Social Media for Orphan Drugs

A pharmaceutical agent that has been especially developed to treat a rare medical condition is known as an ‘orphan drug’.
In concord to the name given the medical condition being treated is also referred to as an orphan/rare disease. Rare diseases are defined differently in diverse parts of the world. In the European Union Life-threatening or chronically debilitating conditions affecting no more than five in 10,000 people are called rare diseases (about 1 in 2,000 people). In USA rare diseases are defined as any disease or condition that affects less than 200,000 persons in the United States (about 1 in 1,500 people). In Japan, the legal definition of a rare disease is one that affects fewer than 50,000 patients in Japan (about 1 in 2,500 people).

The designation of orphan status to a disease and the drugs developed to treat it is generally a matter of public policy in most states.
Governments and rare disease patient advocacy organisations such the U.S.'s National Organization for Rare Disorders (NORD) and the European Organization for Rare Diseases (EURORDIS) have emphasized the need for fiscal incentives to encourage Pharma companies to develop and market medicines for the many ignored and "orphaned" rare disease patients. The Orphan Drug Act (1983) and European legislation for orphan drugs passed in 2000 has addressed this requirement and provides major incentives for pharma to engage in production and R&D for orphan drugs.  

Social media and orphan drugs

However, simply passing legislation for orphan drugs is not enough; these drugs need to be provided to qualified patients along with information on latest research and developments. This is where social media steps in.
Previously the search for orphan drugs for rare diseases was limited to going to a specialist and depending upon his expert advice, but with the recent explosion of Web 2.0 technology patients and their families can log on to the internet and share stories, experiences; research latest drugs and trials; order prescriptions and or locate retailers for orphan drugs and much more.
Presented below is a detailed SWOT analysis of the benefits of social media in orphan drugs.

Surf with care

Social Media now provides a niche for every group of people with likeminded ideas, needs and philosophies. Thanks to increasingly sophisticated social media, search tools and databases, patients are able to be informed and connect with others. Skype ,Blogs , Twitter, Facebook and Flickr provide ample oppurtunities to connect with stakeholders in the orphan drug sceanario.Paradoxically, the amount of information and exchange opportunities on the web can be overwhelming and increasingly difficult to exploit. Although it is now extremely easy to access information on the net it has also become necessary to validate that information and exert caution in relying on unverified blogs and posts.  
Patients and their families can keep abreast of specific areas of interest via newsletters, subscriptions and tools such as RSS feeds, bookmarks, Google lists and searchclouds.
Governments are also participating in social media through RSS feeds of the European Medicines Agency (EMA) and US Food & Drug Agency (FDA) websites and the FDA's "Track" system, where one can follow anything that passes through FDA through an e-mail alert system. (Tejada, 2010)

Popular social media sites

For many rare diseases, available information is seldom adequate.
Health professionals are often deficient in appropriate training and awareness to be able to diagnose and adequately treat these diseases. The low incidence of these diseases allows only a limited number of health professionals, usually in specialized centers, to build expertise with diagnosing and providing medical care to people affected by such a rare disease.
Here the internet is a fount of information.
For patients and doctors looking for information about specific rare diseases and orphan treatment, Orphanet is the best medically validated resource and, NORD as well as Genetics Home Reference are still some of the best databases available today. Big clinical trial gathering sites such as: clinicaltrials.gov, trialreach.com, clinicalresearch.com, PubMed, hon.ch, IFPMA and NIH's Medline Plus and NCCAM are also popular for orphan drug research information.
Furthermore, individual subscription sites like the Kaiser Family Foundation and the Center for Disease Control and Prevention are also popular drug R&D sites.
Many of these orphan speciality sites including Unique, Raredisease day etc are actively linked to social networks like Facebook, Twitter, Flickr, YouTube and Linkedin etc. Making it easier for people to share their stories with others and exchange experiences with similarly affected patients.

