Advocacy

In advance of Rare Disease Day 2026, GHMC convened its International Advisory Panel* for a global discussion on the realities of working in rare diseases. With experience spanning dozens of rare conditions across multiple markets, our teams reflected on this year’s theme, “More Than You Can Imagine.”

There are more people with rare diseases than you can imagine. Despite each individual rare disease having a small population, once combined, 1 in every 17 people is thought to have a rare disease. This adds up to millions of people.

Rare diseases also demand more than you can imagine: more empathy, more flexibility, more patience, and more strategic precision. They also offer more than you might expect—deeper partnerships and powerful patient stories that give us the opportunity to create meaningful change for communities that are too often overlooked.

From a global perspective, several themes related to biopharma industry communications seem universal: pricing, regulatory approval, funding, advocacy and other aspects. However, in rare disease these themes are also more nuanced than you can imagine, especially when working across markets and region

1. Emotional sensitivity is not optional

Rare disease patients and families often live with prolonged uncertainty—delayed diagnoses, limited treatment options and difficult access pathways. Communication about clinical trials, particularly ineligibility, requires exceptional care because every interaction has the potential to carry significant weight.

2. Marginalization and stigma are real

In many countries, rare disease communities have limited advocacy infrastructure. Stigma and social isolation can compound medical challenges. Communications must be culturally sensitive, locally informed and rooted in respect.

3. Access pathways are rarely straightforward

Reimbursement and regulatory environments vary dramatically. From alternative funding mechanisms in Singapore to evolving health system integration in Argentina and lengthy processes across parts of Latin America, rare disease innovation often moves through non-traditional and highly scrutinized pathways. Strategy must reflect this complexity.

4. With small patient populations, every story is consequential

Some conditions may affect only a handful of individuals in a given country. When numbers are this small, communication becomes both more personal and more consequential. Ethical storytelling and privacy considerations are paramount.

5. Foundational education is often the first step

Rare diseases frequently lack baseline understanding among policymakers, payers, clinicians and the public. Before driving awareness or action, communicators must often establish the fundamentals—what the disease is, how it presents and why it matters.

6. Pricing and public debate require thoughtful navigation

Therapies—especially those involving children—can attract intense media scrutiny. Public discourse around pricing and reimbursement can become emotional and politicized. Clear, balanced communication grounded in patient need and long-term value is essential.

7. “Rare disease” is not one category

There are thousands of rare diseases, and only a fraction have approved treatments. Many patients lack representation or advocacy support, leading to varied levels of engagement across disease types. Each disease area demands a tailored approach rather than a one-size-fits-all strategy.

8. Depth matters more than breadth

Traditional awareness metrics don’t always apply. Broad reach may be unrealistic and unnecessary. Success in rare disease communications is often measured by depth of engagement, strength of relationship, and trust built within small but highly connected communities.

9. Patients are partners, not spokespeople

Listening to patients’ diagnostic journeys and supporting them in telling their stories—on their own terms—builds authentic advocacy. Empowerment strengthens not only communications outcomes, but also community resilience.

10. Community-building drives impact

Grassroots campaigns have influenced policy decisions and accelerated inclusion of therapies in national programs. Collective advocacy amplifies voices. Visual storytelling and, when appropriate, public figures can help reduce isolation and broaden understanding.

Rare disease work requires more imagination, more patience and more responsibility than many other therapeutic areas. But it also offers the chance to make a disproportionate difference.

Need help interpreting global complexity for your brand? If you’re navigating a rare disease innovation and need experienced, globally informed communications support, we’d welcome the conversation.

* The GHMC International Advisory Panel brings together a curated network of senior healthcare communications experts from across more than 20 key global markets with more than 200 years of combined experience. The panel serves as a real-time sounding board for the most important trends, challenges and developments shaping the international healthcare landscape.

Designed to move beyond theory, the Advisory Panel translates global insight into practical guidance, helping organizations anticipate change, stress-test strategies and stay ahead in a rapidly evolving health ecosystem.  We stay connected to share insights and perspectives between different markets, but we all share the same desire to connect medical advances with those who need them.

As my first week representing VIVA! Communications, Australia as part of the GLOBALHealthPR Professional Exchange Program at Spectrum in Washington D.C. drew to a close, my head was spinning with all of the health PR-related information I’ve recently gleaned.

This week I’ve participated in several interesting brainstorming sessions focusing on campaign concepts and new business development. Observing the communications process involving Spectrum team members across the U.S., from Washington D.C., and New York to Atlanta, and those working from ‘virtual’ offices at home, has proven a truly fascinating exercise.

