> Click here to download the full press release

Shire is also a Platinum Sponsor at the upcoming World Orphan Drugs Congress Asia, held from 18-19 June 2013 at Grand Copthorne Waterfront Hotel, Singapore.

Healthcare at Home and QED partner on orphan diseases

OrphanReach alliance will see companies provide product lifecycle services

Healthcare at Home and QED Clinical Services have formed a strategic alliance under the OrphanReach banner to support the launch of products for rare and orphan diseases.

Homecare and patient support company Healthcare at Home and QED Clinical Services, a clinical research organisation (CRO), said the alliance would serve a unique market with a unique proposition.

Mike Gordon, CEO for Healthcare at Home, said: “OrphanReach will solve some key problems facing biopharmaceutical companies launching new medicines. It will ensure that pre-launch plans are data rich, evidence based and patient focused. It will ensure that market access is optimised and product launches are as successful as can be.”

Services offered by the UK firm’s new OrphanReach alliance will include clinical support and integrated patient management with an ambition to provide “the most integrated clinical development programme and launch plan within rare and orphan conditions”.

Healthcare at Home has 15 years’ experience of working in rare and orphan diseases and has supported the launch of many products across Europe, where it has been tasked with services that include running homecare arms in clinical trials.

Meanwhile, global CRO QED was founded in 2002 and has worked on more than 70 rare disease projects.

Its CEO, Thomas Ogorka, said: “We are delighted to be working alongside Healthcare at Home in this strategic alliance. We have a rich track record of optimising clinical development within rare and orphan markets and this alliance enables QED to take the range of services we can offer to a new level but more importantly support clients in a far deeper and more strategic way.”

QED is sponsoring the World Orphan Drug Congress 2013, taking place this year on the 14-15 November, at the Starling Geneva Hotel & Convention Center, Geneva, Switzerland. For more information about the event, please visit the website >

Siren Interactive

Content marketing is currently a hot trend, but in the rare disease space it’s been an essential tactic for years. I like the definition of content marketing as “the art of communicating with your customers and prospects without selling.” In the rare disease niche this is an important concept: educate, then sell.

If a pharmaceutical company can offer value to patients, they will gain trust, develop a relationship over time and ultimately obtain brand loyalty. In the rare disease space the way to add value is with information and tools. Patients and families want accurate and user-friendly information on common symptoms and how to get diagnosed and treated. They need tools that help them cope with the daily tasks of living with the disorder. They want to hear the stories of people like them. Unlike more common diseases, like diabetes, these resources are not available, so rare disease patients and caregivers look to pharma to provide them.

Providing value yields brand loyalty

Why is content so valuable for rare disease patients?
In many cases information is scarce. It’s estimated that only half of rare diseases have an advocacy organization. This leads to a huge gap in education and support.

1. It takes an average of nine years for a rare disease patient to get accurately diagnosed, according to the National Organization for Rare Disorders. These patients and caregivers spend many hours searching online for answers.
2. Many rare disease therapies aren’t easy to adhere to and require ongoing support. They may entail infusions administered by a healthcare professional, self-injections or multiple daily treatments.
3. Physicians may not know much about the disease or the available treatments. The constraints of time and training limit most physicians’ abilities to effectively diagnose and treat rare disorders. This leads to many rare disease patients and caregivers becoming unusually empowered. These patients can and often do play a lead role in educating physicians on various aspects of rare diseases. They look for the latest information about research, clinical trials and treatment options.

For all these reasons, patients welcome pharma’s participation as long as pharma is adding value by providing information, tools and support that can’t be found anywhere else.

Content is definitely king
In order to develop valuable content, your target audience must always be in mind—their needs, their concerns, their challenges. The content should address those relevant issues and utilize the tone and language familiar to that audience.

Just as critical as the message is the delivery. Since rare disease patients are often seeking support along their journey, email can play a key role. In addition, the number of people accessing information on smartphones and tablets continues to rise. Content calendars can effectively manage all patient touchpoints. Valuable content—whether it appears on a website, in an email or on a mobile device—should be optimized for its platform and easy to access for the target audience.