The unique website is a popular resource for patents and other orphan stakeholders. Key features of the site include (Betuw, 2010):

1. >7300 members, 77 countries
2. Hard copy magazine posted >3570 member families
3. Electronic copy emailed to nearly 3000
4. Discussion forum website 2500 members
5. 3658 fans on Facebook, 50 new fans per week
6. 1200 – 1500 visitors to Facebook page each week
7. 130 followers on Twitter
8. >9000 unique visitors website per month
   
   
   

Social media usage

Pharma companies use social media for orphan drugs to manage and reach stakeholders including doctors' patients and caregivers; to inform them of where to get reliable information; gather their ideas, priorities and interests, and connect them with other professionals and patients with related interests and expertise. The benefit of such networking includes:

1. greater collaboration between stakeholders
2. connecting patients and professionals with common interests
3. ease in recruiting rare disease patients for clinical trials
4. Improve internal processes with input from stakeholders
5. Increase the effectiveness and efficiency managing regulatory compliance
6. Enable doctors and patients to more easily access needed information
7. Increasing the efficiency in the delivery care through innovation and collaboration

When such media is meaningfully managed (media is interactive and patient centric) then the adoption by stakeholders is high; however (Fig 1) social media strategy for healthcare and especially in/ orphan sector requires a change in behaviour from professionals and pharma to encourage adoption by patients.

For example many pharma companies have started their own Twitter accounts (Fig 2) and have a considerable following, however the follow back policy of many  companies is inadequate (Fig 3) where follow back = listening and caring.

Figure 3

Viz. simply creating a presence on the social networks is not enough; stakeholders must actively contribute and listen to the conversations online to maintain connectivity.
This includes regular updates and real time interactions between patients, doctors and pharma. Specifically in the case of orphan drugs active contribution is required from regulatory bodies such as FDA, NORD, EMA and EURODIS to regulate and monitor the usage of social media.

Social media in Global access programs

Governments have established systems that permit patients with unmet medical needs to access life saving orphan drugs before their authorization for general marketing. Unapproved orphan drugs are delivered through Expanded Access Program (EAPs) in the US and Named Patient Program (NPPs) in the rest of the world. NPPs are controlled and restricted access program SET up by pharma companies under which doctors and pharmacists can access trial therapies on a 'named' patient basis. Here social media is of particular use in making and sustaining links between pharma, doctors and patients for the enrolment in trials and access to unapproved drugs.

Social media sites such as Facebook and twitter can be used for sending out alerts, updates and feedback for the special access programs. Closed social media sites may also be put up which allow only disease related patients and caregivers to participate in special programs. Furthermore social media in global access management solutions helps companies in recruiting trial participants; SM can help reach a small patient population that may be distributed across the world. Seeking approval in dozens of countries may be impractical; access management solutions powered by social media would help companies reach such populations effectively. Social media also eases the process of handling and vetting requests; helps in reviewing physicians (information on professionals is widely available in social media) requesting the drug and procedures and the handling of adverse event reporting via pharma blogs, updates and tweets.  (Estcourt, 2010)

Regulating social media

The FDA attempts to regulate health-related information produced by social media, with the input from patients and other stakeholders. According to Rob Camp “For me, the take-home message was that we, as patients and patient representatives, cannot be sitting idly by; but taking part in it all, and voicing our opinions, which are listened to! There are pros and cons to all social media, and patients and patient representatives need to be part of finding a balanced solution - or laws will be made and applied where we have not had input." (Tejada, 2010)  ongoing reforms are also being made to the European ‘Information to Patients’ legislation and debated the necessity for European policy makers to think globally by working with the FDA in initiating specifically ‘patient-focused’ regulations regarding social media marketing by pharma. It is clear that, while social media offers opportunities to information-seeking patients, these should be balanced with respect for the legal principle banning direct-to-consumer advertising orphan medicines, or unscrupulous exploitation of patients by encouraging risky trials and treatments.

