Why rare disease strategy needs a connected view

Consumer Health

Why rare disease strategy needs a connected view

Rare disease strategy presents a distinctive challenge.

In the United States, an estimated 25–30 million people live with a rare disease, yet that population is spread across more than 7,000 conditions. Individual patient groups can be small, routes to diagnosis can be long and inconsistent, and many conditions have no approved treatment.

Given the complicated and sometimes unknown challenges these patient groups face, it is key to look beyond simply reaching the small, standalone groups, and instead working to understand the connected experiences that shape their needs and decisions.

Disconnected perspectives create strategic blind spots

The patient journey, caregiver experience and HCP decision pathway develop in parallel, but they are often researched and planned separately. That creates an information gap for marketers: it becomes harder to see where needs align, where barriers compound and where communications or support could have the greatest effect.

A patient may need help finding a specialist before diagnosis, clearer information when considering treatment options, or practical support as the condition affects daily life. A caregiver may be coordinating appointments and influencing treatment decisions while also trying to find accurate information and balance other responsibilities. At the same time, an HCP may be navigating referrals, treatment availability, and piecing together information from multiple sources.

These are connected experiences. Rare disease strategy is stronger when they are understood together.

But how can perspectives be connected effectively?

When developing a rare disease strategy, you should consider:

The full journey
Where do delays, misdiagnosis, referrals, treatment changes, adherence challenges and financial pressures occur, and how do information needs change along the way?
2. Where perspectives intersect
How do patient and caregiver needs align—or conflict—with the clinical, access and practical factors influencing HCP decisions?
3. Meaningful differences within each group
Which demographic, attitudinal, behavioural, care-pathway and professional factors create distinct patient, caregiver and HCP groups?
4. Information and engagement preferences
Which sources, formats, channels and communities are trusted and used at different stages of the journey?
5. Changing digital behaviour
How are AI, search, social media and professional digital tools affecting information-seeking, expectations and clinical conversations?

Connecting both sides of the rare disease journey

M3 MI has created a new syndicated solution for 2026: the Rare Disease Studies.

Covering more than 90 conditions, the patient and caregiver study comprises of more than 1,000 respondents, and the HCP study includes more than 570 respondents. Both studies contain over XXX data points, examining a variety of related topics, with some repeated questions unlocking multiple perspectives, and some distinct questions reflecting individual experience.

More than 90 conditions are covered, grouped across 8 therapeutic area clusters:

Autoimmune/immune disorders
Blood disorders
Cardiac conditions
Growth disorders
Infectious diseases
Metabolic diseases
Neurology Oncology

Bringing the full picture into focus

By combining insight on the full journey, intersecting perspectives, group differences, engagement preferences and changing digital behaviour, the Rare Disease Studies turn a fragmented picture into a more connected basis for action.

Whether used to shape support, address barriers, guide content and strategy or strengthen planning, each study delivers practical value independently. But together, they reveal where patient needs, caregiver influence and HCP decision-making meet, helping teams develop more relevant, evidence-based rare disease strategies.

Find out more

M3 MI’s 2026 Rare Disease Studies are now available – read more here or contact the team to find out more.

Source: M3 MI’s 2026 MARS Consumer Health Study.

Information

For more information on detailed study results from M3 MI, including at the specialty level, contact us at
info@M3-MI.com.

Consumer Health