I was also granted the opportunity to meet with some of the business leads at Spectrum D.C., including Public Affairs lead, Al Jackson, who explained the pivotal role that communication plays in U.S. health and medicines-related politics. Citing several case studies, Al broke this somewhat complex subject matter down with ease, affording me unique insights into the invaluable role that both he, and the Public Affairs division plays at Spectrum, to ensure fairness of communication for U.S. healthcare providers, pharmaceutical companies and patients.

The highlight of my week was obtaining a glimpse into U.S. healthcare policy and public affairs. On Tuesday, I visited Capitol Hill to observe a U.S. Senate hearing into health policy, focusing on the potential ramifications of introducing Food and Drug Administration (FDA) approval for laboratory-developed tests (LDT’s) of a certain type of in-vitro diagnostic. Currently, no such approval is required.

The hearing armed me with fascinating insights into the American approach toward deliberating the role of administration, and government in diagnosing illnesses and delivering precision medicines to the public. Those in favour of FDA regulation (primarily the Democrats) argued LDTs had not been sufficiently validated, and were therefore at risk of inaccuracy, and that patients are not informed that their tests fail to undergo rigorous FDA approval. Those Senators opposed to drug regulation claimed that such approval would slow the process of diagnosing illnesses and introducing life-saving medications to market. Furthermore, those against (mainly the Republicans) stated the labs (including branches of leading care centres and research hospitals) would go bankrupt if they had to pay for such regulation.

Further to this, I was able to meet many members of the Spectrum team, including Kaitlin Bowen, who, like me, is participating in the 2016 GHPR exchange program. This allowed us a great opportunity to chat about the program, and I was able to provide some insight as to what I had uncovered about Spectrum, while allowing Kaitlin to ask questions about VIVA!, and how we work in Australia.

As a former journalist, I also really enjoyed my visit to D.C.’s Newseum, which offered me a great insight into how journalism has been involved in capturing and shaping American history. Each of the exhibits in the Newseum offered fascinating insight into the history of US news reporting, and allowed me an idea of some of the most iconic news stories ever published in the US.

For me, it was amazing to learn about the role that the first amendment has played in the history of news reporting in the USA, while also being allowed to recognise the sacrifice made by many journalists in telling the story, with a wall of remembrance to those who had lost their lives in the service of news reporting.

On Thursday, I was given the opportunity to present about my work, and the way we work at VIVA! Communications in Australia to a “full house” at the D.C. office brunch and learn.

The response from the team who tuned in, and listed to me talk was really great. It was really humbling to listen as the team responded to the presentation, with lots of questions being asked, and great feedback from everyone.

And, on Friday, my final day in Washington D.C., I was granted a fantastic opportunity to visit the advocacy office of the American Cancer Society (ACS), and to spend some time with Bob Chapman, Managing Director of Global Relay for Life, who provided me with some phenomenal insight around the history and aims of ACS and how it works to achieve its goals, both in the U.S. and globally.

After meeting with Bob, he invited me along to listen to a talk being given by Uruguayan cardiologist, and one of the survivors of the 1972 plane crash in the Andes mountains, dubbed the “Miracle of the Andes”, Dr Roberto Canessa.

Listening to Dr Canessa talk was captivating, as he shared the story of his survival, and the impact it had on his life moving forward. It was definitely a highlight of my time in D.C.

Every day I gleaned new and invaluable insights into the U.S. health communication marketplace. I can’t wait to see what unfolds this week in New York City.

To Jonathan, Andrew and all of the team in D.C. I want to extend my wholehearted thanks. You were wonderful hosts, and provided me an opportunity, friendships and an experience I will remember for the rest of my life. Thank you all for that.

Be sure to follow my exchange with GLOBALHealthPR at #GHPRConnects.

Recent changes to medicinal marijuana laws and regulations globally have expanded opportunities for medical research and patient care.

These actions are part of a broader global movement of governments and advocates to recognise the potential medicinal benefits of marijuana, which has long been considered solely a harmful recreational drug. These purported medical benefits include aiding in the treatment of  epilepsy, PTSD, chronic pain and more.

Here we present three examples of GLOBALHealthPR member countries – Australia, Canada and the United States – that are tackling the legislative issues surrounding the use of medical marijuana.

Australia:

Last month, New South Wales (NSW) became the first state in Australia to approve legislation that legalises marijuana for medicinal purposes. The Poisons and Therapeutic Goods Act allows doctors to request approval from NSW Health to prescribe medical marijuana to patients who “exhausted standard treatment options.”

The announcement follows extensive trials of marijuana as a medicinal therapy. The Hon. Mike Baird MP hoped to make NSW a global leader in medicinal marijuana research and rallied for it to be accessible for those in need.

“People who are seriously ill should be able to access these medicines if they are the most appropriate next step in their treatment,” said Mr. Baird announcing the legalization of medicinal marijuana on July 31.

