Home Heart Disease Treatment An Unwarranted Attack On Rare Disease Research

An Unwarranted Attack On Rare Disease Research

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Dr. Milton Packer is a noted cardiologist at the Baylor University Medical Center who has been a key investigator in many important clinical trials on heart failure drugs. On the Baylor website it is said that “his research established the cornerstone of the current modern treatment of heart failure.” This isn’t hyperbole.

But, Dr. Packer has recently savaged a different medical area - research into treatments of rare diseases. As defined by the National Institutes of Health (NIH), rare diseases are ones that afflict fewer than 200,000 individuals. Amazingly, there are 7,000 such known diseases. While some are exceedingly rare, some are quite well known: muscular dystrophy, cystic fibrosis and multiple sclerosis. In fact, many forms of cancer can be considered rare diseases. While termed “rare”, the patients with such diseases aren’t. The NIH estimates that there are 25 million Americans so impacted.

Being a physician, one would think that Dr. Packer would be sympathetic to the plight of these patients in desperate need of treatment. That unfortunately doesn’t appear to be the case. In a recent op-ed piece for Medpage Today entitled “Has Drug Development for Rare Diseases Reached an Extreme?”, he makes some provocative statements about R&D in this area:

1) “the pharmaceutical industry is obsessed with them (rare disease drugs)”;

2) “the FDA is Iess fussy about the kind of data that it requires for approval for a new drug for a ‘rare’ disease”;

3) “pharmaceutical companies have found that they can charge exorbitant prices for drugs that target a rare disease”;

4) “if a company decides to develop a new drug for a rare disease, the costs of development will be comparatively low, the return on investment can be enormous, and the sponsor will have marketing exclusivity for many years.”

It sounds like the greedy drug companies are up to their old tricks again. Or are they? Let’s examine Dr. Packer’s claims.

The pharmaceutical industry’s obsession with rare disease drugs is a bit of an exaggeration. Take Pfizer, a company for which Dr. Packer has been a consultant, as an example. In Pfizer’s most recent pipeline update (July 31st), 98 programs are listed in their pipeline. Of these only 13 are rare disease drug programs, which stands in contrast to 43 oncology programs and 22 inflammation/immunology programs. If rare disease research is such a “gold mine for the pharmaceutical industry”, you would think that Pfizer would invest more than 13% of its R&D dollars into this area. Yes, companies have entered this research space, but for most it represents a relatively minor part of their portfolio.

Is the FDA really “less fussy about the kind of data that it requires for approval of a new drug for a rare disease“?

For anyone who has had dealings with the FDA on new drug application, it’s hard to believe the FDA is less fussy in ANY of its reviews of New Drug Applications. Certainly, last year’s very public debate about Sarepta’s Duchenne muscular dystrophy drug, eteplirsen, shows the challenges that the FDA has in balancing the safety/risk profile of a rare disease drug versus the demand that patients have for access to a new medication for which they clamor. What is true is that the FDA can more easily monitor the performance of a rare disease drug once it is approved especially when less than 1,000 patients will be prescribed it. Thus, the FDA might view the risk/benefits of a rare disease drug differently from a drug likely to be prescribed to tens of thousands of patients immediately after launch.

What about the claim that “pharmaceutical companies can charge exorbitant prices for drugs that target rare diseases”? It is true that these drugs command a high price. The rationale is that even at a high price, these drugs can save the healthcare system significant money. If an untreated rare disease costs the healthcare system $200,000/patient/year, and a new drug for this disease not only ameliorates the disease, but also reduces other associated healthcare costs, a price of $100,000/patient/year can be a bargain – as well as help the patient. Interestingly, the biggest critics of drug prices – payers - can accept high prices for rare disease drugs. Recently, Ultragenyx launched Crysvita, a transformative X-linked hypophosphatemia treatment for this genetic bone disorder that improves the health of both children and adults. The price for Crysvita was set at $160,000/year for children and $200,000/year for adults. Dr. Steve Miller, the chief medical officer of Express Scripts and a noted critic of drug pricing, said the following about Crysvita’s price: “It’s not inexpensive, but I do believe that the right word is responsible.” There is no doubt that the pharmaceutical industry’s rationale in carrying out R&D in rare diseases is driven by the high prices these drugs can command. But such prices are needed to justify their commercial viability due to the small patient populations typical of these diseases. Dr. Packer’s inference of price gouging is unfair.

Finally, with respect to market exclusivity, certainly Dr. Packer must be aware that all drugs, be they for rare diseases or for what Dr. Packer calls “common diseases”, get the same length of patent exclusivity. Furthermore, there can be tremendous competition in rare disease R&D leading to the potential for multiple entrants to treat specific rare diseases – competition that also can drive down prices.

Dr. Packer’s attack on rare disease R&D might stem from the decline of R&D dollars into heart failure - the focus of his own clinical research career. This is a valid concern, but the rationale for the decreased focus on heart disease in major companies is understandable. To better understand this, it is instructive to examine the experience that Novartis has had with its heart failure drug, Entresto. Entresto is a breakthrough medication that, in a trial of 8,442 heart failure patients called PARADIGM-HF, reduced the risk of death from cardiovascular causes or hospitalizations due to worsening heart failure by 20% - a remarkable number. (Dr. Packer was a lead investigator in this study.) A study such as PARADIGM-HF is expensive and risky as it probably cost hundreds of millions of dollars and success is not guaranteed due to the high bar for success.

Novartis’s roll of the dice proved to be a winner. The company subsequently priced the drug at $12.50/day or about $4,500/year. Clearly, this is far less than the annual price of a rare disease drug, but given the enormous number of patients with heart failure, this drug was anticipated to be a major commercial success. Yet, Novartis’ revenues for 2017 for Entresto were only $507 million – a major disappointment given early expectations. Novartis did all the right things with the Entresto development program. However, this shows the challenges in doing R&D in the so called “common diseases”. A company has to go through great lengths to demonstrate the superiority of a new drug over older, generic and very good drugs. And, even if you can achieve this, payers and physicians may still not be convinced to utilize a new drug over existing viable treatments. Given these challenges, to focus all of a company’s R&D resources on “common diseases” would not be prudent.

Finally, Dr. Packer’s lack of empathy for rare disease patients is disappointing. Hearing a parent agonize over the plight of their child who suffers from a rare disease is heart wrenching. For these families, research into rare disease drugs hasn’t come close to “reaching an extreme”.

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