With Rare Disease Day Comes Opportunity and Responsibility

By: Mark Krueger, MPH

February 29, 2016 is Rare Disease Day around the world, marking another year of advancement and improvement in diagnosis and treatment of men, women and children who are too frequently ignored. We at MK&A (Mark Krueger & Associates, Inc.) salute and applaud patients, families, and their partner organizations for their design and support of basic and clinical research, their willingness to participate in clinical trials and their provision of support services as well as their advocacy for more responsive policies with elected and appointed officials.

For the second consecutive year, the US Food and Drug Administration approved more drugs for rare diseases than ever before in its history. In 2015, nearly half (47 percent) of all novel drugs approved in the US (21 of 45) were for rare diseases.1 The European Medicines Agency also had a record breaking year, recommending the highest number of orphan-designated medicines. Among those 93 drugs, 18 had an orphan designation.2 Never before have there been so many approved drugs targeting rare diseases, and through continued and novel partnerships with industry and in many cases patient group-led research networks these numbers will continue to grow.

The theme of Rare Disease Day 2016 is “Patient Voice,” and the events of the day are centered on amplifying the impact of patients and their families. It is important to acknowledge how far we have come in representing the patient voice through leading organizations such as CORD, EURORDIS, Global Genes, NORD and other impressive groups. However, it is equally important to reflect upon the work that is yet to be done to address the needs of people living with rare diseases for improved access to treatment.

Cost remains a barrier in the access to rare disease therapeutics for many patients. On February 24-25, 2016 EURORDIS and its partners will hold a multi-stakeholder symposium on improving patients’ access to rare disease therapies. During this meeting industry, patient leaders, academics, regulators and payers will come together to discuss the current state of play and how to shape a more effective way to address value determination, appraisal, pricing and reimbursement of orphan medicines. MK&A commends EURORDIS for continued committed to bring these issues to the forefront, to promoting honest dialogue in an age of budgetary constraints and recognition of the necessity of demonstrating value. This meeting will set the stage for all stakeholders to work together in creating the much needed innovative solutions to pricing and access.

  1. Novel Drugs Summary 2015. US Food and Drug Administration
  2. Human Medicines Highlights 2015. European Medicines Agency


About the Author:

Mark Krueger, MPH

Mark Krueger, MPH is president of MK&A.  He can be reached at +1 212 620 2770 or at mkrueger@mkanda.com.

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