The NSW government is also establishing the Centre for Cannabis Research and Innovation, which has been granted $21 million over the next four years to conduct medicinal marijuana trials. Other Australian states, including Queensland, look to follow suit, with multiple trials studying the medicinal benefits of marijuana occurring in the Sunshine State for children living with drug-resistant epilepsy.

Look here to learn more about how medicinal marijuana will be used therapeutically in Australia.

Canada:

Canadian regulations of medicinal marijuana have been in a state of flux over the years. In 2014, the Marijuana for Medical Purposes Regulations (MMPR) established Canada’s first commercial industry of highly-regulated, licensed producers (LPs) of medicinal marijuana, replacing the previous Marijuana Medical Access Regulations (MMAR). To date, 35 LPs are authorised to produce dried marijuana for medical purposes.

In June 2015, the Supreme Court of Canada (R. v. Smith) decided that restricting legal access to only dried marijuana was unconstitutional. As a result, some of these producers are also authorised to produce and sell fresh cannabis and cannabis oil.

The Federal Court of Canada found that “requiring individuals to get their marijuana only from licensed producers violated liberty and security rights protected by section 7 of the Canadian Charter of Rights and Freedoms,” (Allard v. Canada). In response, the Access to Cannabis for Medical Purposes Regulations (ACMPR) replaced MMPR, allowing for limited personal cultivation of marijuana for medical purposes.

Alongside this developing industry, Canada’s current federal government has committed to exploring the legalisation of recreational cannabis. The Task Force on Marijuana Legalization and Regulation was launched in June 2016 and will rely on expert and public consultation to draft recommendations related to this new system. New legislation is anticipated in 2017.

United States:

As of this year, 25 states – as well as the District of Columbia, Guam and Puerto Rico– have legalised marijuana for medicinal use with a recent push in 17 other states to allow a low dosage prescription in limited medical situations.

Despite the progress being made on a state-by-state basis, medicinal marijuana is still in question on an federal level. Last month, the Drug Enforcement Administration (DEA) rejected petitions to remove medicinal marijuana from Schedule I, which rejects marijuana for medicinal use and prohibits doctor prescriptions.

“Marijuana has a legacy reputation. Views are evolving but in the meantime, patients, prescribers and researchers are caught between states that allow medical use of cannabis and the federal government, which maintains that all marijuana use is illegal,” said Al Jackson, EVP Spectrum.

However, the DEA has approved a policy to enact more registered marijuana manufacturers for research purposes. Until this recent change, the University of Mississippi was the only entity in the United States that had the federal license to grow marijuana for medical research.

What’s next?

Long considered a drug that was at best recreational and at worst incredibly harmful, marijuana is starting to see a shift in perceptions as studies are being undertaken to explore its clinical benefits; even big pharma is getting in on the act. One could make the argument that the science is influencing legislation, as studies provide evidence of clinical benefit, more and more governmental bodies are willing to reduce restrictions on the medical use of cannabis. Could this feedback cycle of promising science provide the impetus for medical legalisation in even more countries? Its likely too early to tell, but as seen above, momentum is certainly shifting in favor of fewer restrictions.

What do you think the future of medical marijuana looks like? Sound off in the comments below.

 This blog was a collaborative effort by GLOBALHealthPR Australia partner VIVA! Communications, Canada partner energiPR, and U.S. partner Spectrum.

Today’s post comes to us from our GLOBALHealthPR Australia partner, VIVA! Communications.

Today we are teaming with the Epilepsy Foundation to release new research coinciding with Purple Day – a global, awareness raising initiative for epilepsy. Already today, the Epilepsy Foundation has raised $100,000 for this worthy cause, and is still counting!

New research released by the Epilepsy Foundation today reveals almost one-in-two Australian adults living with the country’s most common, chronic brain disorder are three-times more likely to experience abnormally high levels of anxiety compared to the general population. [Read more…] about Heightening Epilepsy Awareness on Purple Day

How appropriate that GLOBALHealthPR Australia partner VIVA! Communications chose Alexion’s David Kwasha to share his personal and Company views on rare diseases on World Rare Disease Day during our two-day annual general meeting in Sydney, Australia last week.

He challenged our group of leading, independent health-focused public relations firms to do more to advance the cause of rare diseases, and in particular, ultra-rare diseases.

In the U.S., a rare disease affects fewer than 200,000 persons, and an ultra-rare disease, fewer than 6,000.

David seemed happy to know that GLOBALHealthPR has already supported the location and identification of nearly 100 percent more children with Progeria, most definitely an ultra-rare disease in which children age rapidly. To date, about 110 children have been identified living with Progeria, an increase from 50 or so just five years ago. We titled our campaign “Find the other 150” because at the time, about 200 Progeria children were estimated worldwide. [Read more…] about Realizing Rare Opportunities