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Insights from The World Orphan Drug Congress: Advancing orphan drugs and rare diseases

Insights from The World Orphan Drug Congress: Advancing orphan drugs and rare diseases


Maurice Leonard, Medical Affairs Director at Uniphar shares his key takeaways from The World Orphan Drug Congress, Barcelona. As a company dedicated to enabling small to medium biotech companies to launch their therapies in Europe, Uniphar’s medical team attended to join the conversation around advancements in orphan drugs and rare diseases.

What was your general reflection on the event?

The World Orphan Drug Congress is a really important platform for patient groups, researchers, and industry to join forces and exchange insights. I found the inclusion of many patient support groups very refreshing, offering a great opportunity to hear and understand their perspectives.

What were the hot topics of the event?

The keynote set the tone for the meeting and had real impact. For example, the presentation from Peter Marks (Director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration), offered interesting insights into crucial developments and ongoing discussions around global harmonisation for regulatory dossiers, and in particular for drugs that are being developed for orphan disease. This is a really important development, and while detailed specifics weren’t fully disclosed, there’s a lot of anticipation for an upcoming announcement.

There’s been a lot of work done to harmonise clinical development across regions through ICH. Recently there have been excellent initiatives such as the introduction of the international regulatory framework where there is a fast-track process for MAH holders who have been approved by ‘recognised’ regulatory authorities in different regions in the world. It’s great to hear about harmonisation within the regulatory arena.

Dr Marks also shared considerations being explored to support accelerated approval for Rare Disease Therapies such as incorporating surrogate biomarkers as endpoints into trials, rather than the hard conventional endpoints that have been traditionally requested and are much harder to demonstrate. There was discussion around initiatives like the Start program in the US, aimed at accelerating reviews for orphan molecules which highlights how the Agency are looking to prioritise rare disease.

The other key highlight to note came from Robert Califf, Commissioner at the FDA. He shared how his division in the FDA are working with the EMA and looking at establishing common methods between the two agencies. He noted that they are aiming to establish a cloud-based review in the future, which is likely to tie in to the harmonisation piece that Peter Marks referred to. Such developments would stop the need for individual dossiers being compiled and ensure more timely review processes. These things take time, but great intent has been shown by the FDA to drive things forward.

What are the biggest obstacles facing pharma companies in terms of ensuring equitable access to medicine?

The current legislation appears to lack sufficient incentives to motivate drug developers to ensure that they will bring the therapy to all markets. Looking at the access that patients have across Europe, you can see there’s an imbalance, there is a very clear socio-economic split.

In terms of availability, part of the pharmaceutical legislation reform was to try and level the playing field. Despite efforts to reduce market exclusivity to a certain extent, the reform aims to offer incentives for addressing high unmet medical needs and also offers incentives for conducting R&D in the EU. Are the incentives enough to drive change? My concern is that the incentives outlined in the policy documentation may not translate into a significant impact during implementation. Stronger incentives, such as extended exclusivity periods for R&D, could potentially transform when and where clinical trials would be conducted and consequently allow patients across Europe earlier access to innovative medicines.

Some incentives provided for drug developers targeting orphan diseases look promising – such as granting an additional two years of exclusivity for drugs addressing high unmet medical needs. However, the criteria for eligibility lacks clarity. My fear is that a rigid criteria may prevent many from accessing these incentives, so further clarity is needed here.

How can Uniphar support pharma and biotech?

Uniphar is well-placed to support the whole industry. We deliver highly effective solutions and services across the product and brand lifecycle, and boast a robust global supply chain.

“Nearly 50% of launches in Europe are being managed by biotech.”

Uniphar can work with small to medium biotechs, and help launch with ease in Europe. We offer just one touchpoint to manage that, rather than a network of vendors which becomes unmanageable.

We’re also anticipating a rising demand for our brand fostering solution as portfolios evolve over their lifecycle. With a heavy focus on recent launches, some brands will be potentially divested, and companies will require third-party support to effectively manage these.



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