Developing Drugs for Rare Diseases

Rare diseases pose particular drug development challenges for companies. Issues include: small patient populations that make it difficult to identify and "find" patients with the relevant disease; patient's natural histories are often missing or unavailable; and it is often difficult to justify the investment. In addition, once these products reach the market, state Medicaid and private insurer policies such as prior authorization requirements, use of specialty tiers, and step therapy requirements all restrict patient access to these products.

Post by Michael Werner

Developing Drugs for Rare Diseases Session Panelists:

• Michael Werner -- Holland & Knight LLP – Moderator
• Diane Dorman – Vice President, Public Policy – National Organization for Rare Disorders
• Tim Cote, MD – Director, Office of Orphan Product Development, FDA
• Jayne Gershkowitz – Director, Patient Advocacy and Public Policy
• Amy Waterhouse – Vice President, Regulatory and Government Affairs, BioMarin

Rare diseases pose particular drug development challenges for companies. Issues include: small patient populations that make it difficult to identify and "find" patients with the relevant disease; patient's natural histories are often missing or unavailable; and it is often difficult to justify the investment. In addition, once these products reach the market, state Medicaid and private insurer policies such as prior authorization requirements, use of specialty tiers, and step therapy requirements all restrict patient access to these products.

As a result, all the challenges facing biotech and pharmaceutical companies – such as greater data requirements for product approval, as well as pricing pressures and patient access concerns, are felt even more acutely by companies developing drugs for rare diseases.

An effective way to respond to drug development challenges used by successful companies, is to pro-actively develop mutually supportive relationships with key patient advocacy groups. These efforts improve communication and information sharing between patients and researchers and build trust that lasts throughout the drug development process.

New policy threats are on the horizon. Health care reform proposals and biosimilar legislation pose new challenges for rare disease drug development. Policy makers must ensure that patients will have access to high quality care at a price that is affordable, while creating a climate that promotes innovation and development of the medicines to treat currently unmet medical needs.

In addition, since information about active moieties for orphan indications are already in the public domain, patent protection is often not as strong as for other products. Thus, companies are concerned that without added statutory exclusivity protection, biosimilars legislation will hurt existing incentives to develop drugs for rare diseases.

FDA's Office of Orphan Product Development has launched several initiatives to improve the current situation. Examples include:

• Training programs for FDA reviewers re: alternative clinical trial designs;
• EMEA harmonization activities;
• Analytical activities to better understand the orphan disease environment; and
• Identification of products with orphan designation that have an IND but are not under active investigation.

Despite these efforts, there are other regulatory reforms that would remove development obstacles. For example, harmonizing the disparate approval requirements that currently exist between the US and the EU, increased discussions with FDA regarding study design, and allowing greater input from clinical experts in discussions about surrogate endpoints would ease the drug development burden while still protecting patients.

Michael Werner , Partner Holland & Knight michael.werner@hklaw.com www.hklaw.com