The Great Orphan Drug Debate - Round II

As the temperature in Philadelphia soared into the 90s, a blistering series of debates was taking place indoors as Compass Healthcare Marketers hosted the second installment of their Innovation Lab: Great Orphan Drug Debate series. The debate, which was also sponsored by Medical Marketing Economics (MME) and Idis, featured representatives from Genzyme, BioMarin, Enobia, Pfizer, and GlaxoSmithKline who were in town attending CBI's 7th Annual Rare Disease and Orphan Drug Leadership Congress.

Despite the heat, the debaters remained cool and inspired as they continued the dialogue among more than 35 industry leaders at this closed-door event. Like past debates, this evening proved one of the best ways to connect those in the orphan drug space and discuss some of the challenges impacting our tight-knit community.

Debate #1
Big Pharma vs Biotech: Will They Ruin Everything or Accelerate the Cause?

Big pharma's entrance into the rare disease space has caused some discomfort for biotech companies, patient advocacy organizations, and other stakeholders. This debate focused on the role of big pharma and its impact on the orphan drug community. Will the presence and attention of big pharma hurt the rare disease space? Or will the high profile nature of big pharma's work actually contribute to our community?

Pfizer's Mahesh Kolar argued that since science is evolving, industry should evolve as well to better serve the rare disease space. Big pharma can help fill existing gaps. For example, Pfizer is well positioned to support the rare disease communities with science, policy-making, and the ability to reach ex-US markets. Kolar noted that big pharma has already been involved in rare diseases, but only now are they learning how to talk about that commitment.

Chad Holland opined that biotech companies have more credibility, compassion, and trust within the community than big pharma companies. He believes that biotech companies have more capability and flexibility to serve the patient—starting and ending with their needs in mind. Kolar responded that with a large company it takes longer to steer the ship.

Both Kolar and Holland agreed that the future role of the small to mid-size biotech is still yet to be determined after the acquisition of small biotech companies in recent months.

Debate #2
Expanded Access/Compassionate Use: Is the Effort Worth It?

Expanded access and compassionate use are particularly sensitive for pharma companies in the rare disease space. While they are an effective method to serve individual patients and gain real-world experience, expanded access may limit resources for clinical funding and put future patient programs and even regulatory approval at risk. But what is morally right?

Simon Estcourt of Idis argued against expanded access/compassionate use. He asserted that if a company provides a product through expanded access, it is morally wrong because it is too risky for a company to put the benefit of a few patients above the benefits of the greater community. Completing a trial and bringing a drug to market ensures the drug is available for all patients. If a company follows this process, the whole community benefits.

John Lagus, also of Idis, took the opposite perspective, and noted that social media and the international patient community can increase demand for a drug. The interconnected rare disease communities can magnify the differences in care from country to country.

However, Both Estcourt and Lagus agreed that these decisions often come down to economics. Expanded access or compassionate use is expensive. Companies have a choice to provide a product free-of-charge, or at cost, but determining the cost often has far-reaching implications.

Debate #3:
Can the Orphan Drug Pricing Model Be Sustained? A New Beginning or the Beginning of the End?

Douglas Paul and Jos Pluijmakers, both of MME, argued both sides of the orphan drug pricing model. Paul noted, “rare is the new black” since many pharma companies are focusing more on rare therapies. However, despite this newfound focus, and to put orphan drugs in perspective, Lipitor had more sales than all orphan drugs combined. As a matter of fact, orphan drugs account for only 5% of pharma spending, according to Pfizer.

Together, Paul and Pluijmakers discussed the philosophical approach to our healthcare system, and how this reflects our society's values. In the end, both agreed “no patient left behind” is part of our social contract—it's in our DNA.

Where do we draw the line? (For example, how much is an additional month of life worth in the rare oncology space?) How do we meet those needs? What other decisions will be determined as we move forward? In the end, Paul and Pluijmakers ran out of time, but stay tuned for the next series of conversations at Compass Healthcare Marketers' annual Innovation Lab in Cavallo Point on October 25.