AFT Orphan Blog

Secure OD supply chains and PV reporting in Asia Pacific

Fri, 16 Mar 2012 04:24:00 GMT

Every company with a commercialized Orphan Drug has gone through the blood, sweat and tears required to register their product in either the US or EU. If that first registration was gained in the EU then every effort will be made to ensure introduction in the US as soon as possible - these two markets are the key global commercial opportunities. They also offer secure supply chains and predictable pharmaco-vigilance (PV) reporting in a way that ensures the minimum amount of risk is created through commercial activities.

Unfortunately it isn’t that simple in Asia Pacific and there are real fears concerning non secure supply chains and inferior pharmaco-vigilance reporting that might jeopardise marketing back home in the West.

The problem is that quality standards tend to break down when multiple partners are involved due to their varied (and at times insufficient) training on supply chain integrity and PV reporting. This is exacerbated when trying to market an orphan across wide geographies with partners using different reporting systems.

Whilst supply chain and reporting standards in some of the Asia Pacific countries such as Australia and New Zealand are just as high as anyway in the world there can be considerable variation across the Asia Pacific region. World class, multi-country, secure, temperature controlled specialist pharmaceutical distribution companies do exist but at present few companies can offer coverage of all territories – as a result careful partner choice becomes an immediate priority.

Taking into account all of the considerable complexities associated with commercialization and secure supply it is important for new entrants to be guided by partners that can advise across the whole region – taking a piece meal approach to supply chain integrity by allowing multiple partners to use their own distribution and partner networks can lead to confusion, poor PV, unnecessarily expired stock and disgruntled local partners.

Innovative solutions are possible and if you would like to learn more and make a conscious decision which benefits all parties please contact AFT Orphan via giles@aftpharm.com.

Following on............with Regulatory strategy for Orphan Drugs in Asia Pacific

Tue, 06 Mar 2012 09:57:00 GMT

Standard regulatory factors also need to be taken into account when formulating the overall Asia Pacific regulatory strategy. Some countries have relatively low governmental fees (e.g. Vietnam) whilst others are expensive (e.g. Taiwan and China) and this needs to be put into context of

The number of patients with the disease,
Their likelihood of receiving reimbursement, and/or
Their ability to self fund treatment.

It’s important therefore to have a good handle on the overall commercial viability before setting out on an exhaustive filing process.

Standard regulatory complications include the multitude of file formats required as there remains considerable variation across the region e.g. filings can be ICH, CTD, ACTD, or Chinese FDA depending on the country. There is also considerable variation in stability data requirements where zone 2 stability data may be acceptable for registration in New Zealand, Hong Kong and China for instance. In contrast zone 3 stability data will be necessary in some other countries and as of the adoption of the ASEAN guidelines all SE Asia countries are moving (at different paces) to the considerably more challenging zone 4b requirements of 30C:75% RH.

In the short term it is possible to file in some countries without all the necessary data being available - as long as specific assurances can be given. In other territories this is becoming impossible. It’s important for new entrants to be guided by partners that can advise across the whole region – taking a piece meal approach to registration can lead not only to missed patient and commercial opportunities but to soured relationships between individual distributors. Innovative solutions are possible where regulatory strategy is matched to secure supply chains. This gives reassurance to the orphan originator that their orphan arrives in the physician’s hands in perfect condition.

There are numerous options available for any Orphan Drug company – if you would like to learn more and make a conscious decision which benefits all parties please contact AFT Orphan via giles@aftpharm.com.

There are five main barriers (Regulatory covered this time) which reduce access for Orphan Drugs in Asia Pacific

Mon, 20 Feb 2012 09:18:00 GMT

The five main barriers for Orphan Drugs in Asia Pacific are (1) Market complexity (2) Regulatory strategy (3) Pharmaco-vigilance (4) Ethical marketing and (5) Access to skilled regional management. This article focuses on the area of regulatory strategy.

2. Regulatory strategy

Unlike the US and EU only a limited number of countries in Asia Pacific have formal orphan legislation. This means that in the majority of countries local knowledge relating to what is possible and conversely, what is likely to be impossible is vital for strategy formation.

Registration is necessary and advisable in some countries whilst for others taking on the administrative burden of registration is going to be counterproductive. Special access schemes (for patient access to unregistered drugs) are generally available across the region with most countries providing some form of administrative mechanism. There are however a few sad exceptions - for example in Thailand in 2011 Orphan Drug access proved extremely difficult without full registration – in fact even free humanitarian donations were blocked at times as the authorities couldn’t decide how to handle import duty on these free goods!

Formulating regulatory strategy can therefore be challenging and a number of factors need to be taken into account:

Government registration agency attitudes to Orphan Drugs.

The need in some countries to undertake local clinical trials to support the registration process.
Expected timelines for registration and pricing approval.
Existence of robust special access schemes.
Necessity to supply through local agents and/or semi government local organizations.
Waiver of importation restrictions.

The next blog will look at some of the standard issues that also need to be taken into account e.g. file formats and stability data.

There are five main barriers for Orphan Drugs in Asia Pacific

Thu, 26 Jan 2012 09:11:00 GMT

The five main barriers for Orphan Drugs in Asia Pacific are (1) Market complexity (2) Regulatory strategy (3) Pharmaco-vigilance concerns (4) Ethical marketing and (5) Access to skilled regional management. This article focuses on the first

Market complexity (part one)

The Asia Pacific region offers not only vast populations but also great diversity in how healthcare is delivered. There are for instance 4 reimbursed markets (Australia, New Zealand, South Korea and Taiwan) which have systems and processes in place that are recognizable from a western perspective. In addition there are 3 primary dispensing markets (Malaysia, Singapore and Hong Kong) where diagnosis and dispensing are handled by the physicians and the patient largely funds their own treatment. Then there are a number of large population prescription markets such as the Philippines, Indonesia, Thailand and Vietnam where a physician writes a script which is then dispensed in a hospital or retail pharmacy. Drug reimbursement in these markets can range from extensive, for instance in Thai civil servants to the virtually nonexistent elsewhere.

