopharma investors about our nation’s new leader. Here’s what happened.
Doug Paul provides his perspective on pricing and reimbursement models for gene therapies.
Patti Engel talks about the importance of having experience in the rare disease space.
Doug Paul talks about the challenges that orphan drugs face after regulatory approval.
Too often discussions about the price of orphan drugs focus on the most basic unit of measure: a therapy exchanged for currency.
Time has run short for taking a comprehensive look at one of the most difficult topics in the industry—how best to regulate compassion.
Doug Paul dissects the challenges and future of the orphan drug market.
In this series, David Lapidus talks with members of the Rare Collective to debunk common myths surrounding rare diseases. In this post, Chris Smith flips the script on David for his thoughts, and David talks about the challenges of estimating a treatable population.
In this series, David Lapidus talks with members of the Rare Collective to debunk common myths surrounding rare diseases. In this post, Jean Campbell talks about when orphan drug companies should begin interacting with the patient community.
In this series, David Lapidus talks with members of the Rare Collective to debunk common myths surrounding rare diseases. In this post, Chris Smith shares how our understanding of rare disease is evolving, and why it may be a misleading term.
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