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Rare Diseases competition

Rare Diseases competition: a sustainable model for the pharmaceutical industry

GSK Rare Diseases was set up in 2010 as a dedicated unit to discover, develop and ultimately deliver new and innovative medicines to treat rare diseases. Our ambition is to create a sustainable pipeline and a portfolio of medicines that deliver real value to patients living with often devastating and life-threatening rare diseases.

Patients with rare diseases are currently a significantly underserved population, with insufficient social and medical care.

Recent scientific advances, particularly in the field of genetics, has allowed scientists to pinpoint the source / cause of many rare diseases, making it possible to identify those which could benefit from therapeutic intervention and what mechanisms / approaches have the highest probability of success. However advancing these innovative medicines will require large investments in research and development (R&D).

The small numbers of people affected by individual rare diseases makes it challenging to recoup this R&D investment. In addition, healthcare systems in many countries are already facing funding challenges. This all contributes to an ongoing debate about the sustainability of R&D and access models for treatments for rare diseases.

Announcing the winners of Rare Diseases competition

To further understand how the pharmaceutical industry can develop and deliver medicines of value for rare diseases, we invited schools of public health, business schools, students, scholars, health outcome/economics specialists and individuals with an interest, to submit their views and insights on the subject "Rare Diseases: a sustainable model for the pharmaceutical industry".

We organised the competition to stimulate external, strategic perspectives on how companies can develop and deliver novel medicines for an underserved population.

The top three papers and a further seven shortlisted submissions, which can be read below, captured the diversity of views on the sustainability of R&D in rare diseases and potential access models for the pharmaceutical industry.

Winning perspectives included the need to empower and partner with patients in the drug discovery and development process. Pharmaceutical companies were encouraged to create innovative organisational structures designed to maximise the value of internal assets. And policymakers were urged to create hybrid frameworks, incentivising firms to invest in rare disease R&D through a combination of push and pull incentives.

We hope the competition will stimulate debate about sustainable rare disease business models, ultimately leading to the successful delivery of new orphan drugs. We will consider the recommendations of winning papers to ensure that the GSK Rare Diseases model incorporates the best strategic thinking.

Submissions were assessed by a review committee comprising experts from industry, the patient community and the financial sector.

Rare Diseases competition: a sustainable model for the pharmaceutical industry

Announcing the winners of Rare Diseases competition

The top three papers

Further shortlisted papers

More on GSKvision

In conversation with GSK Rare Diseases

Congratulations to the competition winner