Late Phase Solutions Europe AB

Increased focus on Orphan Drugs
Orphan drug legislation has been in place since 1983 in the USA and since 2000 in Europe. The objective of introducing the legislation was to stimulate and motivate the pharmaceutical industry to develop treatment for patient with rare diseases. Many companies from small biotech companies to big pharma are today showing a growing interest in developing drugs for rare diseases. Before the legislation was put into place there were only a handful drugs approved for patients with rare diseases. Today almost 450 indications have been approved by FDA in US and about 80 by EMA in Europe.

Challenges related to developing Orphan Drugs
There are several challenges involved in developing orphan drugs. The clinical trial design are often one of the most prominent depending e.g. on;

  • few available patients;
  • lack of knowledge of the natural history of the disease and optimal study endpoints.

Many orphan drugs are approved conditionally or under exceptional circumstances with post-marketing obligations for the manufacturer to further establish the safety and long-term benefit of the new treatment and therefore; Real World Data is a fundamental for developing Orphan Drugs:

  • Typically safety and effectiveness of orphan drug in the post-marketing phase is demonstrated through registries;
  • By collecting data into patient registries not only the post-approval commitments can be fulfilled but also increasing knowledge about the rare disease and its optimal treatment;
  • With so few available patients as for rare diseases it is important to avoid duplication in data collection and a public private partnership is strongly encouraged within the rare disease community.

Late Phase Solutions Europe; an Independent International Expert in Real World Data and Orphan Drugs

Late Phase Solutions Europe AB provides you with strategic and operational guidance on Late Phase Drug Development with a focus on Real World Data on orphan drugs both from an international as well as national perspective.

Late Phase Solutions Europe has an in-depth expertise in orphan diseases and drugs as well as designing and managing patient registries within the orphan drug area and has a proven track record of successful collaboration with patient registry stakeholders (patient organizations, treating physicians, authorities, payers) by assuring registry output fulfills their different needs as well as the ability to connect pharma business opportunities with patient registry design, management and outcome.

Our offer includes strategic advice, guidance and project management on processes and activities related to Orphan Drug Development and Market Access.

Our Customers
Our customers are big pharma companies as well as small startup pharma and biotech companies all with a focus on patients with rare diseases and the high degree on unmet medical needs within this area.

Our Services
We provide you with more than thirty years of experience from International Biotech and Pharmaceutical Industry; whereof near twenty in the orphan drug sector and late phase drug development. We have a broad network of international expertise and key stakeholders within the global orphan drug community and can therefore tailor the solution for you by e.g. partnering with relevant stakeholders and an optimal EDC provider/CRO for your specific needs.

Examples on assignments are:

  • Observational studies/ patient registries to collect real world data to fulfill the requirement of conditional market authorization or reimbursements of orphan drugs;
  • Guidance and support in establishing data collection frameworks for internationally interoperable multi-purpose usage;
    – post approval commitments
    – supporting clinical research
    – increase knowledge of the natural history of rare disease
    – improve diagnosis, treatment and care of patients with rare diseases;
  • Guidance and support in the process of developing and implementing patient registry charters and governance principles in partnership with registry stakeholders as e.g. academia and patient advocacy groups.


The founder of Late Phase Solution Europe AB Elizabeth Hernberg-Ståhl has published numerous scientific papers, contributed to books related to orphan drugs. Soon (October 2013) the book “Orphan drugs: Understanding the rare disease market and its dynamics” (Woodhead Publishing Series in Biomedicine ISBN 1 907568 09 3 ISBN-13: 978 1 907568 09 1 authors Elizabeth Hernberg-Stahl, Miroslav Reljanovic) will be released. Elizabeth is also a frequent speaker and trainer at many international venues, organized by the industry, patient organization or other stakeholders within the field of rare diseases and orphan drugs.