Trends in rare diseases to watch in the coming year

By: Mark Krueger, MPH

February 28, 2017 marks the 10th international Rare Disease Day and another unprecedented year of success in the fight against rare diseases.  Notable amongst 2016’s accomplishments is the establishment of the new NGO Committee for Rare Diseases under the Conference of NGOs with Consultative Status to the United Nations Economic and Social Council.  This committee will provide an important advocacy platform for rare disease community collaboration with UN agencies, civil society, national governments, academia, and industry to increase visibility and support for people living with rare diseases and their carers. Here are three additional trends to watch in the coming year:

Stronger demand for pre-approval access

Following the passage of 21st Century Cures Act and Right to Try bills in many states as well as the consideration of a reflection on the topic in Europe and changes in some national regulations, industry can only expect increased demand for investigational treatments. While most sponsors understand that planning for early access is an essential part of their development and commercialization plans, increased public and advocate awareness will likely make demand stronger.

More engagement with patient opinion leaders

Social and digital platforms will result in an even stronger and louder voice for patients and their families on many topics from engagement in research and development to debate about the high cost of novel therapies.  As patients become better educated about their disease and health care systems and as they acquire the tools to advocate for themselves, companies are moving from engaging solely with patient groups to connecting directly with independent patient opinion leaders unaffiliated with an established organization.  Sponsors will need to understand the interrelationships between the two and ensure their advocacy plans account for both.

Increased competition from generics

Pioneering rare disease treatments will soon lose their market exclusivity.  There is already some generic competition in the rare disease space and this will only increase.  Patient groups generally have little understanding of advantages and limitations of biosimilars.


We at MK&A are proud of our 20 years of specialized experience in rare diseases.  We have used our knowledge, networks and experience to create countless early access programs, map changing advocacy communities, and educate about the appropriate role of generics and related medications.   You can reach me at if you have any questions or wish to discuss further how these trends can impact the goals of your company.

About the Author:

Mark Krueger, MPH

Mark Krueger, MPH, founded MK&A (Mark Krueger & Associates, Inc.) in 1996 to help pharmaceutical, biotech, device and diagnostic companies partner with patient, consumer and professional groups to speed drug development and approval, promote appropriate use, and ensure equitable access and reimbursement.  Under Mark’s direction, MK&A has created a unique niche in global health care through its singular focus on advocacy.  The firm has demonstrated repeatedly that integration of patient, consumer and professional groups’ viewpoints can create better health care for all.

He can be reached at +1 212 620 2770 or at

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