European scenario

Although access to high quality health information is fundamental to achieve good management of orphan disease and its treatment, European rare disease patients continue to suffer from the inaccessibility of quality information. To make an educated choice between the bad and the ugly, and to participate in treatment decisions patients frantically seek quality information. Often the consultation time with the doctor is too short to clarify all essential questions and patients feel the need to connect with fellow sufferers to share stories and information. However in most cases information in local language is unavailable, outdated or just too technical. Thus most patients then end up searching internet sites to find relevant information on available drugs and treatments for orphan diseases. Patients with orphan disease are particularly isolated and vulnerable without effective access to information; unlike the US, Europeans do not use a common language and are disadvantaged by the lack of both social and official media in regional languages. Only those with a grasp of English can search and verify orphan therapies currently available and participate actively in orphan communities. Furthermore, European legislation on ‘Information to Patients’ is confusing and a comprehensive EU strategyon ‘Information to patient’ is required.

For example individual member states (and possibly pharma industry) can have very different guidelines for treatment of certain conditions with the same product; such as the use of hydroxocobalamin being prescribed for Pernicious Anaemia. In some EU Countries the standard treatment guidelines would be once a month IM injection whilst other EU member states recommend the same product to be used only once every 3 months. In some EU member states hydroxocobalamin is available over the counter at any pharmacy whilst in other EU countries the same product requires a prescription. This irregularity further increases confusion amongst patients and professionals.

Therefore it is necessary that core quality principles developed by the official bodies provide a framework that should be strictly adhered to by all information providers, whether official, public or private stakeholders. Some successful examples in Europe include the FASS portal in Sweden which provides information about all prescription medicines in Swedish lay language, including trial results, safety information and packaging information. The need of the day is not just reliable information sites but also social media easily accessible in regional languages (Geissler, 2010)

Conclusion

The SWOT points towards the many Strengths and Opportunities to be had through social media. Although some Weaknesses and Threats do endanger this movement these can be countered through monitoring technologies and judicious use of this medium; overall the Strengths and Opportunities outweigh any negatives for the adoption of social media in the promotion of orphan drugs. Social media has emerged as a vital medium of connection for all stakeholders in orphan drugs and diseases. Healthcare professionals, patients and pharma all over the world need to involve themselves in the new era of ehealth where digital connectivity is a must. An online presence is the best way to become visible and take active part in orphan disease treatment and management scenario. Orphan drugs play an extremely important role in the life of patients and their families; most patients of rare diseases suffer from great isolation and emotional anxiety along with the obvious physical drawbacks. Social media connects them with valuable resources and updates on the latest developments in this sector and as such social networking has become an important part of orphan drug R&D, accessibility and distribution to patients, caregivers, doctors and pharma.

Bibliography

1. Ariyanchira, S. (2010, July 15). Big Pharma Steps Into Orphan Drug Market. Retrieved October 20, 2010, from GEN: http://www.genengnews.com/gen-articles/big-pharma-steps-into-orphan-drug-market/3329/?page=1
2. Betuw, A. v. (2010). Taking part in the online evolution. Satellite Workshop Be Connected, Be Informed. Krakow: Eurordis Conference.
3. Estcourt, S. (2010, October 12). Need for speed. Retrieved November 9, 2010, from PM Live: http://www.pmlive.com/find_an_article/allarticles/categories/General/2010/october/features/need_for_speed
4. FDA. (2009, July 28). Designating an Orphan Product: Drugs and Biologics. Retrieved October 18, 2010, from U.S. Department of Health & Human Services: http://rarediseases.about.com/gi/o.htm?zi=1/XJ&zTi=1&sdn=rarediseases&cdn=health&tm=209&f=00&su=p736.9.336.ip_&tt=2&bt=1&bts=1&zu=http%3A//www.fda.gov/orphan/progovw.html
5. Geissler, J. (2010, April). Information to Patients Debate 2010 – as if the Internet was still a walled garden. Retrieved October 27, 2010, from Rare Diseases Blogs: http://www.rarediseaseblogs.net/2010/04/21/information-to-patients-debate-2010-as-if-the-internet-was-still-a-walled-garden/
6. Tejada, P. (2010). Finding information on the Internet. EURODIS.