FREQUENTLY ASKED QUESTIONS
Understanding how AFT Pharmaceuticals is improving access to medicines for people with rare diseases
Who are orphan drugs designed to help?
Basically people with rare conditions who are not getting access to the treatments they need. It’s not just a question of access to drugs though. For many people with rare conditions, finding, and then getting continued access to specialists with expertise in the relevant condition can be difficult. Once they are diagnosed, the problem for many people is that the available treatments don’t exist or are too expensive.
What are some of the challenges getting orphan drugs to people who need them?
Diagnosis initially. Then finding physicians who are on the leading edge of treating these small number of patients who suffer from highly challenging and specific diseases. Then actually getting the necessary drugs to them. The whole process can be really challenging for people with rare conditions.
Why is the Asia-Pacific region especially problematic when it comes to access to Orphan drugs?
The Asia-Pacific region has relatively few fully reimbursed markets and this causes financial hardship for patients who have to bear the brunt of funding their own treatment. In addition, the regulatory frameworks are diverse and, at this stage, don’t have a full understanding of orphans and how they are developed in the Western markets.
This situation is made worse when, generally speaking, orphan drugs can be expensive and as they treat only small number of patients the pricing is exacerbated by poor economics. For example, it’s often impossible economically to manufacture and label whole batches of drugs for only one country as most of the batch expires before they are used.
How does AFT Orphan work to get orphan drugs to people?
One way we do this is to partner with US and European companies who have made breakthroughs in therapy, but who don’t understand the unique challenges involved in bringing these drugs to Asian and Pacific countries. The challenges can be quite extensive. For example, it may require regulatory approval for a product with limited clinical data, or selling across multiple territories to improve commercial viability. It may mean making products available to ‘named patients’ in advance of registration, or conducting clinical trials, so that local physicians and patients can experience new drugs that haven’t been accessed before. So there’s lots of challenges in this part of the world, but we've picked up quite a lot knowledge along the way.
Do you partner with governments, companies or organisations?
Yes, we partner with government agencies. One example is the Early Point Project (EPP) status we were awarded by the Malaysian government (2013) to develop orphan drug manufacturing and to develop an export hub in Malaysia. And we partner with commercial companies to improve access across to orphan drugs multiple countries. We’ve also been an integral part of establishing (link to: http://apoda-orphan.com) the Asia Pacific Orphan Drug Alliance (APODA) which is comprised of pharmaceutical companies operating within this region who have expertise in orphan drugs.
What does the future hold for AFT Orphan?
AFT Orphan is looking to license its own portfolio of orphan treatments. This makes us well placed to target specific conditions that are not being adequately treated here. A number of these products also have the potential to be licensed to countries outside of the Asia-Pacific region. We being kept very busy through clinical studies, manufacturing (specifically for Asian and Australia and New Zealand patients) and working to break down the barriers to easier access for patients. And the key point is that we’re very much committed to Asia-Pacific in the long-term. Orphan drugs are quite a specialist area. Success requires trusted local relationships, having a sound medical understanding of relevant conditions, good connections, and a ‘can-do’ attitude. It’s definitely not a short-term game.