How are you using content marketing to build trusted relationships with patients and families?

For more information on orphan drugs and rare diseases, check out the World Orphan Drug Congress USA. Siren Interactive was a silver sponsor of the 2013 event.

Siren Interactive

Physicians are trained to arrive at a medical diagnosis by starting with the potential causes that are most common. The adage is “When you hear hoofbeats, think horses—not zebras.” This is logical and effective if a patient has a common condition, but the diagnosis of an orphan condition requires a different approach. The National Organization for Rare Disorders estimates that it takes an average of 9 years for a rare disease patient to get an accurate diagnosis.

This means that many patients, caregivers and physicians are online searching for answers, using search engines and forums. But, if you’ve ever tried searching Google or Bing for rare disease symptoms, you know that this is not effective. Enter Radu Dragusin from the Technical University of Denmark and a few colleagues who have launched a new search engine dedicated to the diagnosis of rare diseases called FindZebra.com.

Their study published in the International Journal of Medical Informatics shows FindZebra outperforms Google. The research explains that the algorithms of popular search engines (such as Google) favor websites that have a number of other websites linking back to them. These links validate that the information has been found useful to others. But by its very nature, information on rare disorders is typically scarce and has fewer backlinks.

How does FindZebra work?
The study notes: “It uses a specially curated dataset of rare disease information, which is crawled from freely available online authoritative resources. This means that FindZebra searches for rare disease information from a repository of ‘clean,’ specialized resources, unlike web search engines that search the whole web and are hence likely to return spurious, commercial and less relevant results.”

How does FindZebra compare to Google?
This blog post describes the success: “Evaluating on a set of 56 queries…Zebra easily beats Google. Zebra finds the correct diagnosis in top 20 results in 68% of the cases, while Google succeeds in 32% of them.” I did my own comparison and searched “purple urine,” which is a common symptom of a group of orphan conditions called Porphyrias. On Google, Porphyria doesn’t appear on the first three pages of the results, but for FindZebra it’s the second choice, with Acute Intermittent Porphyria showing as the seventh and eighth results.

On FindZebra is a warning that it is a research project and it is to be used only by medical professionals. It will be interesting to see how FindZebra evolves and if it gains wide adoption among healthcare professionals and patients. I know I’ll be using it. Will you?

For more information on orphan drugs and rare diseases, check out the World Orphan Drug Congress USA. Siren Interactive was a silver sponsor of the 2013 event.

Observed annually on the last day of February, Rare Disease Day is coordinated at the international level by the European Organisation for Rare Diseases (EURORDIS) and the National Organisation for Rare Disorders (NORD) in the US. The Rare Disease Day 2013 slogan is “Rare Disorders Without Borders” to convey the global solidarity of the rare disease community worldwide and the need for collaboration and mutual support in the field of rare diseases.

> Click here to download the full press release

Shire is a platinum sponsor of the upcoming World Orphan Drugs Congress Asia that is happening from 18-19 June 2013 at Grand Copthorne Waterfront Hotel, Singapore.

The much-celebrated orphan drug Kalydeco (ivacaftor) will be made available to the estimated 13 eligible patients with cystic fibrosis in Wales, confirmed Mark Drakeford, Welsh Assembly Minister for Health and Social Services, last Friday (May 10^th). Not only will this be great news for the manufacturers, Vertex, but it’s also great news for patients and those who campaigned for the drug to be made equally accessible across the UK.

“We are delighted that the Minister has recognised that patients in Wales should have the same access as people in the UK to this medicine,” said Ed Owen, Chief Executive of the Cystic Fibrosis Trust. “I would like to thank Mr Drakeford for listening to the voice of people with cystic fibrosis in Wales.”

The news comes after the All Wales Medicines Strategy Group (AWMSG) had recommended against funding the orphan drug – which is estimated to be about £180,000 per patient per year.

According to the Cystic Fibrosis Trust, about 4% of patients across the UK have the G551D mutation for which Kalydeco is licensed. In a statement, Vertex said they were encouraged to learn the Chief Pharmaceutical Officer in Wales has been asked by the minister to set up a review of the Welsh appraisal process for orphan and ultra-orphan drugs.

