An Interview With Pfizer on the Orphan Drug Industry: Part II

In Advocacy, Clinical Development, Market Access, Partnering & Investment, Regulation & Government by Matt Turner


In follow up to Part I of our recent interview with Adam Woodrow, VP of Global Commercial Development of the Specialty Care Business Unit at Pfizer, the remainder of the dialogue provides insight into streamlining the clinical development of orphan drugs and the importance of long term sustainable rare disease research:

How can orphan drug developers streamline their orphan clinical development and overcome clinical hurdles?

Discovering and developing treatments for rare diseases and genetic disorders is a key component of our overall strategy, and Pfizer has research, medical and commercial units that focus exclusively on driving innovation in rare diseases.

Pfizer’s approach is to create a balanced and robust portfolio of medicines based on unmet need, scientific tractability and commercial viability, aligned with the strengths and expertise we have in specific areas of research, development and commercialization. We have prioritized areas of focus in hematology, neuroscience, neuromuscular disorders, pulmonology and oncology to ensure the greatest chance of successfully delivering needed medicines.

By partnering with patient communities and advocacy organizations, we hope to raise awareness of rare diseases, aid in diagnosis, ensure access to treatments, and advocate for supportive policy, regulatory and reimbursement environments. Pfizer is also in active dialogue with healthcare stakeholders and the communities we serve to help create solutions that may provide increased access to treatments for patients in need. Additionally, our global network and reach allows us to educate physicians on the disorder in countries where the disease exists and the awareness is low.

Further, Pfizer is committed to continuing conversations with regulatory bodies regarding validated endpoints for clinical trials, as well as understanding more about the natural history of specific diseases.

What can developers do to continue a sustained development of orphan drugs through the clinic to global rare disease patients?

Partnerships—be they with commercial organizations or with patients, advocates, regulatory authorities and academic researchers—will be an important aspect to achieve this goal. While the binding commonality in most rare diseases is the low incidence and prevalence of an individual condition, each of those diseases is benefitted by a community of stakeholders, each with unique expertise, specialized relationships and resources, and important perspectives.

Of note, scientific and regulatory advances will also have a role in transforming the market for medicines to treat rare diseases. Some regulatory authorities are increasingly receptive to the use of biomarkers and surrogate endpoints in the clinical study of medicines to treat rare diseases, which will help provide a path to demonstrate appropriate efficacy in small populations. And, as the underlying genetic root of illness is better understood, even common conditions will be increasingly understood as a collection of “rare” subtypes, which may require different, more targeted therapeutic approaches.

It is important to continue to foster and build relationships with key constituents, keep the lines of communication open and actively understand the goals and needs of the regulatory bodies in various countries.

Adam Woodrow will be presenting on The Collaboration Imperative: why cross-stakeholder partnerships are essential to advancing progress in orphan medicines development at the forthcoming World Orphan Drug Congress in Geneva.

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