Each market within these broad categories requires different infrastructures, commercial tactics and at times specific local partners to ensure the best structure is in place for successful commercialization – market complexity and resource management are therefore a challenge for the most experienced of Asia Pacific specialists. Given the fact that many Orphan Drugs are commercialised by small biotech companies (with limited commercial experience of Asia Pacific) the result is that the challenges appear too daunting to tackle - quite understandable when you consider that the primary focus for these companies is success in the US and Europe (needed to keep their investors happy).

It is often argued that the reason Asia Pacific stands out badly is due to lack of funding. The vast majority of patients have to contribute, or indeed find all of the money, to pay towards Orphans Drugs from out of their own pockets. Ten or fifteen years ago families could not afford to help - but with the explosion of wealth at the top end of society and the creation of large populations of middle class citizens (with cash to spend) it is now clear that self funding or part funding of Orphan Drugs is feasible and does take place.

It is a little known fact that each country Health Ministry has well established special access schemes which allow importation of unlicensed medicines. Many in fact also provide unofficial funding for patients being treated in selected specialist clinics in university or top government hospitals. These Ministries are also aware that a full registration process is either unlikely from a commercial perspective or is not possible due to lack of specific orphan legislation and the differences in clinical proof available for orphans.

Innovative access opportunities for Orphan Drugs therefore exist across Asia Pacific – if you have an Orphan Drug and want to explore Asia Pacific please contact AFT Orphan via giles@aftpharm.com.

China is changing its attitude to Orphan Drugs

Tue, 03 Jan 2012 09:51:00 GMT

China is changing its attitude to Orphan Drugs – what used to be a barren waste land could become the next major Orphan Drug opportunity.

China has long been a difficult market for orphan drug companies - no conducive legislative frameworks, the need for local clinical trials and then, even with registration, more issues have been encountered with sampling of imported products on arrival at customs. For this reason few Orphan Drugs have successfully managed to enter a market where the WHO estimates there are more than 10 million sufferers of rare diseases.

Culturally the “needs of the many” have always outweighed the “needs of the few” but over the last decade the lack of access to modern Western drugs has unsettled this assertion and brought forward the real possibility of not only access but also reimbursement for Orphan Drugs. China is reforming the national healthcare system and health insurance plans with a “Bill of Rare Diseases” under review by the national People’s Congress (NPC) of China.

Proposals include:

Establishment or reimbursement mechanisms and succour networks for the diagnosis and treatment of rare diseases.
Active importation of Orphan Drugs by governmental agencies rather than taking a passive stance.
Support and encouragement of local companies conducting R&D into rare diseases.

These changes mean that a number of the barriers to Orphan Drug success in China are coming down opening the prospect that meaningful gains will be possible in China in the future. If you have a commercialized Orphan Drug and would like access to expert advice on market entry please contact giles@aftpharm.com.

This blog posting is part of a series of articles that focus on the opportunity to bring Orphan Drugs to patients in Asia Pacific.

Expanding patient access for Orphan Drugs in Asia Pacific

Sun, 11 Dec 2011 11:00:00 GMT

Expanding patient access for Orphan Drugs in Asia Pacific - vast differences in patient access to Orphan Drugs exist around the world and unfortunately Asia Pacific stands out for all the wrong reasons – there is a clear lack of access to these life changing medicines. For companies with commercialized orphans the patient opportunity is vast but the barriers they encounter can appear overwhelming.

Many are aware that over 370 Orphan Drugs are marketed in the US and a further 64 are marketed in Europe (as of May 2011). When looking at these lists it can be seen that there is little overlap between the two lists and therefore more than 400 marketed Orphan Drugs are potentially available to patients worldwide. Unfortunately only a small fraction of these orphans reach Asia Pacific patients.

Could the reason be that there is no patient opportunity for orphans in Asia Pacific? By definition orphan indications affect a small percentage of the population – but the reality is that vast populations live in the Asia Pacific region and this means many patients are not getting treatment. The figure shows that in the selected Asia Pacific countries below 2 billion people currently live (Vs less than 310 million in the US and less than 510 million in Europe). Even if we ignore China just three countries (Indonesia, the Philippines and Vietnam) account for more than 400 million people – many of whom are living with rare diseases.

If we extrapolate for one orphan indication e.g. we look at the prevalence of idiopathic pulmonary fibrosis (estimated from the literature at between 14-42 per 100,000 of population) we can see that somewhere between 275,000 patients and 826,000 patients are currently suffering from idiopathic pulmonary fibrosis across the region – this represents a huge potential patient pool with no readily available medicine.

Even the smallest countries such as Singapore and New Zealand each have between 600 and 1800 patients who could benefit from treatment if therapy was available and funding achievable. It is therefore clear that many patients could benefit from Orphan Drugs and that lack of patient opportunity is not the major reason for why we find very low penetration of orphans in Asia Pacific.

This blog posting is part of a series of articles that focus on the opportunity and the challenges faced when bringing Orphan Drugs to patients in Asia Pacific. If you would like more information about the series please contact giles@aftpharm.com.