Back home…

I’ve just landed back in the UK after the end of the World Orphan Drug Congress. I’m feeling rather jetlagged, but very happy with how it went. The quality of presentations on the last day was again excellent, particularly because of the patient focus.

There was a wonderful presentation from Jayne Gershkowitz, Senior Director of Patient Advocacy and Public Policy at Amicus Therapeutics, about how they have integrated patients into the development of their strategy and work. It was a shining example of how to ensure patients are at the centre of drug development, thanks in no small part to Amicus’s CEO, John Crowley, who has two children with a rare disease.

I was particularly impressed by how Amicus has set up a Patient Advisory Committee that is as important as its Scientific Advisory Group. They’ve set up systems to involve patients, taking into account their needs, and ensuring their voices are heard. It’s had a significant impact on the organisation by giving everyone a much more intimate understanding of the diseases and focusing everyone on what they hope to achieve.

I had the privilege of being on a panel on patient partnerships alongside Mary Cobb, Senior Vice-president for Membership and Organizational Strategy at the National Organization for Rare Disorders (NORD), Durhane Wong-Rieger, President of the Canadian Organization for Rare Diseases (CORD), and moderated by Penny Bemus , Vice-president for Business Development at Centric Health Resources.

We discussed the issues facing patient partnerships with industry. Everyone highlighted the need for industry to involve patients as early as possible if they want to develop a successful lasting relationship. We debated the rising influence of social media and online communities in the surge in the global rare disease movement and encouraged industry to get on board and interact.

I’m convinced that we’re at a tipping point in the rare disease movement. The work carried out by NORD, EURORDIS, CORD, other umbrella groups and numerous individual patient groups over the past decades is bearing fruit, amplified by the power of the internet and the increased interest from pharma companies. The challenge will be for us to ensure that this positive momentum continues and accelerates and that all actors – patients, industry, regulators and academia – work together.

I’m taking a week’s family holiday, so see you again after Easter!

Day 2 of the World Orphan Drug Congress USA

It’s the second day of the World Orphan Drug Congress in Washington. It’s been fascinating and an excellent way to get a better understanding of how the rare disease industry works. We’ve heard about the success of companies such as Genzyme, Shire and Novartis in their quest to find cures for rare diseases.

Most interesting is to see how industry overcomes the hurdles it faces. Time and again, they report that strong links with patient groups are a crucial factor determining the success of a drug development programme. For instance, in one instance, supply of a drug was proving problematic, and it was thanks to the support of patient groups that procedures could be put in place for a transition while another drug was brought to market.

I was particularly impressed by a presentation from the Alpha 1 Foundation. It’s a patient group that has raised more than $42m for research into Alpha-1 (a rare disease affecting the lungs), including grants to more than 70 academic institutions. It’s set up a non-profit company to help with the disease management of patients with Alpha-1, proving that there is a need to develop new hybrid business models for treating rare diseases.

John Crowley, one of the stars of the orphan disease movement, gave a keynote speech. John is the father of two children with Pompe disease, a devastating neuromuscular disorder. A book – The Cure – was written about his quest to find a cure for them, and then inspired a film – Extraordinary Measures – which starred Harrison Ford and came out in 2010. John is now CEO and Chairman of Amicus Therapeutics, a company that focuses on rare diseases. He talked about his journey and why focusing on patients is so important.

This afternoon, we’re hearing about latest developments in gene therapy clinical trials for rare diseases. I’m on a panel tomorrow about partnerships with patient organisations. So it’s a broad programme over all, and definitely one that would be useful for othe rare disease groups.

 

The World Orphan Drug Congress

I’m convinced that rare disease patient groups need to interact much more with industry. They need to go out there, attend the conferences, speak to the industry groups, find those with an interest in their disease, and work with them to develop clinical trials.

Of course, there are all kinds of hurdles to overcome, particularly relating to increasing government regulation around how industry can interact with patients in order to prevent conflicts of interest. But if you don’t go out their to dialogue with industry, you’re chances of helping your patients will be greatly reduced.

I’m attending the World Orphan Drug Congress in Washington next week. It will bring together hundreds of industry experts on rare diseases, as well as government institution representatives (such as the NIH) and some patient groups (but not enough).

I’ve been invited to speak as part of a panel on patient partnerships on the Friday of the conference. It promises to be an excellent debate and a particularly important one. Without the participation of patient groups, it’s much more difficult to develop effective treatments.

So if you’re a rare disease patient group, my advice is to find out what conferences are coming up, make sure you attend, then get networking.

The best place to find where these events are taking place is the CheckOrphan website.