Are you pleased to see Kalydeco being made increasingly available in Europe?

This news comes amid reports that the orphan drug market is set to reach $127 billion by 2018. This infographic shows some more important facts about orphan drugs and rare diseases.

If you want to know more about innovation and strategy in the orphan drug market, you might be interested in attending the World Orphan Drug Congress Asia, 18-19 June 2013, Singapore, or the 4th World Orphan Drug Congress Europe, in Geneva on the 14th November 2013.

• The first and only FDA approved drug to target the defective CFTR protein
• Approaches and strategies to deploy biomarkers research in the orphan drug discovery process

World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.

For more information, go to www.terrapinn.com/orphandrug.

• Identifying and supplementing the natural history
• Developing endpoints that reflect the disorder, the impact of a therapy, and that can be utilized in multiple sites and acceptable to regulatory authorities
• Establishing the regulatory path
• Identifying patients (particularly when targeting subsets of patients (eg patients with a nonsense mutation)

World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development.

For more information, go to www.terrapinn.com/orphandrug.

Siren Interactive

If it seems like we are always asking ourselves this question, that’s probably a good thing. All relationships require nurturing and regular self-examination. The only way to create strong partnerships is to ask ourselves now and then what we’re contributing and whether or not it is what the other party in the relationship is truly seeking.

So what do patients want from pharma? The obvious answer is, of course, effective and safe treatment. That never has and never will change. But a recentstudy from Manhattan Research has me thinking about what else patients are seeking from pharma.

Patients want support
One thing patients want, it appears, is access to support. Thirty percent of online consumers with a chronic condition and 38 percent of caregivers are interested in registering for a patient support program, according to the Manhattan Research study. These programs appear to be working, since 75 percent of online consumers who used online pharma patient support programs said they feel confident the prescription they have is right for them or those they take care of because of these services. The most popular features mentioned by the 6,607 US adults in the study were financial assistance, meal plans and recipes, tools to track and manage a condition and a registered nurse hotline.
For rare diseases, which are often challenging and lifelong conditions, assistance like this may be especially valuable, so long as it truly meets the needs of the people who are affected–and providing that patients and caregivers know how to access assistance. Monique Levy, Vice-President of Research, notes that “…pharma isn’t top-of-mind as a destination, so marketers must consider partnering with other health resources such as general health websites, pharmacies and hospitals in an effort to gain traction for these programs.”

The key word is partnership
After last year’s World Orphan Drug conference, Melissa Hogan wrote a compelling post entitled “What We Want from (Big) Pharma,” on her Saving Case blog, that is definitely worth a second look. She talks about four things that caregivers want from companies that go beyond the foundation, which is excellent science:
- Transparency
- Commitment
- Compassion
- Partnership.

Here is how Melissa Hogan elaborates on the last one: “We see it as a partnership and expect to be treated as an equal partner in this process of trying to save the lives of our children. It’s not even necessarily ‘patient-centered’ relationships as that term is now being bantered about so much, it is partnered relationships, with parents, physicians, payors, regulators, and pharma as equal partners. We’ll play our part, and we’ll even give you guidance as to how to play yours.”

Know your partners’ needs
What do patients and caregivers want?  All you have to do is ask.  We are constantly impressed with how open-hearted rare disease patients and caregivers are, sharing their lives and stories to help others who face the same challenges…and how willing and even anxious they are to help us understand their needs.

There are rules and regulations to observe of course, but to echo Melissa Hogan, let’s not forget that we are truly partners in improving the lives of people with rare diseases. The relationship between patients, caregivers and pharma companies is one of mutual interest, but it also must be one of mutual respect.

And if you have any questions, just ask.

For more information on orphan drugs and rare diseases, check out the World Orphan Drug Congress USA. Siren Interactive was a silver sponsor of the 2013 event.

> Click here to download the full press release

Shire is also a Platinum Sponsor in the upcoming World Orphan Drugs Congress Asia, happening in Grand Copthorne Waterfront Hotel, Singapore from 18-19 June 2013.